Hereditary neurological disease

This study tests a new gene therapy called DB-OTO for children with hearing loss caused by changes in the otoferlin gene. The treatment is injected into the inner ear to help restore hearing. Up to 30 children and infants will take part to check safety and how well it works.

This Phase 1 trial tests an experimental vaccine called AV-1980R that targets tau protein, a key player in Alzheimer's disease. The study enrolls 48 cognitively healthy adults aged 65-80 who have biological signs of early Alzheimer's. Researchers will check if the vaccine is safe…

This phase 3 trial is testing whether adding a targeted therapy called Sacituzumab tirumotecan (Sac-TMT) to standard chemotherapy and immunotherapy (pembrolizumab) can better eliminate cancer before surgery in people with high-risk, early-stage triple-negative or hormone-receptor…

This study tests brain surgery as a treatment for people whose epilepsy doesn't improve with medication. About 300 children and adults aged 8 and older will have surgery to remove or treat the part of the brain causing seizures. The goal is to stop or greatly reduce seizures, tho…

This study tests whether clemizole HCl (EPX-100) can safely reduce seizures when added to current treatments for Lennox-Gastaut syndrome, a severe form of epilepsy. About 260 children and adults aged 2 to 55 will receive either the drug or a placebo. The main goal is to measure c…

This Phase 3 trial tests an oral drug called gildeuretinol acetate (ALK 001) in 230 people aged 8 to 45 with Stargardt disease, a genetic condition that causes progressive vision loss. Participants take the drug or a placebo daily for 24 months. The goal is to see if the drug can…

This study tests an oral medication called LUM-201 for children with growth hormone deficiency who have never been treated before. The goal is to see if taking a pill every day for 12 months can improve growth rate compared to a placebo. The study involves 150 prepubertal childre…

This study tests whether adding a personalized mRNA vaccine (V940) to standard immunotherapy (pembrolizumab) can stop advanced melanoma from growing or spreading. About 160 people with stage III or IV melanoma that cannot be removed by surgery will receive either the vaccine plus…

This study follows about 300 people with Duchenne muscular dystrophy who are taking the drug givinostat as part of their normal care. Researchers will track safety issues like low platelet counts and bleeding, and see how patients' muscle function changes over up to 5 years. The …

This study tests whether programming a cochlear implant based on each person's unique inner ear structure improves hearing compared to the standard one-size-fits-all approach. Adults with severe to profound hearing loss who already have a MED-EL cochlear implant will receive both…

This phase 3 trial tests whether adding the experimental drug sacituzumab tirumotecan (sac-TMT) to standard immunotherapy (pembrolizumab) and a targeted therapy (bevacizumab) helps people with metastatic cervical cancer live longer. About 1,000 participants whose cancer has sprea…

This study tests the drug abemaciclib, typically used for cancer, in people with neurofibromatosis type 1 (NF1) who have atypical neurofibromas that cannot be surgically removed. The goal is to find a safe dose and see if it can shrink or stabilize these tumors. Participants take…

This study tests two experimental drugs, sapanisertib and serabelisib, combined with standard therapy for people with HR+/HER2- advanced or metastatic breast cancer. The goal is to find safe doses and see if the combination can shrink tumors or slow the disease. About 32 adults w…

This phase 3 trial tests whether adding the experimental drug MK-2870 to the standard immunotherapy pembrolizumab helps people with advanced non-small cell lung cancer live longer compared to pembrolizumab alone. The study enrolls about 614 adults whose tumors have high PD-L1 lev…

This study investigates whether a homemade bottle-based breathing device can strengthen the muscles used for exhaling and improve balance in people with Parkinson's disease. Parkinson's often weakens respiratory muscles, leading to breathing and coughing problems. The trial will …

This study tests a new automated method to fine-tune adaptive deep brain stimulation (aDBS) for people with Parkinson's disease. aDBS uses brain signals to adjust stimulation in real time, but setting it up is complex. The trial will compare automated aDBS to standard continuous …

This study is checking the long-term safety of an implant called OTX-TKI (axitinib) for people with wet age-related macular degeneration (AMD). The implant is placed in the eye and slowly releases medicine to control the disease. About 850 people who already completed two years o…

This Phase 3 study tests a drug called zorevunersen in 170 children aged 2 to 17 with Dravet syndrome, a severe form of epilepsy. The drug is given as an injection into the spine and compared to a sham procedure. The goal is to see if it reduces seizures and improves daily living…

This phase 2 trial tests a powerful mix of short-course radiation, chemotherapy (mFOLFOX6), an immunotherapy (PD-1 antibody), and a targeted drug (cetuximab or bevacizumab, depending on genetic markers) in people with high-risk rectal cancer that is mismatch repair proficient (pM…

This clinical trial tests an oral drug called SKY-0515 in 400 adults with Huntington's disease. The goal is to see if it can lower harmful proteins linked to the disease and improve symptoms like movement and thinking. Participants take the drug or a placebo daily for 18 months.

This study checks the long-term safety and lasting effects of a gene therapy called IPS101A in 6 people with severe Parkinson's disease who already received it in an earlier trial. Researchers will monitor side effects and measure changes in movement symptoms over time. The goal …

This early-stage trial tests whether implanting specially grown dopamine-producing cells into the brain is safe and can help people with Parkinson's disease. Six participants receive their own lab-grown cells placed into a brain region that controls movement. The study measures c…

This study tests an experimental drug, fosigotifator, in adults and children with Vanishing White Matter disease, a rare genetic brain disorder. The main goals are to check the drug's safety and how the body processes it. About 50 participants will take the drug for up to 201 wee…

This Phase 3 trial tests whether adding immunotherapy and a targeted drug to short-course radiotherapy and chemotherapy improves outcomes for people with high-risk rectal cancer. About 204 participants will receive either the standard treatment or the intensified combo. The study…

This study tests a single injection into the eye of a new drug (SAR446597) for people with geographic atrophy, a progressive form of age-related macular degeneration that causes vision loss. About 104 participants aged 60 and older will be followed for 2 years, with an optional 3…

This Phase 2 trial tests an oral drug called NEU-411 in 150 adults aged 40-80 with early Parkinson's disease who have a specific genetic marker (LRRK2-driven). The study is double-blind and placebo-controlled, meaning neither patients nor doctors know who gets the real drug. Rese…

This study is testing an experimental drug called DNL628 in people with early-stage Alzheimer's disease. The main goal is to see if the drug is safe and how the body processes it. About 68 participants will receive either DNL628 or a placebo. This is an early-phase trial, so it f…

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This early-stage trial tests a new gene therapy called SKY-GJB2 for children aged 9 months to 7 years with hearing loss caused by changes in the GJB2 gene. The therapy is given as a single injection into the inner ear using a special delivery device. The study will enroll 10 chil…

This study tests a drug called tivozanib in people with bile duct cancer that cannot be removed by surgery and has already been treated with chemotherapy. The goal is to find the safest dose and see if the drug can shrink tumors or slow the cancer. Participants take tivozanib by …

This study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and …

This study tests a new medicine called tiespectus (EYE201) for wet age-related macular degeneration, a leading cause of vision loss. About 960 people will receive either tiespectus or the standard treatment aflibercept via eye injections. The goal is to see if tiespectus can impr…

This study tests whether HBS-301 (pitolisant) can reduce excessive daytime sleepiness and cataplexy (sudden muscle weakness) in adults with narcolepsy. Participants receive either the drug or a placebo for several weeks, followed by an open-label phase where everyone gets the dru…

This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is v…

This study tests an experimental drug called EPX-100 (clemizole) in 150 children and adults with Dravet syndrome, a severe form of epilepsy. The goal is to see if adding EPX-100 to current treatments can safely reduce the number of motor seizures. Participants will be randomly as…

This study tests a new drug called tiespectus (MK-8748) against the current standard treatment, aflibercept, for people with wet age-related macular degeneration (AMD). About 960 participants will receive injections into the eye and be followed for a year to see if tiespectus can…

This study tests whether a special probiotic supplement can change fat-related molecules in the blood of people with ALS and frontotemporal dementia (FTD). Researchers will compare those taking the probiotic to those taking a placebo over 24 weeks. The goal is to see if the probi…

This study follows 300 adults with Parkinson's disease, essential tremor, or dystonia who receive deep brain stimulation (DBS). DBS uses a device implanted in the chest to send electrical pulses to brain areas controlling movement. Researchers will track symptom severity, medicat…

This early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body proc…

This phase 3 trial tests whether a new drug (MK-2870), alone or with another drug (pembrolizumab), works better than standard treatments for people with a common type of advanced breast cancer (HR+/HER2-). About 1,200 participants whose cancer has worsened after hormone therapy w…

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This study tracks how well the drug Koselugo (selumetinib) works and what side effects it causes in people with Neurofibromatosis Type 1 and painful nerve tumors. About 200 patients in South Korea will be followed during their normal doctor visits. The goal is to confirm the drug…

This Phase 2 study tests whether a single dose of AAV2-GDNF gene therapy, delivered directly into the brain, can improve motor function in adults with moderate Parkinson's disease. About 127 participants will receive either the gene therapy or a sham surgery. The main goal is to …

This study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic …

This early study tests whether a smart brain implant can help people with spinocerebellar ataxia type 6, a condition that causes trouble with balance and coordination. Five adults will get the implant, which records brain signals and adjusts stimulation automatically. The goal is…

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This study tests a new radioactive drug, [212Pb]VMT-alpha-NET, in people with advanced gastrointestinal neuroendocrine tumors or pheochromocytoma/paragangliomas that have already been treated with radiation therapy. The drug is designed to attach to a protein on the surface of th…

This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This early-stage trial tests a new medicine called EYC-0305, given as an eye injection every 24 weeks, in 30 people aged 50 and older with wet age-related macular degeneration (AMD). The main goal is to check if the treatment is safe and tolerable. Researchers will also measure h…

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to…

This study tests whether a new drug, sacituzumab tirumotecan (Sac-TMT), given alone or with pembrolizumab, helps people with advanced triple-negative breast cancer live longer or keep their cancer from growing. About 1,000 adults whose cancer has spread and who have not had prior…

This study tests a gene therapy called ABBV-RGX-314 for people with wet age-related macular degeneration (AMD), a leading cause of vision loss. The goal is to see if a single treatment can reduce the need for frequent eye injections while keeping vision stable. About 561 adults a…

This early-stage study tests a new drug called RG6496 in people who carry the gene for Huntington's disease. The main goal is to check if the drug is safe and how the body handles it. About 40 participants will receive a single dose injected into the spine, with some getting a pl…

