Hereditary neurological disease

This trial tests a gene therapy called SENS-501 for children aged 6 to 31 months who are severely or profoundly deaf due to mutations in the otoferlin gene. The therapy is injected directly into one inner ear to deliver a working copy of the gene. The goal is to restore natural h…

This study tests whether adding an experimental drug (RO7204239) to an existing SMA medicine (risdiplam) can help people with spinal muscular atrophy build stronger muscles and move better. The trial includes about 259 children and young adults, ages 2 to 25, who can walk. Resear…

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 study tests a new gene therapy for people with chronic granulomatous disease (CGD), a genetic disorder that weakens the immune system and makes them prone to serious infections. The treatment takes the patient's own blood stem cells, adds a missing gene in the lab, and puts …

This study follows 403 adults and children with spinal muscular atrophy who are already taking risdiplam (Evrysdi) as prescribed by their doctor. Researchers will track side effects and how well the drug works over about two years. The goal is to see if risdiplam remains safe and…

This study tests a vaccine called AADvac1, which targets tau tangles in the brain, a hallmark of Alzheimer's. It will be given alone or with another drug (donanemab) to 450 adults aged 50-80 with very early Alzheimer's. The goal is to see if this approach can reduce tau buildup a…

This phase 3 trial tests the long-term safety of GTX-102 (apazunersen) in 255 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants have already completed an earlier GTX-102 study and will continue receiving the drug. The main g…

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 UK trial tests whether cladribine tablets, taken for just 8-10 days a year over two years, can slow the loss of hand function in people with advanced multiple sclerosis (MS). The study includes 204 participants with significant disability (EDSS 6.5-8.5). Half receive cladrib…

This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under age 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and can help produce a key muscle protein. The children will be followed for about 5 years.

This study tests a drug called BBP-418 (Ribitol) in 81 people aged 12 to 60 with limb girdle muscular dystrophy type 2I (LGMD2I), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo for 36 months to see if it slows the di…

This study tests a device called a motor neuroprosthesis, an implantable brain computer interface designed to help people with severe paralysis control digital devices like computers or tablets. The device aims to bypass damaged motor pathways and transmit brain signals directly …

This study tests satralizumab, a drug that calms inflammation, in 30 children aged 8 to 17 with Duchenne muscular dystrophy. The goal is to see if it improves bone density and muscle function. Participants receive injections for several months while continuing standard steroid th…

This phase 2 trial tests whether prasinezumab, given as an IV infusion every 4 weeks, can slow motor decline in people with early Parkinson's disease. The study includes 586 participants who are already on stable Parkinson's medication. Researchers are measuring how long it takes…

This phase 3 trial tests EYP-1901, a new eye injection that may last longer than current treatments for wet age-related macular degeneration (AMD). About 400 participants will receive either EYP-1901 or the standard drug aflibercept. The goal is to see if EYP-1901 can maintain or…

This phase 3 trial tests EYP-1901, a new eye injection that may last longer than current treatments for wet age-related macular degeneration (AMD). About 400 people with wet AMD will receive either EYP-1901 or standard aflibercept injections over 2 years. The main goal is to see …

Researchers are testing whether radotinib, a drug used for leukemia, can help people with Parkinson's disease. The trial will give the drug or a placebo to about 43 adults aged 40 to 80 who were diagnosed within the past three years. The study measures safety, how the drug moves …

This study tests omaveloxolone, a drug already approved for adults with Friedreich's ataxia, in children aged 2 to 15. Researchers want to see how the drug moves through children's bodies and whether it is safe. The study involves two parts: a single dose followed by daily dosing…

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 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 trial tests whether low intensity focused ultrasound (LIFU), a non-invasive technique that uses sound waves to stimulate deep brain regions, can safely ease symptoms of obsessive-compulsive disorder (OCD). Researchers will target the ventral striatum, an area linked to OCD, …

This early-phase study tests a new drug called MK-2870, either alone or with the immunotherapy pembrolizumab, in Japanese patients with advanced solid tumors or a type of lung cancer. The main goal is to check the safety and side effects of these treatments. About 30 people will …

This trial tests an adaptive deep brain stimulation system for people with severe obsessive-compulsive disorder (OCD) that has not improved with standard treatments. The device, implanted in the brain, aims to detect brain signals linked to OCD distress and adjust its stimulation…

This trial tests three deep brain stimulation (DBS) settings in people with Parkinson's disease who already have a Boston Scientific Vercise system implanted. The settings are pure anodic, pure cathodic, and a mix of both. Researchers want to see which setting offers the best sym…

This study follows people with Parkinson's disease who previously received AAV-GAD, a gene therapy infused into a brain region that helps control movement. The goal is to check how safe the treatment remains over many years, watching for any drug-related side effects. Researchers…

This study tests whether deep brain stimulation (DBS) can improve thinking and memory in people with early Alzheimer's disease. Two participants will have electrodes implanted in a brain area that produces a key chemical for memory. The goal is to see if daily, one-hour stimulati…

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 whether a new drug called zanidatamab, combined with chemotherapy and sometimes another drug (tislelizumab), works better than the current standard treatment for advanced HER2-positive stomach and esophageal cancers. About 920 adults whose cancer has spread or ca…

This study tests a drug called selumetinib in children and young adults (ages 3-18) with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink or slow the growth of these tumors. Participants take the dru…

This phase 2 trial is testing whether a combination of two drugs, talazoparib and temozolomide, can shrink tumors in people with rare cancers that have spread. The study includes 14 participants with advanced rare solid tumors, such as pheochromocytoma or paraganglioma, who have …

This trial tests the long-term safety and effectiveness of apitegromab, an investigational antibody that blocks myostatin, a protein that limits muscle growth. People with type 2 or type 3 spinal muscular atrophy who completed earlier apitegromab studies can join. Researchers wil…

This study is testing whether implanting a small piece of a patient's own nerve tissue into the brain during deep brain stimulation (DBS) surgery is safe and doable for people with Parkinson's disease. The nerve tissue contains cells that produce growth factors, which might help …

This early-stage trial tests whether implanting a small piece of a patient's own nerve tissue into the brain during deep brain stimulation (DBS) surgery is safe and feasible. Ten people with Parkinson's disease will receive the implant, and researchers will monitor them for a yea…

This Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …

This study tests an experimental implant (AR-14034) that slowly releases medication inside the eye to treat wet age-related macular degeneration (AMD). The goal is to see if it is safe and can work as well as or better than standard anti-VEGF injections, which require frequent do…

This early study tests the safety of TheraSphere PCa, tiny radioactive glass beads injected into the prostate to treat localized prostate cancer. The trial involves 36 men with favorable intermediate-risk cancer and aims to find the maximum safe radiation dose. Researchers will m…

This study compares two surgical approaches for treating idiopathic normal pressure hydrocephalus (iNPH) in people aged 60 and older. One group will receive standard ventriculoperitoneal shunt surgery under general anesthesia, while the other will receive a lumbar-peritoneal shun…

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 phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…

This early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows a…

This study tests a one-time gene therapy called TSHA-102 in girls aged 2 to 4 with Rett syndrome, a severe genetic disorder. The therapy is given as a single injection into the spine. The main goal is to check if it is safe and tolerable, and to see early signs of whether it help…

This early-stage trial is testing a new drug called VOY-101 for people with advanced dry age-related macular degeneration (AMD) and geographic atrophy, a condition that causes blind spots. The drug is injected into the eye and aims to slow damage to the retina and preserve vision…

This study tests a new drug called zifibancimig for people with wet age-related macular degeneration (AMD), a leading cause of vision loss. The drug is given either as an eye injection or through a small implant placed in the eye that releases the drug over time. The trial involv…

This study tests a granule (sprinkle) form of the drug selumetinib in children aged 1 to under 7 years who have neurofibromatosis type 1 (NF1) with painful, inoperable tumors. The goal is to find the right dose, check safety, and see if it shrinks tumors. About 36 children will t…

This trial is testing an investigational drug called DYNE-251 in boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The goal is to see if the drug can safely help their muscles produce dystrophin, a protein that is missing in DMD. Participants recei…

This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any…

This study tests the long-term safety and effectiveness of XEN1101 (azetukalner) in 880 adults with focal or tonic-clonic seizures. Participants who completed earlier studies will take XEN1101 capsules for up to 6 years. Researchers will track side effects and changes in monthly …

This phase 3 trial is testing the safety and tolerability of fenfluramine (Fintepla) in 25 infants aged 1 to 2 years with Dravet syndrome, a severe form of epilepsy. The drug is given as an oral solution twice daily alongside other seizure medications. Researchers will monitor he…

This early-stage trial is testing whether a single same-day treatment using a patient's own bone marrow cells is safe and tolerable for people with Parkinson's disease or related conditions. The procedure involves drawing bone marrow from the hip, giving one portion through an IV…