This study tests a drug called radiprodil in people with GRIN-related neurodevelopmental disorder, a rare genetic condition that often causes seizures and developmental delays. The trial includes two groups: one with frequent seizures and one with fewer or no seizures. Participan…

This early-stage study tests a new medicine called PYC-001, given as an injection into the eye, for people with a rare genetic eye disease (autosomal dominant optic atrophy) caused by a change in the OPA1 gene. The main goal is to check the safety of different doses and schedules…

This Phase 2 study tests atacicept, a drug that may reduce immune system attacks on the kidneys. About 250 adults with various autoimmune kidney diseases (like IgA nephropathy or membranous nephropathy) will receive weekly injections. Researchers will check safety and measure cha…

This study tests a new targeted therapy pill called MK-1084, alone or with other drugs, for people with advanced non-small cell lung cancer that has a specific genetic change called KRAS G12C. The goal is to see if the treatment is safe and can shrink tumors. About 130 participan…

This study tests whether adding an experimental mRNA vaccine (V940) to the immunotherapy drug pembrolizumab (Keytruda) can keep lung cancer from returning after surgery. It includes 680 people with stage II to IIIB non-small cell lung cancer whose tumors did not fully respond to …

This Phase 2 trial tests FWY003, an experimental drug, in 272 people with geographic atrophy (a form of advanced age-related macular degeneration). The goal is to see if different doses can slow the growth of damaged areas in the eye and preserve vision. Participants receive eith…

This phase 3 trial compares a new drug, BMS-986365, against standard treatments (docetaxel or other hormone therapies) in 960 men with metastatic castration-resistant prostate cancer that has stopped responding to hormone therapy. The main goal is to see if the new drug delays ca…

This phase 2 study tests belzutifan, a daily pill that blocks a protein called HIF-2α, in people with advanced rare cancers like pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumors, VHL disease-associated tumors, and certain other solid tumors. The goal is to see if …

This study tests several new drug combinations, with or without the immunotherapy pembrolizumab, in people with advanced esophageal cancer that has continued to grow after standard treatment. About 230 participants will receive different drug regimens to see which are safest and …

This study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

This phase 2 trial is testing whether giving the immunotherapy drug pembrolizumab (Keytruda) along with chemotherapy before surgery can eliminate all signs of cancer in people with resectable stage II to IIIB non-small cell lung cancer. About 60 participants will receive the comb…

This study tests an adaptive deep brain stimulation (aDBS) system that automatically adjusts stimulation based on real-time brain signals. It aims to improve symptom control for 62 people with Parkinson's disease in China who already have a compatible brain implant. The main goal…

This study tests whether a steroid injection given during retinal detachment surgery can replace the usual course of antibiotic and steroid eye drops afterward. About 168 adults with a first-time retinal detachment will receive either the injection or standard drops. The goal is …

This study tests a gene therapy called AAVB-039 for Stargardt disease, an inherited condition that causes vision loss. About 75 adults with a specific gene mutation will receive a single injection under the retina. The main goal is to check safety, but researchers will also measu…

This study tests a new drug called tulmimetostat combined with standard hormone therapies (darolutamide or abiraterone) in men whose prostate cancer has spread but still responds to hormone treatment. The goal is to see if the combination is safe and works better than standard ca…

This study is for adults with recurrent glioblastoma, an aggressive brain cancer. It tests whether adding a precise type of radiation (stereotactic radiosurgery guided by a special PET scan) to a device that delivers electric fields to the brain (TTFields) helps people live longe…

This phase 3 trial is testing whether a targeted drug (trastuzumab deruxtecan) combined with immunotherapy (rilvegostomig or pembrolizumab) works better than standard chemotherapy plus pembrolizumab for people with advanced or recurrent endometrial cancer that has a specific prot…

This early-stage trial tests a new oral drug called MF1 in healthy adults and people with Parkinson's disease. The drug aims to block a harmful protein (alpha-synuclein) linked to Parkinson's. The main goal is to check safety and how the body processes the drug, not yet whether i…

This study tests a new medicine called ION337 in children aged 2 to 12 with Dravet syndrome, a severe form of epilepsy. The main goal is to see if the drug is safe and tolerable when given as a spinal injection. Researchers will also measure how the drug moves through the body an…

This early-stage trial is testing whether specially engineered immune cells (called CAR-T and CTL cells) and a dendritic cell vaccine can safely treat people with neurofibromatosis or schwannomatosis, conditions that cause nerve tumors. The study will enroll 100 participants aged…

This extension study follows people with von Hippel-Lindau (VHL) disease or advanced solid tumors who are already taking belzutifan in another trial. The goal is to see how long they live and how safe the drug is over the long term. About 450 participants will take belzutifan by …

This study tests a new drug called ARO-MAPT-SC in healthy people and those with early Alzheimer's. The goal is to check if it is safe and how the body processes it. Participants receive either the drug or a placebo by injection under the skin. The trial is still in early stages, …

This Phase 2 trial tests whether adding APL-3007 to the existing drug Syfovre (pegcetacoplan) can better slow geographic atrophy, an advanced form of dry age-related macular degeneration that causes vision loss. About 240 adults aged 60 and older with the condition will receive e…

This study looks at switching people with wet age-related macular degeneration (AMD) to a higher dose (8 mg) of the drug aflibercept. The goal is to see if it can extend the time between eye injections while still controlling the disease. Researchers will compare injection interv…

This study follows 120 adults with advanced Parkinson's disease in Belgium who are prescribed a drug called Foslevodopa/Foscarbidopa. The drug is given as a continuous infusion under the skin to help control symptoms. Researchers will track how much 'off' time (when symptoms like…

This study tests several new drug combinations, including pembrolizumab (an immunotherapy), with or without chemotherapy, in people with advanced non-small cell lung cancer who have not had prior treatment. The goal is to see if these combinations can shrink tumors and control th…

This phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely impro…

This study compares Tai Chi and Qi-Gong, two gentle movement therapies, to see which better improves balance and motor function in people with stage I Parkinson's disease. Fifty participants aged 50 to 65 will receive either Tai Chi or Qi-Gong alongside standard physical therapy …

This study is testing a device called a flow modulator to treat wide-necked brain aneurysms. The device is placed inside the artery to redirect blood flow away from the aneurysm, helping it close off. Researchers will check if the device is safe and effective in 214 adults over 1…

This study tests whether adding a substance called hyaluronan to the liquid used during embryo transfer can improve pregnancy rates in IVF. Researchers will compare live birth rates between women whose embryos are placed in a hyaluronan-rich medium versus a standard one. The tria…

This early-stage trial tests whether transplanting lab-grown retinal cells into the eye is safe for people with geographic atrophy from dry age-related macular degeneration. Twenty participants aged 55 and older will receive a transplant of their own reprogrammed cells into one e…

This study tests a traditional Chinese medicine called Jiedu Huayu oral prescription in 436 people who have had a brain bleed due to a condition called cerebral amyloid angiopathy. The goal is to see if the herbal treatment can lower the chance of having another stroke or blood v…

This early-stage study tests a new gene-editing medicine called PBGENE-DMD in 18 boys aged 2 to 7 with Duchenne muscular dystrophy. The treatment aims to correct the genetic mistake that causes the disease, potentially restoring muscle strength. Researchers are first checking if …

This study tests a refillable eye implant (Port Delivery System) that slowly releases the drug ranibizumab to treat wet age-related macular degeneration. The implant is refilled every 36 weeks, potentially reducing the need for frequent eye injections. About 250 participants who …

This Phase 2 study tests two new antibody-drug conjugates (R-DXd and I-DXd) against standard chemotherapy in 96 people with advanced nonsquamous non-small cell lung cancer that has worsened after prior immunotherapy and chemotherapy. These 'guided missiles' deliver a cancer-killi…

This study tests a new drug called AZD9750, alone or with another drug (saruparib), in 300 men with metastatic prostate cancer that has progressed despite standard treatments. The goal is to check safety, find the right dose, and see if it can lower PSA levels, a marker of cancer…

This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (…

This study tests a personalized version of deep brain stimulation (DBS) for people with severe obsessive-compulsive disorder (OCD) that hasn't improved with other treatments. Researchers will adjust the stimulation to target specific brain circuits linked to each patient's sympto…

This study tests a biodegradable eye implant called TO-O-1007 for geographic atrophy, a form of advanced dry age-related macular degeneration that causes blind spots. The implant slowly releases medication over 6 months to slow disease progression. About 31 adults aged 50 and old…

This Phase 3 study tests an experimental drug called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The drug is given as a spinal injection and aims to improve expressive communication and cognitive skills. Partic…

This Phase 3 trial tests whether the drug votoplam can slow the progression of Huntington's disease in people with early symptoms. About 770 adults aged 21 to 70 with a confirmed genetic diagnosis will receive either votoplam or a placebo. The main goal is to see if votoplam pres…

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…

This study investigates whether a wrist-worn sensor (KinesiaU) can accurately measure how long people with Parkinson's disease experience 'good on-time' — periods when medication works well and symptoms are controlled without troublesome movements. Researchers are testing IPX-203…

This Phase 3 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if a single dose can keep blood counts stable so patients can stop their regular enzyme replacement or substrate reduction therapy. The study will enroll 45 people who…

This study tests an experimental drug called NIO752 in 300 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. Participants are randomly assigned to receive either the drug or a placebo. After the main study, everyone can rec…

This study tests a cell therapy called OpRegen for people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes blind spots. About 60 participants will receive a one-time injection of retinal cells under the retina during eye surgery. The m…

This study tests a new daily pill, ETX-636, for people with advanced solid tumors (including breast cancer) that have a specific genetic change called PIK3CA. The trial has two parts: first, finding a safe dose of ETX-636 alone or with another drug (fulvestrant); second, checking…

This study looks at 100 people with wet age-related macular degeneration (AMD) who have been getting regular eye injections with older drugs. They will switch to a newer drug called faricimab. The goal is to see if faricimab can extend the time between injections, meaning fewer t…

This study tests an experimental drug called GTX-102 (apazunersen) in 60 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants range from 1 to 65 years old and are grouped by age and genetic type. The goal is to see if the drug …

This early-stage trial tests a brain-computer interface called BrainGate for people with paralysis from conditions like ALS or spinal cord injury. A small sensor is placed in the brain to interpret movement-related signals, allowing users to control a tablet computer just by thin…