This Phase 3 trial tests a drug called GTX-102 (apazunersen) in 129 children with Angelman syndrome, a genetic condition causing severe developmental delays. The study compares the drug to a sham procedure to see if it improves cognitive function and other symptoms over about 11 …

This trial tests whether a non-invasive technique called temporally-interfering electric field stimulation (TIEFS) can reduce abnormal brain activity linked to epilepsy. Researchers will measure seizure-related signals in patients with medial temporal lobe epilepsy who are alread…

This study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial …

This early-phase study tested a new gene editing treatment called HG302 in 4 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy uses CRISPR technology to fix the genetic error and help muscles produce dystrophin, a protein essential for musc…

This study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 714 adults aged 50-89 who currently need regular eye injections will receive either the gene therapy or standard treatment. The goal is to…

This trial tests whether a culturally adapted program of case manager support can improve survival, reduce hospital readmissions, and boost quality of life for adults recovering from accidental burn injuries in Pakistan. Participants receive up to eight sessions with a case manag…

This study tests whether faricimab, an already-approved eye injection, can be given every 24 weeks (about twice a year) to people newly diagnosed with wet age-related macular degeneration (AMD). The trial involves 274 participants and will measure how well vision is maintained ov…

This study tests a gene therapy called OCU400 for people with retinitis pigmentosa, an inherited eye disease that causes vision loss. The treatment is injected under the retina to help light-sensitive cells work better. The trial enrolled 140 people aged 3 and older and measures …

This study tests a gene therapy called OCU410 for geographic atrophy, a severe form of dry age-related macular degeneration that causes vision loss. About 60 adults aged 50 and older will receive a single injection of OCU410 into the eye. Researchers will monitor safety and check…

This study looks at the long-term safety and effects of a drug called EDG-5506 (sevasemten) in people with Becker muscular dystrophy, a muscle-weakening disease. About 260 adults and teens who were in earlier EDG-5506 studies will take the drug and be monitored for side effects a…

This study tests a new gene therapy called OCU410ST for people with Stargardt disease, an inherited eye condition that causes vision loss. Early results show the treatment slowed damage to the retina by nearly half and improved or stabilized eyesight in all treated patients. The …

This phase 2 trial tests an experimental drug called AOC 1044 in people with Duchenne muscular dystrophy whose genetic mutation makes them candidates for exon 44 skipping therapy. The drug is given by intravenous infusion. Researchers are evaluating long-term safety and whether i…

This Phase 3 study tests whether the drug leriglitazone can help adult men with cerebral adrenoleukodystrophy (cALD), a rare and serious brain disease. The main goal is to see if the drug delays death or the need for permanent breathing support compared to a placebo. About 40 men…

This study tracks the safety and effectiveness of Spinraza (nusinersen) in people with spinal muscular atrophy (SMA) who receive the drug as part of routine care in Korea. Researchers will monitor for side effects and measure changes in motor skills over time. The goal is to see …

This study tests lecanemab, an antibody that targets amyloid plaques in the brain, in 40 people who carry a gene mutation that causes early-onset Alzheimer's. Participants previously received another anti-amyloid drug and will now receive lecanemab to see if it can fully clear am…

This study tests whether two drugs, E2814 and lecanemab, can slow the progression of Alzheimer's in people who carry a gene mutation that causes the disease. About 197 participants will receive either the drugs or a placebo over 2 to 4 years. Researchers will measure changes in t…

This study tests whether experimental drugs can slow down memory and thinking problems in people who have a gene mutation that causes Alzheimer's disease. About 490 adults aged 18-80 with this genetic risk are taking either a study drug or a placebo. The goal is to see if the dru…

This trial tests whether giving the immunotherapy drug camrelizumab before surgery can help completely remove T4 colon cancers that have a specific genetic feature (dMMR). These cancers often don't respond well to standard chemotherapy. The study will enroll 18 adults who will re…

This study investigates whether intensively lowering systolic blood pressure with FDA-approved medications can reduce the buildup of amyloid and tau proteins in the brain—hallmarks of Alzheimer's disease. The trial enrolls adults aged 60-85 with high blood pressure who are cognit…

This study tests an experimental drug, NPI-001 (AT-001), in people with hereditary cystatin C amyloid angiopathy (HCCAA), a rare genetic condition that causes repeated brain bleeding and stroke. The trial includes about 15 participants aged 12 and older who carry the specific L68…

This phase 3 trial tests whether a combination of enfortumab vedotin and pembrolizumab, given before and after bladder removal surgery, works better than standard chemotherapy for muscle-invasive bladder cancer. About 800 participants will be randomly assigned to one of the two t…