This study tests a new type of deep brain stimulation (DBS) that automatically adjusts its settings based on real-time brain signals, unlike standard DBS which uses fixed stimulation. Sixty people with Parkinson's disease will have a special neurostimulator implanted and then exp…

This study tests an experimental drug called ALN-SOD in people with ALS caused by a mutation in the SOD1 gene. The drug is given via spinal injection to target the root cause of the disease. The main goal is to check safety, but researchers will also measure effects on biomarkers…

This study tests a single injection of CTx001 gene therapy into the eye for people with geographic atrophy, a form of advanced age-related macular degeneration that causes vision loss. The trial involves 75 participants and will monitor safety and whether the treatment can slow d…

This phase 3 trial tests whether a new drug called ABP 938 works as well as the approved treatment Eylea HD for wet age-related macular degeneration, a leading cause of vision loss. About 304 people aged 50 and older with untreated wet AMD will receive injections of either drug i…

This early-phase trial is testing a gene therapy called SGT-212 for Friedreich's ataxia, a rare genetic disease that damages the nervous system. Ten adults with the condition will receive the therapy through an injection into the brain and a vein. The main goal is to see if it is…

This study looks at the long-term safety of the drug brivaracetam in children and teenagers with childhood absence epilepsy or juvenile absence epilepsy. About 120 participants who have already taken the drug in earlier studies will continue treatment and be monitored for side ef…

This study tests a single dose of SGT-003, a gene therapy that delivers a working version of the dystrophin gene to muscle cells. About 60 boys with Duchenne muscular dystrophy, aged from infancy to under 18, will receive the treatment and be followed for 5 years. The goal is to …

This study tests an experimental drug called ultevursen for people with retinitis pigmentosa caused by a specific gene mutation (USH2A). The drug is injected into the eye and may help slow vision loss. The trial involves 81 participants, some of whom will receive a sham (fake) in…

This phase 3 trial tests two injectable drugs, pozelimab and cemdisiran, alone or together, to slow the growth of geographic atrophy—an advanced form of dry age-related macular degeneration that causes central vision loss. About 975 participants will receive either the drugs or a…

This study tests an experimental drug called ABS-1230 in children and young adults (ages 1 month to under 22 years) with a rare epilepsy caused by changes in the KCNT1 gene. The goal is to see if the drug is safe and can reduce the number of seizures compared to a placebo. Partic…

This study tests the long-term safety and effectiveness of a daily medication called nizubaglustat in people with two rare genetic diseases that affect the brain and body. About 21 participants who were in a previous study or who have been on a similar drug (miglustat) will take …

This study tests an oral drug called nizubaglustat in children and teens with rare genetic disorders (GM1, GM2 gangliosidosis) that damage the brain and nerves. The goal is to see if the drug can slow disease progression and improve movement and coordination. About 75 participant…

This early-stage study tests an experimental drug called pozelimab, given as an injection into the eye, for people with geographic atrophy (GA) — a condition where parts of the retina stop working, leading to vision loss. The main goal is to check the drug's safety and tolerabili…

This study tests a gene therapy called ZVS203e for people with retinitis pigmentosa caused by a specific RHO gene mutation. The therapy is injected into the eye and uses a harmless virus to deliver a gene-editing tool. The trial includes 18 adults and will check safety and whethe…

This Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease prog…

This Phase 2 trial tests an experimental drug called EYE103 (Restoret) in 160 people with wet age-related macular degeneration or macular edema from a branch retinal vein occlusion. Participants receive three eye injections of either a low or high dose of EYE103, spaced four week…

This study tests an experimental drug called REGN5093 in people with advanced non-small cell lung cancer that has a specific change in the MET gene. The first part finds a safe dose, and the second part checks if the drug can shrink tumors. About 231 participants will take part.

This Phase 2 trial tests an experimental drug called KIO-301, injected into the eye, for people with late-stage retinitis pigmentosa who have little or no light perception. The study will give up to three doses over several weeks to 36 participants to see if it is safe and can im…

This study tests an experimental oral drug, GSK5460025, alone or with other cancer drugs, in adults whose solid tumors have specific genetic changes (dMMR or MSI-H). The goal is to see if the drug can shrink tumors and to check its safety. About 47 people with advanced cancers th…

This early-phase trial tests a gene therapy called PUMCH-E111 for a rare inherited retinal disease caused by RLBP1 mutations. Six adults aged 18-55 will receive a single injection into the eye at a low or high dose. The main goal is to check safety, but researchers will also meas…

This early-stage trial tests whether transplanting 10 million special nerve cells made from stem cells into the brain is safe for people with moderate Parkinson's disease. Ten participants will receive the cells in a single surgery. The main goal is to check for side effects and …

This early-stage trial is testing an experimental drug called MK-0472, alone or combined with the immunotherapy Keytruda or another experimental drug MK-1084, in people with advanced or metastatic solid tumors. The main goals are to check safety, find the right dose, and see how …

This study tests a drug called salanersen in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug helps the body make more of a protein that is missing in SMA, which is needed for muscles to work. Researchers want to see if starting t…

This early-phase trial tests a new drug called ADCT-701 in adults with rare cancers like neuroendocrine tumors, adrenal cancer, and malignant peripheral nerve sheath tumors. The goal is to find the safest dose and see if it can shrink tumors or slow disease. Participants receive …

This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy who can still walk. The goal is to find the best dose, check safety, and see if it helps muscle strength. Participants take the drug or a placebo for 12 weeks, and all continue …

This phase 2 trial tests whether dapansutrile, a drug that targets inflammation, is safe and can reduce brain and body inflammation in people with early Parkinson's disease. 36 participants will take either the drug or a placebo daily for 6 months, with brain scans and spinal tap…

This early-phase trial is testing a new experimental drug called BNT3212, alone or with another drug (pumitamig), in 375 adults with advanced solid tumors who have no other treatment options. The main goals are to check safety, find the right dose, and get an early look at whethe…

This phase 3 study is testing whether adding an experimental pill called calderasib (MK-1084) to the immunotherapy drug pembrolizumab (Keytruda) works better than pembrolizumab alone for people with advanced non-small cell lung cancer that has a KRAS G12C mutation and high PD-L1 …

This study is testing the long-term safety of a daily drug called buntanetap in 500 people with Parkinson's disease over 36 months. It includes two groups: those who have taken buntanetap before and those who have a deep brain stimulation device. The main goal is to see if the dr…

This study tests an experimental drug called ADX-038 in 240 people with geographic atrophy, a form of advanced age-related macular degeneration that causes blind spots. The drug is given as a shot under the skin and is compared to a placebo to see if it can slow the loss of a key…

This study tests whether omaveloxolone, a drug already approved for people 16 and older with Friedreich's ataxia, can safely help children aged 2 to 15. About 255 participants will first receive either the drug or a placebo for a year, then all will receive the drug for two more …

This study tests a new medicine called ABBV-6628 for people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes vision loss. About 66 adults aged 50 and older will receive eye injections of the study drug or an approved treatment (SYFOVRE…

This phase 3 study tests a single injection of Ixo-vec gene therapy against standard aflibercept injections in 284 people with wet AMD. The goal is to see if Ixo-vec can maintain or improve vision while reducing the need for repeated eye injections. Participants must be at least …

This study tests a new drug called Lu AG13909 in 18 adults with Cushing's disease, a rare condition where the body produces too much cortisol. The goal is to see if the drug can safely lower cortisol levels to normal. Participants will receive the drug through an IV or injection,…

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to …

This study tests a new growth hormone injection (GB06) in 192 children aged 3 to 11 with growth hormone deficiency, a condition that causes slow growth. The treatment is given daily for 52 weeks to see if it helps children grow taller at a similar rate to an existing approved hor…

This early-stage study tests a single injection of a gene therapy called INS1201, given into the spinal fluid of young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to check if the treatment is safe and to see how it spreads in the body.…

This observational study will follow 300 people with wet age-related macular degeneration (nAMD) who receive aflibercept 8 mg injections as part of their routine care. Researchers want to see how well the drug improves vision and reduces retinal swelling over 12 months, and what …

This registry is collecting information from 1,500 people with Parkinson's disease who are receiving deep brain stimulation (DBS) with the Vercise system. The goal is to see how DBS affects quality of life in everyday practice, and to evaluate new programming tools. Participants …

This study tests a new way to give the drug rilvegostomig as a shot under the skin instead of through an IV. It is for adults with advanced solid tumors who have already tried standard treatments. The goal is to find a dose that works as well as the IV version and to check for si…

This study is testing a refillable eye implant that delivers the drug ranibizumab to treat wet age-related macular degeneration (AMD). The main goal is to see if the implant affects the cells on the inner surface of the cornea. About 188 people with wet AMD will receive the impla…

This study is collecting information from 500 people with essential tremor who are using a Boston Scientific deep brain stimulation (DBS) system, which is already approved for use. The goal is to see how the device affects quality of life in real-world settings, not just in contr…

This early-stage study tests an experimental drug called ALN-HTT02 in 66 adults with Huntington's disease. The drug is given as a single injection into the spine to see if it is safe and how it affects the body. The goal is to reduce levels of the harmful protein that causes the …

This early-stage trial tests a radioactive drug that seeks out and delivers radiation directly to cancer cells with a specific protein (somatostatin receptor). It is for adults with advanced lung, kidney, head and neck, digestive tract, or adrenal gland tumors that cannot be remo…

This early-stage study tests a new treatment called C.001 for people with retinal diseases that cause vision loss, such as geographic atrophy and Stargardt disease. The treatment is given as a single injection into the eye. The main goal is to check if it is safe, and researchers…

This study tests an experimental drug called LP352 to see if it can safely reduce seizures in children and adults with developmental and epileptic encephalopathies (DEE), a group of severe epilepsy syndromes. About 320 participants will receive either LP352 or a placebo, and rese…

This study tests whether the drug LP352 can safely reduce seizures in children and adults with Dravet syndrome, a severe form of epilepsy. About 160 participants will receive either LP352 or a placebo, and researchers will track changes in seizure frequency over several months. T…

This early-stage trial tests a drug called ALN-APP (mivelsiran) in 60 adults with mild cognitive impairment or mild dementia due to early-onset Alzheimer's disease. The drug is given via a spinal injection to see if it safely lowers certain proteins linked to Alzheimer's in the b…

This Phase 2 study tests an oral drug called RLY-2608 in adults and children with overgrowth conditions (like CLOVES or Klippel-Trenaunay syndrome) caused by PIK3CA gene mutations. The drug is designed to block the faulty protein driving abnormal tissue growth. The trial will enr…

This phase 2 trial is testing a drug called ONC206 in 90 people with advanced pheochromocytoma or paraganglioma, rare tumors of the adrenal glands. Participants must have tumors that cannot be removed by surgery and have not responded to or cannot have standard treatments. The st…

This study tests an experimental drug called ALN-APP in 200 adults with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain. The drug is given via spinal injection and aims to slow disease progression and reduce new brain bleeds. Participants will be …

This study follows about 215 adults with advanced Parkinson's disease who are switching from a pump medication to a new intestinal gel called LECIGON®. Researchers want to see if the gel reduces daily 'off' time—when symptoms return—over 12 months. Participants continue their usu…

This early-stage trial tests the safety and tolerability of a new drug called VX-670 in 52 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either VX-670 or a placebo, and researchers will monito…

This study tests a one-time injection of lab-grown nerve cells (NRTX-1001) into the brain for people with a specific type of epilepsy that doesn't respond to medication. The cells are designed to release a calming chemical called GABA to quiet overactive brain signals. Researcher…

This study tests whether adding risdiplam, a daily oral medicine, can help children under 2 with spinal muscular atrophy (SMA) who stopped getting better or started declining after receiving gene therapy. The study will enroll 28 children and measure changes in motor skills over …

This study tests whether a second focused ultrasound treatment on the opposite side of the brain can further reduce tremors in people with essential tremor who already had one treatment. About 100 participants will be randomly assigned to get the second procedure or continue stan…

This pilot study compares two surgical techniques for closing idiopathic macular holes: using a Tenon capsule graft versus the standard internal limiting membrane (ILM) flap. Twenty participants will undergo vitrectomy and be assigned to one of the two groups. The study will meas…

This study tests a small, refillable implant placed in the eye that slowly releases medication for wet age-related macular degeneration (nAMD). It aims to see if refilling the implant every 24 weeks works as well as getting a standard eye injection every 4 weeks. About 68 Chinese…

This study tests a new drug called STX-478 in people with advanced solid tumors that have a specific genetic change (PI3Kα mutation). The trial has two phases: first, it tests STX-478 alone to find safe doses; then, it combines STX-478 with other cancer drugs like fulvestrant or …

This early-stage trial tests an oral drug called DF-003 in 12 people with ROSAH syndrome, a rare genetic condition causing eye inflammation, enlarged spleen, and headaches. The main goal is to check safety and how the body processes the drug, while also watching for improvements …

This study tests whether D-serine, a dietary supplement, can slow the progression of Parkinson's disease. One hundred people with early-stage Parkinson's will receive either D-serine or a placebo for 58 weeks. Researchers will measure changes in symptoms and brain scans to see if…

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This study tests a new drug called SRP-1005 in 32 adults with Huntington's disease. It is the first time this drug is being tested in humans, so the main goal is to check safety and how the body processes the drug. Participants will receive either SRP-1005 or a placebo by injecti…

This study follows people who already received an eye injection of JNJ-81201887 or a sham in earlier studies for geographic atrophy, a form of advanced age-related macular degeneration that causes vision loss. The goal is to check long-term safety, including side effects and eye …

This study tests an experimental drug called prasinezumab in 900 people with early-stage Parkinson's disease who are already taking levodopa. The goal is to see if the drug can delay worsening of motor symptoms compared to a placebo. Participants receive the drug or placebo by IV…

This study tests a gene therapy called Ixo-vec in the second eye of people with wet age-related macular degeneration (AMD) in both eyes. Fifteen adults aged 50 and older will receive a single injection in the second eye and be followed for about 5 years. The goal is to see if the…

This early-stage study tests an experimental pill called CRN04894 in 18 adults with Cushing's syndrome caused by too much ACTH hormone. The drug works by blocking the receptor that ACTH acts on, aiming to lower cortisol levels and control the disease. The main goals are to check …

This study tests whether a targeted laser treatment, called photothermal therapy, can slow the progression of intermediate age-related macular degeneration (AMD). About 240 people aged 60–90 with intermediate AMD will receive either the laser or a sham procedure. The main goal is…

This study is testing deep brain stimulation (DBS) surgery for people with Parkinson's disease, essential tremor, or dystonia that doesn't improve enough with medication. DBS uses a device like a pacemaker to send electrical pulses to specific brain areas that control movement. R…

This study tests a treatment called bemdaneprocel, which involves transplanting dopamine-producing nerve cells into the brains of people with Parkinson's disease. The goal is to reduce daily 'OFF' time—when symptoms return—and increase 'ON' time without uncontrolled movements. Ab…

This study is checking the long-term safety of a small implant placed in the eye that slowly releases medicine to treat wet age-related macular degeneration, a condition that can cause rapid vision loss. About 1,000 people who have already used the implant in earlier studies will…

This early-stage study tests a new drug called MTX325 in healthy volunteers and people with mild to moderate Parkinson's disease. The main goals are to check safety, how the body processes the drug, and whether it reaches the brain. If successful, MTX325 might one day slow the pr…

This study evaluates PathFinder 1.0, a software that analyzes OCT images of the retina to detect macular abnormalities such as epiretinal membrane, dry age-related macular degeneration, and other retinal conditions. About 400 adults with and without macular issues will undergo st…

This study aims to find better ways to diagnose Parkinson's disease in its earliest stages. Researchers will use wearable devices to track movement and analyze blood samples for specific markers from nerve cells. By combining these digital and biological clues, they hope to creat…

This study tests a new radioactive tracer called 18F-mFBG to see if it can detect nerve damage in the heart caused by Lewy body diseases like Parkinson's and Lewy body dementia. About 20 adults will get a PET scan after an injection of the tracer. The goal is to see if the scan c…

This early-stage study tests a new radioactive tracer called SST001 that lights up on PET scans. Researchers want to see if it is safe and can help tell the difference between multiple system atrophy (MSA) and Parkinson's disease. The study will include 30 healthy volunteers and …

This study enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

This early-phase study tests a new imaging drug called [18F]MK-0947 that works with PET scans to show where a protein called α-synuclein builds up in the brain—a hallmark of Parkinson's disease. Researchers will check safety and how well the tracer works in 22 adults (some with P…

This study is testing a home-based eye-tracking system called AVIGA to see if it can detect when wet age-related macular degeneration (AMD) comes back. Sixty adults aged 50-99 who are being treated for wet AMD will use the AVIGA device at home, and its accuracy will be compared t…

This study tests whether a special PET scan (FET PET/CT) can better locate small pituitary tumors that cause Cushing disease. About 50 adults with confirmed Cushing disease who are scheduled for pituitary surgery will receive the scan before their operation. The goal is to see if…

This study compares a new diagnostic test using mannitol infusion to the standard hypertonic saline test for diagnosing the cause of polyuria-polydipsia syndrome (excessive urination and thirst). Researchers will enroll 144 adults to see if the mannitol test is as accurate and mo…

This early-phase study tests a new radioactive tracer called [¹⁸F]MODAG-009 to see if it can safely highlight abnormal protein clumps in the brains of people with Parkinson's disease or multiple system atrophy (MSA). About 13 participants, including healthy volunteers, will recei…

This study looks at whether taking a small piece of ligament during routine carpal tunnel surgery can help diagnose transthyretin amyloidosis, a serious condition that often goes unnoticed until it affects the heart. Researchers will compare this new biopsy method to the standard…

This study is testing a new blood test that looks for fetal cells in the mother's blood to detect genetic conditions like Down syndrome. The test will be compared to standard diagnostic methods such as amniocentesis or newborn testing. The study involves 1,000 pregnant individual…

This study looks at tissue removed during back surgery to find early signs of a heart condition called ATTR cardiac amyloidosis. Researchers will test spine samples from 1,663 older adults for abnormal protein deposits. If found, participants will get further heart tests to confi…

This study tests a laser treatment called OSC/SS to prevent retinal detachment in people with Stickler Syndrome, a genetic condition that raises the risk of vision loss. About 500 children and adults will receive the procedure in one or both eyes and be followed for 5 years. Rese…

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

This study tests an investigational drug called OHB-607 to see if it can prevent bronchopulmonary dysplasia (BPD), a serious chronic lung disease, in extremely premature infants. About 338 babies will be randomly assigned to receive either the drug or standard care. The goal is t…

This trial tests whether a community-based screening program using advanced eye imaging (OCT, fundus photography, and pressure checks) can prevent vision loss from age-related macular degeneration, diabetic retinopathy, and glaucoma better than simple vision tests alone. About 60…

This study tests a smartphone app designed to help teenagers aged 14-18 who self-harm and are not currently in therapy. The app teaches coping skills and connects them with support through a friendly chatbot. The goal is to see if the app is easy to use and helps reduce self-inju…

This study from Yale University will test whether 20 minutes of moderate cycling can reduce obsessive thoughts, compulsive urges, and improve thinking flexibility in adults with OCD. Fifty participants will be randomly assigned to either moderate or low-intensity cycling. Researc…

This study tests whether adding music-like sounds (sonification) to standard walking rehabilitation can improve gait, balance, and quality of life. 120 adults with Parkinson's disease, stroke, or multiple sclerosis will be randomly assigned to standard rehab or rehab with sonific…

This study tests a group therapy program called the Unified Protocol for adults with emotional disorders such as anxiety, depression, and related conditions. The program teaches skills like emotional awareness, flexible thinking, and facing fears. Researchers want to see if it im…

This study tests whether a four-session mindfulness class (Primary Care Brief Mindfulness Training) can help veterans reduce symptoms of anxiety, depression, and PTSD compared to a problem-solving class. The classes are designed for primary care settings and focus on meditation, …

This study is testing whether a specially designed amino acid supplement can improve nutrition, reduce oxidative stress, and boost physical and mental health in people with Parkinson's disease. Thirty adults aged 60-80 with Parkinson's will take either the supplement or a placebo…

This pilot study tests whether a gentle, non-invasive electrical current applied to the brain can improve how people with tunnel vision (from retinitis pigmentosa, rod-cone dystrophy, or advanced glaucoma) navigate obstacles. Twenty adults will receive both real and sham stimulat…

This study tests whether a mobile app with short weekly exercises can help first-time parents manage emotions and reduce depression during pregnancy. About 572 expectant mothers and fathers in Singapore will use the app or receive standard care. Participants will also wear a Fitb…