This study tests a single-dose gene therapy called LY3884963 in 35 people with frontotemporal dementia caused by GRN gene mutations. The therapy aims to raise levels of a missing protein (progranulin) in the brain. Participants receive the treatment via injection into the fluid a…

This study is for people with FSHD, a genetic disease that causes muscle weakness. It tests the long-term safety and how well the body tolerates a drug called AOC 1020, given through a vein. About 84 adults who completed a previous study will take part. The main goal is to check …

This study tests a new drug called rilvegostomig (AZD2936) in people with advanced or metastatic non-small cell lung cancer. The drug is designed to boost the immune system's ability to fight cancer by blocking two targets (TIGIT and PD-1). The trial will check for safety, side e…

This phase 2 trial is testing whether adding an experimental mRNA vaccine (V940) to standard immunotherapy (pembrolizumab) can prevent kidney cancer from coming back after surgery. About 272 people with high-risk kidney cancer will receive either the vaccine plus immunotherapy or…

This study tests an experimental drug called prasinezumab in 316 people with early Parkinson's disease. The drug is an antibody designed to target and remove clumps of a protein linked to Parkinson's. Participants receive either the drug or a placebo for 52 weeks, with an option …

This study tests whether high-intensity treadmill exercise can slow the worsening of motor symptoms in people with early-stage Parkinson's disease. 370 participants who have not yet started Parkinson's medication will be assigned to either moderate or high-intensity treadmill ses…

This phase 2 trial tests an oral drug called venglustat, given alongside the standard enzyme therapy Cerezyme, in adults with Gaucher disease type 3 (a rare genetic disorder affecting the body and brain). The study has four parts: first, it checks spinal fluid biomarkers to disti…

This study tests a drug called mirdametinib in 114 adults and children with a genetic condition called NF1 that causes nerve tumors (plexiform neurofibromas) that cannot be removed by surgery and cause serious problems. The drug works by blocking a protein that helps tumors grow.…

This study follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective…

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 study tests the drug risdiplam (Evrysdi) in infants up to 6 weeks old who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The goal is to see if early treatment can help them reach motor milestones like sitting without support. The trial involve…

This trial tests a long-acting injection of leuprolide acetate in children with central precocious puberty, a condition where puberty starts too early. The goal is to suppress puberty-related hormones, slowing or stopping early development. The study includes children aged 4 to 9…

This Phase 3 trial tests whether people with wet age-related macular degeneration can use a home optical coherence tomography (OCT) device to guide when they need eye injections, instead of following a fixed schedule. 600 participants will either receive faricimab injections on a…

This early-stage trial tests an oral drug called verdiperstat in 64 people with semantic variant primary progressive aphasia (svPPA), a rare dementia that affects language. The study is double-blind and placebo-controlled, meaning some participants get the drug and some get a pla…

This pilot study tests whether injections of platelet-rich plasma (PRP) made from a patient's own blood can help preserve vision in people with retinitis pigmentosa or a related condition called EMAP. Thirty participants receive three injections over four months, and researchers …

This study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…

This phase 2 trial tests whether allopregnanolone, a natural hormone, can safely help repair the brain in people with early Alzheimer's disease. About 100 volunteers aged 55 to 80 will receive weekly infusions of the drug or a placebo for several months. Researchers will measure …

This pilot study tests whether adalimumab, an anti-inflammatory drug, can help preserve vision in people with retinitis pigmentosa or a related condition called EMAP. Thirty participants will receive three injections into the eye over four months. The main goal is to see if the t…

This study tests a gene therapy called laruparetigene zovaparvovec (AGTC-501) in 10 males aged 12-50 with X-linked retinitis pigmentosa, a genetic condition that leads to vision loss. The therapy is injected into both eyes to deliver a working copy of the RPGR gene. The main goal…

This study tests a new device called the eShunt System for treating normal pressure hydrocephalus, a condition that causes trouble walking, thinking, and bladder control in older adults. The device drains extra fluid from the brain through a small tube. Researchers will compare i…

This Phase 2 trial tests whether N-Acetylcysteine (NAC), an antioxidant, can help people with ME/CFS (chronic fatigue syndrome). Researchers will give 95 participants different doses of NAC or a placebo for four weeks, then measure brain glutathione levels and markers of oxidativ…

This Phase 2 trial tests a drug called RO7204239 in 51 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. The drug is an antibody that blocks myostatin, a protein that normally limits muscle growth, to help pres…