This study tests whether a non-invasive brain stimulation technique called HD-tDCS can improve visual and thinking abilities in people with posterior cortical atrophy (PCA), a rare condition often linked to Alzheimer's. Fifty participants will receive either real or sham stimulat…

This pilot study tests whether a single session of breathing air with slightly higher carbon dioxide and lower oxygen can improve speech, cough, and movement in people with Parkinson's disease. Participants are randomly assigned to one of two breathing patterns, each lasting abou…

This study tests an investigational drug called gemibotulinumtoxinA (a type of botulinum toxin) injected into arm muscles to reduce tremor in adults with essential tremor. About 94 participants will receive either the drug or a placebo injection. Researchers will measure changes …

This study explores whether a mobile app called FlowMD can help adults with mild anxiety. Participants use the app for 1-5 minutes of guided breathwork each day for four weeks. Surveys measure changes in anxiety levels and how easy the app is to use. The goal is to see if this ap…

This study tests whether wearing a headset that delivers a flickering 60Hz white light for 30 minutes a day can help ease depression symptoms. Adults with major depressive disorder will use the device at home for three weeks. Researchers will measure how well people stick with th…

This study tests whether osteopathic manipulative treatment (OMT) — a hands-on therapy using gentle techniques on the head, neck, and ribs — can improve sleep quality in people with Parkinson's disease. Participants are split into two groups: one receives OMT, the other a light t…

This study tests a new online therapy platform called Teleo, designed specifically for children with anxiety or mood disorders. Researchers want to see if kids are more engaged during therapy sessions on Teleo compared to standard video calls. About 156 children will take part, a…

This study tests whether a personalized form of transcranial magnetic stimulation (TMS) can improve motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy. Fifty participants will receive 10 days of targeted TMS…

This study tests whether a non-invasive brain stimulation technique called TMS can help with apathy—a common lack of motivation—in people with Parkinson's disease. Sixty participants will receive TMS to two different brain areas on separate visits and perform a task where they ch…

This study tests whether a videoconference with a psychologist before anterior cruciate ligament (ACL) knee surgery can reduce pain afterward. It includes 126 anxious patients scheduled for first-time ACL reconstruction. The main goal is to compare pain levels the night after sur…

This study compares standard carpal tunnel release surgery to an extended version that also releases a ligament in the forearm. Many people still have symptoms after standard surgery, possibly because the nerve is also compressed higher up. The trial will enroll 110 adults with c…

This study tests whether psilocybin, the active ingredient in magic mushrooms, combined with talk therapy can safely help cancer survivors with depression or anxiety. Twenty adults who have finished cancer treatment and have no brain involvement will receive a single dose of psil…

This study tests whether shining low-level laser light on muscles can reduce fatigue and improve quality of life in 40 adults with chronic fatigue syndrome. Participants will receive either real laser therapy or a sham treatment three times a week for eight weeks. Researchers wil…

This study tests whether walking with a metronome at different speeds can improve how people with Parkinson disease walk. 45 participants will do 12 training sessions on a treadmill and overground, using slow and fast beats to encourage larger and faster steps. Researchers will m…

This pilot study tests whether a peer support program can help young adults (ages 18-27) recently discharged from a psychiatric hospital. Participants get one-on-one and group meetings with trained Peer Support Specialists and recovery organizations, while a comparison group rece…

This study from Stanford University tests whether two breathing techniques—cyclic sighing and box breathing—can lower anxiety, compared to hypnosis or listening to an audiobook about stress. Eighty adults will practice daily for 4 weeks and have their brain activity and heart rat…

This study tests whether a testosterone drug (Depo-Testosterone) can reduce fatigue and improve sexual function, mood, and body composition in young men (ages 18-54) who have survived cancer and now have low testosterone. Participants must be in remission for at least one year an…

This pilot study tests a self-management program based on cognitive behavioral therapy to help people who survived acute respiratory failure and now struggle with anxiety. Sixty ICU survivors will either receive the program or usual care. The goal is to see if the program is prac…

This study looks at whether a new pump-delivered medication (Foslevodopa/Foscarbidopa) can help improve sleep problems in people with advanced Parkinson's disease. About 103 adults in Spain will take part, and researchers will track their sleep quality over 12 weeks using a simpl…

This study tests whether a combination of two existing drugs, nortriptyline and topiramate, can reduce vertigo and other symptoms in people with Meniere's disease. Forty adults with active Meniere's will take the drugs or a placebo for 8 weeks. The goal is to see if this approach…

This study tests whether using a genetic test to guide antidepressant dosing helps teens aged 12-17 with anxiety or depression. About 452 teens will be randomly assigned to either gene-guided dosing or standard dosing. The goal is to see if the genetic approach leads to more remi…

This study tests whether using a hand extension rubber during IV insertion can reduce pain, anxiety, and improve satisfaction. Researchers will enroll 114 adults who need an IV and randomly assign them to use the rubber or not. The goal is to see if this simple, low-cost distract…

This study tests whether a single ultrasound-guided injection of medicine around compressed leg nerves can relieve numbness, pain, and weakness in people with diabetes. 266 participants will be randomly assigned to get the real injection or a sham (fake) injection, and neither th…

This early study tests whether a gentle, non-invasive nerve stimulation device placed in the ear (taVNS) can improve how the body regulates heart rate and blood pressure in people with Parkinson's disease. Researchers will measure changes during simple physical tasks like deep br…

This study tests whether nerve gliding exercises, tendon gliding exercises, or a combination of both can reduce pain and improve hand function in people with mild to moderate carpal tunnel syndrome. Participants will be randomly assigned to one of three groups and followed for ch…

This study tests an internet-based cognitive behavioural therapy (ICBT) program designed specifically for Canadian public safety personnel (like police, firefighters, and paramedics) who have symptoms of depression, anxiety, or posttraumatic stress. Participants choose their leve…

This pilot trial tests whether repeated sessions of pulsed low-intensity focused ultrasound can safely reduce seizures in people with drug-resistant temporal lobe epilepsy. Ten adults who still have seizures despite medication will receive the ultrasound treatment. Researchers wi…

This study will test whether doing two physical tasks at the same time or combining a physical task with a thinking task can help people with Parkinson's disease move better, keep their balance, and do daily activities more easily. About 39 participants will do 45-minute exercise…

This study tests whether a personalized exercise program can improve balance and physical function in adults with rare neuromuscular disorders like Charcot-Marie-Tooth disease, facioscapulohumeral muscular dystrophy, and myotonic dystrophy type 1. Participants will receive a 12-d…

This study tests whether a simple four-square breathing exercise done before breastfeeding can lower anxiety and improve well-being in mothers whose babies are in the neonatal intensive care unit (NICU). About 70 mothers will be split into two groups: one does the breathing exerc…

This study tests whether a special laser (MLS class IV) can reduce chronic pain and fatigue in people with hypermobile Ehlers-Danlos syndrome (hEDS), a condition that causes loose joints and widespread pain. Twenty-five adults will receive 10 laser sessions over 5 weeks, with pai…

This study tests whether using virtual reality (VR) goggles during a colposcopy can lower women's anxiety, stress, and pain. About 60 women scheduled for the procedure will either receive standard care or wear a VR headset showing calming content. The goal is to see if this simpl…

This pilot study tests whether an accelerated course of repetitive transcranial magnetic stimulation (rTMS) can improve movement and thinking in people with mild to moderate Parkinson's disease. Forty participants aged 50 to 90 will receive six sessions of high-frequency magnetic…

This study tests whether adding poverty alleviation (like financial support) to group talk therapy helps low-income rural women in Bangladesh with depression more than therapy alone. Six hundred women aged 18-45 with depression and economic hardship will be randomly assigned to e…

This study tests whether a device that gently stimulates a nerve in the ear (taVNS) can help with Parkinson's symptoms like walking and balance problems. Sixteen adults with Parkinson's will use the device at home for one hour daily over eight weeks. Researchers will check if it'…

This study tests whether a personalized diet plan from a registered dietitian can improve constipation in people with Parkinson's disease. Fifty-four participants will follow the diet for 3 months, with regular check-ins. Researchers will measure bowel movements, gut bacteria, an…

This study tests whether 2-3 ten-minute visits from a trained therapy dog can reduce pain, anxiety, depression, and anger in adults hospitalized after a traumatic injury. Researchers will compare patients who get dog visits to those who only get a visit from a dog handler. The go…

This phase 2 trial tests whether VIM0423, a combination of two drugs, can reduce symptoms of isolated dystonia (a movement disorder causing muscle spasms). About 100 adults aged 18-65 with dystonia in at least two body areas will receive either VIM0423 or a placebo. Researchers w…

This study tests a 20-25 minute online program called Project EMPOWER-OCD for caregivers of people with OCD. The program teaches caregivers how to reduce accommodating behaviors that can accidentally make OCD worse. Researchers want to see if it lowers caregiver stress and improv…

This study tests a speech therapy program for bilingual Spanish-English or Spanish-Catalan speakers with primary progressive aphasia, a condition that slowly damages language abilities. Sixty participants will receive personalized script training via video sessions, while 30 othe…

This study tests a non-invasive brain stimulation technique called temporal interference stimulation (TIS) to see if it can improve cognitive function in people with mild cognitive impairment or early Alzheimer's disease. Sixty participants will receive either real TIS or a sham …

This study tests a new way to deliver deep brain stimulation (DBS) for essential tremor, called coordinated reset DBS. Instead of constant high-frequency pulses, it uses lower-current patterns that may work better and have fewer downsides. About 23 adults with essential tremor wh…

This pilot study tests a mindfulness-based music breathing program for older adults (65+) with mild to moderate anxiety or depression. The goal is to see if it boosts resilience and eases symptoms like loneliness and worry. Forty participants will be randomly assigned to the prog…

This study looks at the best way to fix a detached retina, a serious eye problem that can cause vision loss. It compares doing one surgery (vitrectomy) alone versus doing it together with cataract surgery at the same time. The goal is to see which approach gives better vision, fe…

This study tests a 6-session program called BE-FIT that helps cardiac rehabilitation patients reduce anxiety about exercise. The program uses gradual exposure to feared sensations, prevents safety behaviors, and uses activity monitors for feedback. Researchers will enroll 146 adu…

This study tests whether a 3-week cane training program can improve walking speed and confidence in people with Parkinson's disease. 26 participants will either receive cane training or do stretching exercises. The goal is to see if using a cane properly helps with gait and reduc…

This study looks at whether having a therapy dog present during a dental visit can lower stress in children. About 225 kids aged 4 to 8 will be randomly assigned to have a dental exam with or without a dog. Researchers will measure stress by tracking heart rate and testing saliva…

This study tests if pairing a gentle, non-invasive brain stimulation (tDCS) with personalized physical therapy can help people with cerebellar damage (from stroke, tumor, or degeneration) improve movement and daily function. About 30 adults aged 18-75 will participate. The goal i…

This study is testing a new type of talk therapy called Process-Based Therapy (PBT) for people with anxiety. Unlike standard treatments, PBT adapts techniques from different therapies to target each person's specific struggles. Ten adults with moderate-to-severe anxiety will try …

This study tests a web-based program called TEMPO designed to help men with prostate cancer and their caregivers cope with anxiety and improve physical activity. About 376 patient-caregiver pairs will be randomly assigned to use TEMPO or simply monitor their anxiety for 12 weeks.…

This early study tests a virtual reality rehabilitation device for children with spinal muscular atrophy (SMA), a genetic condition that causes progressive muscle weakness. The device uses games, muscle sensors, and a grip-strength ball to make home exercises more engaging. Resea…

This study tests whether hemp-derived CBD (with or without THC) can help older adults (60+) who use cannabis for pain, anxiety, depression, or sleep problems. Over 8 weeks, 385 participants will take either CBD+THC, CBD alone, or a placebo. The goal is to see if CBD can reduce sy…

This study tests whether a type of talk therapy called Exposure and Response Prevention (ERP) can improve daily functioning and quality of life in veterans with obsessive-compulsive disorder (OCD). About half of the 160 participants also have post-traumatic stress disorder (PTSD)…

This study tests whether real-time fMRI neurofeedback can help teens aged 10-16 with Tourette Syndrome reduce their tics. Participants learn to control activity in a brain region linked to tics by watching their own brain activity on a screen. The trial compares this to a control…

This study tests whether a program of supervised eye exercises can improve physical function, balance, and reduce fear of falling in people with Parkinson's disease. 34 participants will do progressive eye movements and gaze stabilization exercises in a clinic setting. The goal i…

This early-stage study tests a single dose of SER-252 in 40 people with Parkinson's disease who experience motor fluctuations (times when symptoms worsen). The main goal is to check safety and how the drug moves through the body. Participants receive either the drug or a placebo,…

This early study tests whether a spinal cord stimulation device called ARC-IM can safely improve walking and balance in people with Parkinson's disease. Six participants will receive the therapy, and researchers will monitor side effects and measure changes in gait speed and endu…

This study tests a new, non-invasive brain stimulation method called transcranial temporal interference stimulation (tTIS) for people with Parkinson's disease. The treatment targets a deep brain area involved in movement control. 32 participants will receive either real or sham s…

This study tests a handheld device that gives gentle directional cues (haptic feedback) to help people with Parkinson's disease perform arm movements more accurately. Twenty participants will use either the haptic device or a similar non-haptic version during home sessions over 8…

This study tests if reading a personal narrative book, in addition to standard care, can help women of childbearing age manage anxiety, depression, and negative thinking before a hysterectomy. About 156 women will either receive standard information or also read a book. The goal …

This study tests a group therapy program called TAG TEAM for trans and gender diverse teens aged 12-17. The program helps them understand and cope with stress from discrimination and rejection. Researchers will compare the program to a waitlist to see if it reduces anxiety, depre…

This study investigates whether breathing a 50:50 mix of nitrous oxide (laughing gas) and oxygen can reduce pain and anxiety during insertion of a central venous catheter, a common but uncomfortable procedure. Adults aged 18 to 65 who have never used nitrous oxide will be randoml…

This study uses a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) to examine how nerve pathways from the brain to the legs and trunk are affected in people with Parkinson's disease. Researchers will measure brain activity and link it to wal…

This study uses special PET scans to measure inflammation in the brains of people with Alzheimer's, Parkinson's, ALS, and other neurodegenerative diseases. Researchers will compare these scans to those from healthy volunteers to see if inflammation levels differ. The goal is to d…

This study explores whether a small accelerometer worn on the thigh can objectively measure how well treatments work for neurogenic orthostatic hypotension—a condition causing dizziness and fainting upon standing. Participants wear the device for one week on placebo and one week …

This study aims to learn more about methylmalonic acidemia (MMA) and related disorders by observing people with these conditions over time. Researchers will track complications, perform tests like blood draws and MRIs, and look for new genetic causes. The goal is to better unders…

This study at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study is testing whether a skin biopsy can detect abnormal proteins linked to Parkinson's disease in people with essential tremor, a common movement disorder. Researchers will follow 300 participants aged 50-85 for two years to see who develops Parkinson's. The goal is to fi…

This pilot study looks at how belzutifan, a drug already used for some tumors, changes the production of adrenaline-like hormones in people with pheochromocytoma and paraganglioma (PPGL). Over 5 days, researchers measure key substances in the hormone-making process to see if belz…

This study enrolls people with endocrine tumors (such as thyroid, parathyroid, adrenal, or pancreatic tumors) who are scheduled for surgery or biopsy. Researchers collect samples of the tumor, nearby healthy tissue, blood, and urine to analyze genetic and molecular changes. The g…

This study follows 500 people with geographic atrophy, an advanced form of age-related macular degeneration that causes permanent vision loss. Participants receive avacincaptad pegol (IZERVAY) as part of their routine care. Researchers will collect data on treatment patterns, sid…

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study will carefully examine the physical and mental health of 100 adults admitted to NHS mental health wards. Researchers will review medical notes, do physical exams, and use questionnaires to measure conditions like bipolar disorder, schizophrenia, depression, and persona…

This study follows adults who have recently received a cochlear implant to track how their ability to distinguish sounds and understand speech changes over time. Participants complete at-home sound tests frequently during the first three months, then return for lab tests up to on…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study looks at whether anxiety or depression makes asthma harder to control and aims to build a model that predicts when a serious asthma attack might happen. Researchers will follow 256 people with asthma for up to two years, tracking their symptoms, mental health, and lung…

This study investigates whether the quality of parent-adolescent communication at the start of psychiatric inpatient treatment predicts how much symptoms improve six months later. Researchers will track 60 adolescents hospitalized for depression, anxiety, or other mental disorder…

This study follows people with rare genetic disorders that cause harmful substances to build up in the body, leading to brain damage. Researchers will monitor up to 200 participants over time using exams, surveys, and lab tests. The goal is to better understand how these diseases…

This study will test how the heart drug empagliflozin is absorbed and processed in 10 children with Duchenne muscular dystrophy, ages 8 to 18. The goal is to find the best dose for protecting the heart in this rare disease. Participants will take the drug by mouth and have blood …

This study tracks the safety of Fycompa injection in people with epilepsy who have partial-onset seizures (age 4 and older) or primary generalized tonic-clonic seizures (age 12 and older). Researchers will record any side effects and measure drug levels in children aged 4 to 12. …

This study aims to understand what happens in the brain when someone thinks about or attempts suicide. Researchers will use brain scans, sleep monitoring, blood tests, and questionnaires to compare four groups: people with recent suicidal thoughts or attempts, those with past sui…

This study is creating a large collection of health information and biological samples (like blood, saliva, and eye tissue) from people with and without retinal diseases. Researchers aim to use this database to better understand conditions like age-related macular degeneration an…

This study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…

This study looks at how the brains of children with anxiety disorders work differently from healthy children. Researchers will use brain scans and thinking tasks to track changes over a year, before and after standard treatments like therapy or medication. The goal is to find pat…

This study screens males with Duchenne muscular dystrophy (ages 0 to under 25) for antibodies against AAV8, a virus used in some gene therapies. The goal is to find out how many have these antibodies and to identify who might be eligible for future gene therapy trials. No treatme…

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine up to 400 adults, perform skin exams, and take small tissue samples (biopsies) from tumors. The goal is to identify the cells and genetic …

This study aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

This study enrolls 600 adults with Huntington disease to examine specific genetic markers (SNPs) linked to the disease. Participants provide a blood sample and answer questions about their health and medications. The goal is to better understand the genetics of Huntington disease…

This study looks at children under 15 with neurofibromatosis type 2 (NF2), a rare genetic condition that causes nerve tumors. Researchers want to see if skin and mouth lesions can help diagnose NF2 earlier than current methods, which rely on hearing, nerve, and eye symptoms. By e…

This study is testing a new leg exoskeleton designed by the NIH to help children and young adults with muscle weakness from conditions like cerebral palsy or spinal cord injury. Researchers want to see how well the device works in different real-world settings, like walking on a …

This study creates a registry of medical records, scans, and surveys from adults with neurofibromatosis type 1 (NF1) who also have a glioma brain tumor. Researchers will track diagnosis, treatments, and quality of life over time to better understand the disease. The goal is to im…

This study aims to better understand disorders that cause hearing to fluctuate, such as Meniere's disease. Researchers will use advanced MRI scans and blood tests to look for biomarkers linked to hearing changes. The goal is to identify different types of these disorders and pave…

This study examines whether a person's personality traits before they got sick are linked to the thinking and behavior problems seen in certain brain disorders. Researchers will ask caregivers about the patient's personality from the 10 years before the illness started. The goal …

This international study aims to collect genetic and health information from 1,500 people with rare inherited retinal diseases like retinitis pigmentosa. It has two parts: a registry to gather genetic and vision data, and a natural history study that follows participants over tim…

This study looks at gum disease in people with rare kidney disorders like Alport syndrome, Fabry disease, and tuberous sclerosis, as well as lupus. Researchers will compare 100 participants to those with chronic kidney disease and healthy controls. They aim to understand how comm…

This study is looking for early signs of heart amyloidosis in Black adults who carry a specific gene change (V122I TTR) that raises their risk. Researchers will use heart MRI scans and blood tests to detect protein buildup before symptoms appear. The goal is to find ways to diagn…

This study looks at how differences in the NOTCH2NLC gene affect the symptoms and course of neuronal intranuclear inclusion disease (NIID), a rare inherited brain disorder. Researchers will follow 12 members of one family, including those with NIID, gene carriers without symptoms…

This study follows 2,800 unpaid caregivers of people with chronic conditions like diabetes or inherited disorders. Researchers will survey and interview caregivers over up to 5 years to track changes in their health, stress, and social support. The goal is to understand how careg…

This study gathers information from 125 people with von Hippel-Lindau (vHL) disease, a rare genetic condition that causes tumors. Researchers will track disease status, past surgeries and treatments, and quality of life. The goal is to better understand how vHL progresses over ti…