This phase 3 trial tests whether an oral antioxidant called N-acetylcysteine (NAC) can slow the loss of vision in people with retinitis pigmentosa, an inherited eye disease that causes gradual blindness. About 485 adults aged 18 to 65 are taking NAC or a placebo for several years…

This phase 2 trial tests whether lamivudine, an antiviral drug, can improve symptoms in people with Rett syndrome, a rare genetic disorder that affects movement, communication, and behavior. Ten participants will take the drug by mouth for a set period, and researchers will measu…

This early-stage study tests a gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The therapy delivers a gene that helps calm overactive brain cells. Researchers will check if it safely reduces seizure frequency and improves thinking and dai…

This study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…

This study tests a device called the PRIMA System, which includes an implant placed under the retina and a special visual processor worn by the patient. The goal is to help people with advanced dry age-related macular degeneration (geographic atrophy) regain some central vision. …

This study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gen…

This study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy (SMA). About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to…

This study tests an experimental drug called VP-001 for people with a rare genetic eye disease that causes vision loss. The drug is injected into the eye and aims to slow or stop further damage. Researchers will check safety and measure any changes in eyesight over about two year…

This Phase 3 trial tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. It includes 148 males who can and cannot walk. Participants receive a one-time IV infusion of the therapy or a placebo, and are followed for about 128 w…

This study tests whether adding cabergoline to osilodrostat works better than osilodrostat alone for controlling Cushing's disease. About 50 adults with active Cushing's disease will receive either the combination or the single drug. The goal is to see if the combo can lower cort…

This Phase 3 trial tests a single injection of Ixo-vec gene therapy in 311 people with wet age-related macular degeneration (AMD). The goal is to see if it can maintain or improve vision as well as standard aflibercept injections, but with fewer follow-up treatments. Participants…

This study tests a higher dose of nusinersen (Spinraza) in 45 teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken risdiplam. Participants receive two 50 mg loading doses two weeks apart, then 28 mg every four months for about two years, …

This phase 3 trial tests whether a daily injection of elamipretide can slow vision loss in people with dry age-related macular degeneration (AMD). About 313 adults aged 55 and older with early dry AMD will receive either the drug or a placebo for 96 weeks. The main goal is to see…

This study tests an experimental drug called BIA 28-6156 in people with Parkinson's disease who have a specific change in the GBA1 gene. The goal is to see if the drug can delay the worsening of movement-related daily living skills over 78 weeks. About 237 participants will recei…

This early-stage trial tests whether implanting a person's own lab-grown dopamine cells into their brain is safe for Parkinson disease. Nine participants aged 40-75 with Parkinson for at least 4 years will receive the injection. The goal is to see if the cells can replace lost do…

This early-stage trial tests a gene therapy called PBFT02 for frontotemporal dementia (FTD) in people with specific genetic mutations. The therapy delivers a working copy of the GRN gene directly into the brain. The study involves 30 adults and primarily checks for safety and sid…

This study tests a gene therapy called SPVN06 for retinitis pigmentosa, an inherited eye disease that causes gradual vision loss. The treatment is given as a single injection under the retina. The trial includes 33 adults with advanced disease and will check safety and whether it…

This study tests a new medicine called BI 771716 for geographic atrophy, an advanced form of age-related macular degeneration that causes vision loss. About 202 adults aged 50 and older will receive either the new drug or an existing treatment (pegcetacoplan) as eye injections. T…

This study tests whether monthly eye injections of ANX007 can prevent significant vision loss in people with dry age-related macular degeneration (AMD) that has progressed to geographic atrophy. About 659 participants will receive either the drug or a sham injection. The main goa…

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to standard steroid treatment over time. Researchers will measure movement abilities, breathing, and safety, including liver problems. Participants must already…

This study tests an oral drug called SKY-0515 in 120 adults with Huntington's disease. The goal is to see if it can lower harmful proteins in the blood, slow brain changes, and improve symptoms. Participants must be 25 or older with a confirmed genetic diagnosis and a certain lev…

This trial tests a nurse-led health program for women aged 45 to 64 in Hong Kong. The program uses a 5-step approach (ask, advice, assess, assist, arrange) to help women manage conditions like high blood pressure, anxiety, and menopause symptoms. Researchers will compare the prog…

This study follows about 427 adults with advanced Parkinson's disease who are receiving ABBV-951, a continuous infusion under the skin. Researchers want to see how well it controls symptoms like tremors and stiffness over three years in everyday medical practice. Participants att…