This registry will follow 500 people with inappropriate sinus tachycardia (IST) or postural orthostatic tachycardia syndrome (POTS) who have had or will have a heart ablation procedure using AtriCure devices. The goal is to collect real-world data on how safe and effective the pr…

This study follows 75 people aged 6 months or older with congenital myasthenic syndromes (CMS), a group of inherited disorders that weaken communication between nerves and muscles. Over up to 7 visits in 5 years, researchers will use physical exams, heart and lung tests, MRIs, an…

This study aims to understand the link between genes and symptoms in people with inherited movement disorders. Researchers will study up to 2,500 participants, including patients and their family members, to identify new disease-causing genes and improve diagnosis. The goal is to…

This study follows 650 people aged 8 to 99 with androgen insensitivity syndrome (AIS) to better understand their long-term health. Researchers will track hormones, bone density, heart health, metabolism, and tumor risk through regular check-ups and scans. No treatments are being …

This study aims to understand why some people are more prone to fungal infections, especially from Candida yeast. Researchers will collect blood, saliva, urine, and tissue samples from people with immune disorders, their family members, and healthy volunteers. No treatment is giv…

This study will follow about 1,850 people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers will collect information on patients' health, treatments, and outcomes over time to better understand the disease and how current therapies work in every…

This observational study aims to understand why some people are more impulsive or compulsive than others. Researchers will use brain scans, genetic tests, and behavioral surveys in 1,100 participants aged 6 to 80, including those with ADHD, OCD, autism, and conduct disorder. The …

This study follows 90 children and adults with Stargardt disease or similar inherited eye conditions to see how their vision changes over 2 years. Participants will have regular eye exams and imaging every 6 months. No treatment is given—the goal is to better understand the natur…

This study collects information from 200 adults in Russia who have neurofibromatosis type 1 (NF1) with plexiform neurofibromas. Researchers will look at patients' age, symptoms, and medical history to better understand the disease. No new treatments are being tested; the goal is …

This observational study follows 150 people with MEHMO syndrome or related conditions to better understand how the disease progresses. Researchers will collect medical history, imaging, and lab samples to find biological markers that could help monitor the disease. No treatment i…

This study is for people diagnosed with ALS, MND, or PLS. Participants fill out an online survey once a month about their physical function, diet, supplements, medications, and mental outlook. The goal is to see which therapies and behaviors are linked to better outcomes. No new …

This study is observing 400 adults with OCD who are receiving exposure therapy. Researchers will use machine learning to analyze self-reports, behavior, and physiological data from therapy sessions. The goal is to identify which mechanisms lead to better outcomes, so therapy can …

This study is testing a new, painless device called electrical impedance myography (EIM) to measure muscle health. Researchers will use it on healthy volunteers and people with neuromuscular diseases to see how well it works compared to standard tests like ultrasound and nerve st…

This natural history study is collecting information from up to 4,000 people with movement disorders (like Parkinson's disease or tremor) and their family members. Participants undergo exams, blood tests, and imaging, but receive no new treatments. The goal is to gather data that…

This study aims to find how common the KIT D816V mutation is in people with suspected clonal mast cell disease. Researchers will collect blood samples from 450 participants and test them using two sensitive methods. The results may improve understanding and diagnosis of these rar…

This observational study collects data from UK preterm babies with retinopathy of prematurity (ROP) who are treated with the drug aflibercept (Eylea) using a paediatric dosing device. The main goal is to count how many babies receive this treatment and decide if there are enough …

This study follows 269 people with neurofibromatosis type 2 (NF2) for up to 10 years to learn how the disease progresses. Participants will have yearly exams, MRI scans, hearing tests, and blood draws. The goal is to understand tumor growth, hearing loss, and speech or swallowing…

This study tests whether having a general practitioner and pharmacist work together can help people aged 65 and older safely stop or reduce benzodiazepines (drugs for sleep or anxiety). These medications raise the risk of falls, confusion, and dementia in older adults. The trial …

This study is testing whether a special ultrafast ultrasound can better measure muscle changes in boys and men with Duchenne or Becker muscular dystrophy. Researchers will compare muscle stiffness, fat content, and blood flow in 60 participants (patients and healthy volunteers) o…

This study aims to understand how lymphangioleiomyomatosis (LAM) damages the lungs at the cellular and genetic level. Researchers will follow up to 2,000 participants, including women with LAM and healthy volunteers, using advanced imaging and lab tests. The goal is to find the p…

This study interviews 30 patients with wet age-related macular degeneration to understand their views on gene therapy. Researchers want to know if the benefits outweigh the risks of this irreversible treatment, and how patients make decisions about complex therapies. The goal is …

This study is building a large collection of blood, DNA, and tissue samples along with health records from up to 5,000 people with tuberous sclerosis complex (TSC) or a related condition called lymphangioleiomyomatosis. Participants can give blood or cheek swab samples and allow …

This large study is testing many different drug combinations, including the immunotherapy pembrolizumab, in people with advanced non-small cell lung cancer. The goal is to see which treatments work best based on specific tumor features like PD-L1 levels and histology. Over 1,000 …

This study aims to uncover the genetic causes of pituitary and hypothalamic tumors in children. Researchers will collect blood and tumor samples from up to 2,000 participants to analyze DNA and look for inherited patterns. The study also trains doctors and tests new MRI technique…

This study follows children and adults worldwide who have a rare genetic muscle disease called TNNT1 myopathy. Researchers aim to document how the disease progresses over time, including survival and motor milestones. The goal is to identify reliable measures that can be used in …

This study follows 200 children with growth hormone deficiency for three years. Researchers will measure body composition, including muscle and fat, in kids taking either daily or long-acting growth hormone. The goal is to understand why some children gain weight on long-acting f…

This study aims to learn more about Li-Fraumeni syndrome (LFS), a genetic condition that raises the risk of several cancers. Researchers will study up to 5,000 people with LFS or related cancer histories to find new genes, understand how the TP53 gene works, and identify factors …

This study looks at whether certain proteins in the blood can help doctors monitor severe traumatic brain injury and predict who might benefit from hyperbaric oxygen therapy. Researchers will analyze blood samples from 150 patients already enrolled in a larger trial testing diffe…

This study follows up to 10 pregnant or breastfeeding individuals with transthyretin amyloidosis who have taken eplontersen, along with their infants. Researchers will track pregnancy complications, birth defects, and infant health during the first year of life. The goal is to de…

This study will analyze health records from 2009 to 2024 to find out how many people in Germany have three serious eye conditions: wet age-related macular degeneration, diabetic macular edema, and retinal vein occlusion. Researchers will look at data from two large German health …

This study is looking at whether test anxiety during final exams is linked to bowel problems and constipation in university students. About 200 students will fill out questionnaires about their anxiety and digestive health. The goal is to better understand how mental stress might…

This study collects skin, hair, blood, urine, or saliva samples from people with certain eye diseases (like macular degeneration) and from healthy volunteers. Researchers will turn these samples into stem cells in the lab to study how the diseases develop and to test potential ne…

This study looks at whether giving exposure therapy for OCD at different times in a woman's menstrual cycle changes how well it works. Researchers will use brain scans to see if hormones affect the brain's fear network and OCD symptoms. The goal is to find the best time in the cy…

This study looks at how tiny changes in your RNA (a molecule that reads your DNA) might help predict whether depression treatments will work. Researchers will follow 120 adults with unipolar or bipolar depression to see if these RNA changes match up with improvements in mood. The…

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study tests a new, non-invasive MRI technique to detect early brain changes linked to Alzheimer's disease. Researchers will compare brain scans from 50 people with mild memory concerns to healthy volunteers. The goal is to find a simple, affordable way to identify those at r…

This study works to improve questionnaires that measure pain, daily activities, and physical function for people with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas. Participants aged 5 and older will give feedback on existing surveys through group discussions or…

This study investigates the specific cognitive strengths and weaknesses of children and adults with Sturge-Weber Syndrome (SWS), a rare condition affecting blood vessels in the brain, skin, and eyes. Participants complete a series of tests measuring memory, attention, language, a…

This study tests whether eye-tracking tasks can detect changes in attention and learning in people with Rett syndrome who are starting a new medication called trofinetide. Researchers will compare eye movements and heart rate before and after 4 weeks of treatment in 20 participan…

This study compares carbon dioxide emissions from orthopedic surgeries performed with different materials and setups. Researchers will track waste, energy use, and sterilization methods for procedures like carpal tunnel release or wound revision. The goal is to identify ways to r…

This study is following 1,500 adults aged 55 and older to learn how early age-related macular degeneration (AMD) turns into the advanced form that can cause vision loss. Researchers will use eye imaging, genetic tests, and other measurements to find clues that predict progression…

This study watches how vision changes over two years in 50 people aged 65-90 with geographic atrophy (a form of dry age-related macular degeneration) who are already taking the drug pegcetacoplan. Researchers will use a special vision test that maps light sensitivity near damaged…

This study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

This study looks at how different mutations in the MORC2 gene lead to different symptoms, from Charcot-Marie-Tooth disease to a more complex condition called DIFGAN. Researchers will collect skin and blood samples from 45 participants to measure epigenetic and genetic markers. Th…

This study is looking at whether people with a common inner-ear condition called benign paroxysmal positional vertigo (BPPV) have lower vitamin D levels than those without it. Researchers will compare vitamin D in 160 adults — half with BPPV and half without. The goal is to see i…

This study is a disease registry that will collect information from 200 people in Egypt who have neurofibromatosis type 1 (NF1), a genetic condition that causes tumors to grow on nerves. The goal is to understand how the disease progresses over time, what treatments patients rece…

This study aims to improve how doctors find and understand pheochromocytomas and paragangliomas, rare tumors that can cause dangerous high blood pressure. Researchers will use advanced imaging scans and genetic testing in up to 3,000 participants to learn more about these tumors.…

This study is gathering medical information and samples from up to 5,000 people with neurosurgical conditions like brain tumors, epilepsy, and Parkinson's disease. Participants receive standard care while their data is collected for future research. No new treatments are being te…

This study tracks eye pressure in 100 people receiving a larger-volume injection of Aflibercept (0.07mL) for macular diseases like age-related macular degeneration. Researchers measure pressure right after the injection and at several time points, then check for any nerve damage …

This study looks at the long-term health of people who were cured of Cushing disease before age 21. Researchers want to understand how past high cortisol levels affect the body and mind years later. Participants will complete surveys and have check-ups every 5 years. The goal is …

This study is for people with a rare genetic muscle condition called congenital myasthenic syndrome (CMS). It does not test any new drug or treatment. Instead, researchers will collect information from up to 100 participants over 4 visits to better understand how the disease affe…

This study follows 300 people with Parkinson's disease who still have movement symptoms despite taking standard medications. Researchers will collect data over up to 5 years through questionnaires, diaries, medical records, blood samples, and a digital device. The goal is to bett…

This study investigates how the gut and brain communicate in people with Irritable Bowel Syndrome (IBS) who also experience anxiety or depression. Researchers will compare 100 IBS patients with anxiety or depression to 100 healthy volunteers using brain scans, stool samples, and …

This study looks at three drugs (bevacizumab, ranibizumab, and aflibercept) used to treat age-related macular degeneration (AMD), a leading cause of blindness in older adults. Researchers will analyze costs and quality of life for 600 patients in France to see which treatment off…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will examine DNA from up to 3,500 people with and without lung disease to find genetic differences. The goal is to better understand what causes these cond…

This study gathers information from the medical records of 1,200 people with ALS and similar conditions. Researchers will use this data to better understand the disease and improve future studies. No experimental treatments are given—the goal is simply to learn from real-world pa…

This study looks at anxiety levels in 100 living liver donors before and after their surgery. Participants will fill out questionnaires to measure their worry and stress. The goal is to better understand how anxiety affects recovery and to improve care for future donors.