This Phase 3 study tests two new drugs, tarcocimab tedromer and tabirafusp tedromer, against the standard treatment aflibercept for wet age-related macular degeneration (AMD). The trial involves 675 participants who have not been treated before. The goal is to see if the new drug…

This study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The treatment aims to help the eye produce its own medicine to stop leaky blood vessels, potentially replacing the need for repeated eye injecti…

This Phase 3 study tests whether fenfluramine (ZX008) can safely reduce seizures in 87 children and adults with CDKL5 deficiency disorder, a rare genetic condition causing severe epilepsy. Participants receive either the drug or a placebo, followed by an open-label extension wher…

This pilot study tests a device called Travagus One, which stimulates a nerve in the ear to reduce inflammation in boys with Duchenne muscular dystrophy (DMD). Twenty boys aged 5-17 will use the headset at home for 5 minutes twice a day for one week. Researchers will measure infl…

This study follows about 80 boys with Duchenne muscular dystrophy who have already taken vamorolone in earlier studies. Researchers want to see how safe the drug is over a longer time, especially its effect on bone health, like spine fractures. The boys continue taking vamorolone…

This study follows about 300 adults with advanced Parkinson's who are already using Lecigon, a gel given through a pump to control movement problems. Researchers will track how well it works and what side effects occur over up to two years in everyday medical care. The goal is to…

This study tests whether a pre-filled syringe is as safe as the traditional vial when giving the medicine bevacizumab for certain eye diseases. About 120 people with wet age-related macular degeneration, diabetic macular edema, or branch retinal vein occlusion will receive inject…

This study tests the drug alpelisib, already approved for some breast cancers, in people aged 2 months to 30 years with certain blood vessel growth disorders caused by PIK3CA or TIE2 gene changes. Participants take the drug for 2-3 years and attend up to 16 clinic visits to check…

This study offers fenfluramine to people with Dravet syndrome whose seizures have not improved with other medications. The goal is to provide access to the drug and monitor its safety. Participants must have a genetic diagnosis of Dravet syndrome and have tried all available seiz…

This study tests a phone-based care management program for adults with depression, anxiety, or risky drinking. Participants get regular check-ins and support from a mental health technician, while their primary care doctor receives treatment recommendations. The goal is to see if…

This pilot study tests whether deep brain stimulation (DBS) of specific brain pathways can safely reduce seizures in people with Lennox-Gastaut syndrome, a severe form of epilepsy. Five participants receive the electrical stimulation as an add-on to their usual treatment. The goa…

This program offered vatiquinone, an experimental liquid medication, to patients with inherited mitochondrial diseases like Leigh syndrome who had already completed a previous safety study. The goal was to continue treatment for those who might benefit, but enrollment is now clos…

This early-stage trial tests a daily injection of ASA-001 in two adults with a rare genetic muscle disease (ADSS1 deficient myopathy). The main goals are to see if the drug is safe and if it can slow the disease. Participants will take the drug for 8 months and have clinic visits…

This phase 2 trial is testing whether a combination of two drugs, dasatinib and quercetin, is safe and feasible for people with early-stage Alzheimer's or mild cognitive impairment. The drugs aim to clear aging 'senescent' cells that may contribute to brain damage. 48 participant…

This study tests whether high-dose nicotinamide (vitamin B3) is safe and can help people with dominant optic atrophy, a rare genetic disease that slowly damages the optic nerve and causes vision loss. Researchers will give 25 adults 3 grams of nicotinamide daily and monitor for s…

This phase 3 trial tests whether the drug tofersen can delay or prevent ALS in adults who carry a SOD1 gene mutation but have no symptoms yet. About 158 participants will receive either tofersen or a placebo, and researchers will track how many develop ALS within two years. The g…

This study tests the drug selumetinib in adults with neurofibromatosis type 1 (NF1) who have painful, inoperable nerve tumors called plexiform neurofibromas. About 145 participants will receive either selumetinib or a placebo to see if the drug can shrink the tumors and reduce pa…

This study follows 25 people aged 2 to 65 with cerebral palsy or brain damage from lack of oxygen who have already received a new gene therapy given as a nose spray. The therapy delivers 15 genes meant to repair the brain, reduce inflammation, and improve movement and thinking. R…

This study tests a new painless nerve growth factor eye drop (CHF6467) in 36 children and young adults with optic pathway glioma, a brain tumor that often causes vision loss. The goal is to see if the drops can protect or improve vision without the pain side effects of standard n…