This study is creating a central database and tissue bank for rare diseases affecting the kidneys and liver, such as ARPKD and Joubert syndrome. Researchers will collect medical information, genetic samples, and tissues from 200 participants to help doctors and scientists better …

This observational study, led by the Michael J. Fox Foundation, aims to understand how Parkinson's disease progresses over time. It will follow 4,500 people, including those with Parkinson's, those at risk, and healthy volunteers, collecting clinical, imaging, and genetic data. T…

This study is testing whether a carbon dioxide (CO2) challenge can predict which patients with anxiety, OCD, PTSD, or panic disorder will not respond to exposure-based therapy. Researchers will enroll 600 adults and measure their reaction to breathing CO2-enriched air before star…

This study follows 214 people in China with multiple system atrophy, a rare and fast-worsening brain disease that affects movement and automatic body functions. Researchers will track symptoms over time to learn how the disease progresses and what factors influence it. The goal i…

This study is for people with rare kidney diseases like Minimal Change Disease, Focal Segmental Glomerulosclerosis, and Membranous Nephropathy. Researchers will collect health information and biological samples from 1,200 participants over many years. The goal is to better unders…

This study reviews the medical records of up to 5,000 people who have received deep brain stimulation (DBS) for Parkinson's disease, essential tremor, or dystonia. Researchers want to see how well DBS improves symptoms over time in real-world settings. No new treatments are given…

This study looks at people with cavovarus foot deformity (a complex foot shape) due to Charcot-Marie-Tooth disease. Researchers want to see if a simple physical exam can identify different foot types, which currently requires special software. If successful, this could help surge…

This study is following 700 adults with myotonic dystrophy type 1 (DM1) over two years. Researchers are measuring walking speed, lung function, and muscle tissue changes to find reliable markers of disease progression. The goal is to improve future clinical trials by better under…

This study aims to learn more about the brain activity behind risk-taking and reward-seeking behaviors in people with Parkinson's disease. Researchers will record brain signals during deep brain stimulation surgery while participants play a gambling task. The goal is to understan…

This study helps people with certain kidney diseases (like nephrotic syndrome and FSGS) find clinical trials that match their specific disease profile. Researchers analyze participants' molecular data to recommend targeted therapies in ongoing trials. The goal is to improve treat…

This study aims to learn more about the brain activity behind cognitive impairment in Parkinson's disease. Researchers will record brain signals from 75 people during deep brain stimulation surgery while they perform a gambling task. The goal is to understand how certain brain ar…

This study is for children up to 4 years old with congenital myotonic dystrophy type 1 (CDM). Researchers want to find better ways to measure how the disease affects motor skills and language, and to identify biological markers. The goal is to improve future clinical trials and c…

This study follows 800 adults with synucleinopathies—diseases like Parkinson's, Lewy body dementia, and multiple system atrophy—to learn how they develop and change over time. Researchers will track symptoms, biological markers, and early signs like dizziness on standing or dream…

This study at Yale investigates how people with OCD process fear and safety signals in the brain. Researchers will use mild electrical stimulation (tDCS) to see if it can improve fear extinction learning, which is key to exposure therapy. 180 adults with OCD will undergo brain sc…

This study looks at whether a special nursing care model helps people with long-term health conditions like diabetes, heart disease, or Parkinson's work better with their advanced practice nurse. About 420 adults will take part. The goal is to see if this approach improves how pa…

This study is testing whether a watch-like device worn on the ankle can measure how well toddlers with Duchenne muscular dystrophy (DMD) move during their daily activities. Researchers will ask 30 boys with DMD and 30 without (ages 1-3) to wear the device for three 28-day periods…

This study is collecting information from 200 people with Parkinson's disease, dystonia, essential tremor, or other movement disorders who are scheduled to receive radiofrequency (RF) ablation—a procedure that uses heat to target small areas in the brain. The goal is to see how p…

This study looks at brain activity in 100 adults with epilepsy who already have electrodes implanted for seizure monitoring. Researchers will record brain signals while participants answer questionnaires and perform memory and impulse-control tasks. The goal is to find links betw…

This study looks at how a specific gene change (LRRK2 mutation) affects brain chemicals in people with Parkinson's disease. Researchers will compare 15 participants with the mutation to others without it using brain scans, thinking tests, and movement tests. The goal is to better…

This study looks at how deep brain stimulation (DBS) affects sleep in people with Parkinson's disease. Twenty participants who already have a DBS implant will spend two nights in a sleep lab—one night with the device on and one night with it off. Researchers will measure sleep qu…

This study observes 75 adults aged 55 and older with geographic atrophy (a late stage of age-related macular degeneration) to see how the condition progresses over a short time. Researchers will measure changes in eye structure and function, and explore links to genetics and life…

This study is testing a device called the AlgometRx Nociometer to see if it can measure pain and other abnormal sensations in people with neurofibromatosis type 1 (NF1). The device uses a small electrode on a finger or toe to send painless signals and a camera to record changes i…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

This study tests whether a gentle brain stimulation technique called tDCS, done at home, can improve sleep quality in adults with moderate depression. Researchers will track sleep changes and see if better sleep leads to better mood. The study involves 35 participants and aims to…

This study looks at how people with ME/CFS (chronic fatigue syndrome) and people with heart conditions feel after a bike exercise test. Researchers want to see if the type and timing of symptoms after exercise are different between the two groups. 80 adults will take part, and th…

This study analyzes dried blood spots from 630 newborns to find protein and RNA markers that could predict which babies with congenital CMV will develop hearing loss or other problems. Researchers will compare samples from infected babies who had complications, those who did not,…

This study follows 65 people with Friedreich ataxia who also have a thickened heart muscle (cardiomyopathy). Researchers will measure changes in heart structure over time using imaging. The goal is to better understand how heart disease progresses in this condition, which could h…

This study follows 20 adults with multiple sclerosis who are already scheduled for a stem cell transplant. Researchers will measure levels of an immune-suppressing drug called ATLG and track how the immune system rebuilds itself afterward. The goal is to learn more about immune r…

This study is collecting information from 850 people with wet age-related macular degeneration or diabetic macular edema who are being treated with faricimab (VABYSMO) in routine clinical practice. Over two years, researchers will track vision changes, treatment schedules, and sa…

This study examines whether climate anxiety is linked to suicidal thoughts and behaviors in young people aged 16 to 24 who are receiving psychiatric care. Researchers will use questionnaires to measure climate anxiety, general anxiety, and suicide risk in 108 participants. The go…

This study is watching for side effects in 50 children in India who are prescribed somatrogon, a long-acting growth hormone injection for growth hormone deficiency. The goal is to see how safe the drug is during routine care over 3 years. Participants do not have to visit the cli…

This study gathers medical information from people with MNGIE, a rare genetic disease that affects the nerves and digestive system. Researchers want to learn how the disease progresses and find better ways to measure it. Up to 50 patients worldwide can join, and no new treatments…

This study aims to learn why myotonic dystrophy type 1 affects children differently than adults, and why symptoms vary even within the same family. Researchers will observe 100 children (ages 0-17) through video calls and simple at-home activities, and analyze their genes from a …

This early-stage study is testing a new oral drug called SK-09 in 72 healthy adults to see if it is safe and how the body processes it. The study is split into two parts: one where participants get a single dose, and another where they get multiple doses. No one knows who gets th…

This study uses a special camera called an Adaptive Optics Scanning Laser Ophthalmoscope (AOSLO) to take very detailed pictures of the retina in people with inherited retinal diseases like retinitis pigmentosa. Researchers will measure how the light-sensitive cells in the eye cha…

This study tests whether real-time brain scans (fMRI) can help young adults with anxiety learn to regulate their emotions better. Participants will practice reappraising negative thoughts while seeing their own brain activity. The goal is to understand which brain areas are invol…

This study looks at brain activity in a person with a rare genetic condition called PRRT2 mutation, which causes sudden, uncontrollable movements. The participant will undergo brain scans (fMRI and EEG) during and after these episodes, and also when they voluntarily mimic the mov…

This study is for people aged 3 and older with neurofibromatosis type 1 (NF1), a genetic condition that often causes noncancerous nerve tumors. About half of people with NF1 get these tumors, and sometimes they turn cancerous. Researchers want to test a new method to predict whic…

This study is creating a large database of eye measurements from 3,000 people with inflammatory eye diseases like diabetic retinopathy and age-related macular degeneration. Researchers will use advanced imaging to collect detailed information from both the front and back of the e…

This study looks at how deep brain stimulation (DBS) changes motivation and decision-making in people with Parkinson's disease. Researchers will have 70 participants play a tablet game that measures risky vs. safe choices while their brain activity is recorded. The goal is to und…

This study follows 25 people aged 10 and older with Stargardt-like macular dystrophy (STDG3), a rare inherited eye disease that causes vision loss. Over three years, researchers will measure changes in the retina using eye exams, imaging, and vision tests. The goal is to understa…

This study is testing a new radioactive tracer called [18F]MODAG-009 in 6 healthy adults. The goal is to see how the tracer moves through the body and how much radiation exposure it causes. Participants get one injection and then a full-body PET scan. This is an early-phase study…