This early-phase study is testing whether stem cell therapies and experimental drugs can help repair inner ear damage in people with hearing loss or tinnitus. Researchers will monitor 120 adults over time using hearing tests and symptom surveys. The goal is to see if these regene…

This study tests a drug called tinlarebant in 60 people with Stargardt disease, a genetic condition that causes vision loss. The goal is to see if the drug can slow damage to the retina and preserve eyesight. Participants take the drug or a placebo, and researchers measure change…

This study aimed to see if a special nutritional supplement (AMS2434) could help people with Parkinson's disease build stronger muscles and improve brain function. It was designed for adults aged 55 and older with mild to moderate Parkinson's. However, the study was withdrawn bef…

This study tests whether a personalized, data-driven hearing rehabilitation program can improve hearing, thinking, and quality of life in older adults with mild to moderate hearing loss. Two hundred first-time hearing aid users will be randomly assigned to either the new personal…

This study tests whether mindfulness-based therapy can help people with obsessive-compulsive disorder (OCD). Researchers will compare mindfulness to standard cognitive behavioural therapy (CBT) in 420 adults with OCD. The goal is to see if mindfulness reduces OCD symptoms and to …

This study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…

This early-stage trial tests a combination treatment for retinitis pigmentosa, a genetic disease that causes blindness. Ten adults receive a single injection of GS030 gene therapy into one eye, then use special glasses that send light signals to the treated retina. The main goal …

This study tests a drug called lanreotide in 10 people with advanced or metastatic paraganglioma or pheochromocytoma, rare tumors that release hormones. The goal is to see if lanreotide can slow tumor growth compared to before treatment. Participants receive injections every 4 we…

This study tests a gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The treatment is given as one or two injections into the eye and aims to produce proteins that block abnormal blood vessel growth, potentially replacing …

This Phase 3 trial tests whether higher doses of eteplirsen (Exondys 51) can safely improve muscle function in boys with Duchenne muscular dystrophy whose genetic mutation allows exon 51 skipping. About 160 ambulatory boys will receive one of three doses (30, 100, or 200 mg/kg) t…

This study looks at how well a drug called foslevodopa-foscarbidopa works for people with advanced Parkinson's disease in France. About 250 adults whose doctors have already prescribed this drug will be followed for up to 12 months. The goal is to see if the treatment improves th…

This phase 2 study tests pembrolizumab (Keytruda) in 40 men with metastatic castration-resistant prostate cancer that has specific genetic flaws (mismatch repair deficiency or CDK12 inactivation). The goal is to see if the drug can shrink tumors or slow the cancer. Researchers wi…

This study tests whether painting roofs with a special reflective coating can lower indoor temperatures and improve mental health. About 800 people in Ahmedabad, India, who live in homes with poor insulation will be randomly assigned to get a cool roof or not. Researchers will me…

This Phase 2 trial tests whether a sleep-deepening drug (lower-sodium oxybate) or a nerve-stimulating device (non-invasive vagus nerve stimulation), or both, can help clear amyloid-beta protein from the brain in people with cerebral amyloid angiopathy (CAA). Sixty participants wi…

This study tests whether lower doses of the drug bevacizumab (Avastin) can effectively treat a severe form of retinopathy of prematurity (ROP) in premature infants. The trial includes 77 babies with type 1 ROP in zone I, the most dangerous type. Each infant receives a single inje…

This Phase 2 trial tests a genetically modified herpes virus (RP1) alone or with the immunotherapy drug nivolumab in people with advanced solid tumors, including melanoma, non-small cell lung cancer, and certain skin cancers. The study aims to see if the combination is safe and c…

This study is testing a radioactive drug called Lutathera in teenagers aged 12 to 17 who have rare tumors of the digestive system or adrenal glands. The goal is to see if the drug is safe and how much radiation reaches organs. Eleven teens are taking part, and researchers are wat…

This early-phase study tests an experimental oral drug (GSK4418959) in adults with advanced solid tumors that have specific genetic changes (dMMR or MSI-H). The drug works by blocking a protein (WRN) that these tumors need to survive. The study aims to see if the drug, alone or w…

This study tests a new medicine called PRAX-562 in 77 children with rare genetic epilepsies (SCN2A or SCN8A). The goal is to see if it safely reduces motor seizures. The trial has two parts: a double-blind phase where some children get the drug and some get a placebo, followed by…

This Phase 3 trial tests whether a daily pill called tinlarebant can slow the growth of geographic atrophy, an advanced form of age-related macular degeneration that causes blind spots. About 429 adults with the condition will receive either the drug or a placebo for up to 2 year…

This early-stage study tests a one-time gene therapy injection into the spinal fluid of children aged 1-18 with CLN7 Batten disease, a rare and fatal genetic brain disorder. The main goal is to check safety, but researchers will also measure changes in movement, thinking, and vis…

This study tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…

This first-in-human trial tests a stem cell therapy called STEM-PD for Parkinson's disease. Eight participants with moderate Parkinson's will have dopamine-producing cells transplanted into a brain region affected by the disease. The main goal is to check safety over 12 months, w…

This study aimed to improve how deep brain stimulation (DBS) is targeted for people with severe obsessive-compulsive disorder (OCD). It planned to use brain scans to personalize the placement of electrodes. However, the trial was withdrawn before enrolling any participants, so no…

This study compares two drugs, aflibercept and brolucizumab, for wet age-related macular degeneration (AMD). Instead of the usual three monthly loading doses, it tests starting with longer intervals between injections right away. The goal is to see if this approach works just as …

This study compares two types of surgery for people with a detached retina. One group gets standard repair surgery, the other gets an extra step where a thin membrane is peeled away. Researchers want to see if this extra step lowers the chance of re-detachment and improves vision…

This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINR…

This study tests a vaccine called ACI-7104.056 in 150 people with early-stage Parkinson's disease. The goal is to see if it is safe and helps the body fight a protein linked to Parkinson's. Participants must already be on stable low-dose levodopa. This is not a cure, but may help…

This early-phase trial tests a single infusion of a gene therapy called FBX-101 in 9 people with Krabbe disease who have already received a stem cell transplant. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor function and saf…

This study tests an experimental drug called AVD-104, given as an eye injection, for people with geographic atrophy, a late stage of dry age-related macular degeneration that causes blind spots. The trial involves 300 participants and aims to see if the drug is safe and can slow …

This study compares two drugs, Avastin and Eylea, given as eye injections for wet age-related macular degeneration (AMD). The main goal is to see which drug requires fewer injections over two years while still controlling the disease. Researchers will also check vision, eye thick…

This study tests a single dose of AVASPA gene therapy given directly into the brain of children with Canavan disease, a rare and severe genetic disorder that damages white matter. The therapy aims to restore a missing enzyme to help the brain produce myelin and slow disease progr…

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children and teens aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle damage markers. Participants are rand…

This study tests the long-term safety and effectiveness of a cell therapy called deramiocel (CAP-1002) in people with Duchenne muscular dystrophy who completed the earlier HOPE-2 trial. Participants receive an infusion of deramiocel every 3 months for about 5 years, with the opti…

This study tests a custom-made drug called an antisense oligonucleotide in a single person with TUBB4A-related leukodystrophy, a rare genetic disorder that affects the brain's white matter. The drug is designed to target the specific genetic mutation causing the disease. Research…

This study tests a higher dose of the drug aflibercept (8 mg) given as an eye injection every 4 weeks to people with wet age-related macular degeneration (nAMD) or diabetic macular edema (DME). The goal is to see if this dosing schedule is safe and works well for those who have a…

This study explores whether altering gut bacteria with an antibiotic (rifaximin) can improve motor symptoms and medication response in people with Parkinson's disease. Researchers will first analyze how gut bacteria relate to symptoms, then give some participants rifaximin and ot…

This study tests whether two mechanical rotation chairs—the TRV and the newer Rotundum—are equally accurate at diagnosing benign paroxysmal positional vertigo (BPPV), a common cause of brief dizzy spells triggered by head movements. About 200 adults with typical BPPV symptoms wil…

This study tests an AI tool called AIDP that analyzes MRI scans to help diagnose Parkinson's disease and two similar conditions (MSA and PSP). Researchers at 21 sites will compare the AI's diagnosis to that of movement disorder specialists. The goal is to see if the tool can impr…

ScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…

This study compares a new, non-invasive PET-MRI scan to the standard invasive test (IPSS) for finding pituitary tumors in people with Cushing's disease. About 43 adults with confirmed Cushing's will get both tests to see which is more accurate. The goal is to make diagnosis safer…

This study evaluates RDX-Aneurysm, a computer-assisted detection software designed to help radiologists find saccular intracranial aneurysms (bulging blood vessels in the brain) on MRI scans. Researchers will analyze 550 past scans—half with confirmed aneurysms and half without—t…

This study is testing whether a simple saliva sample can help diagnose amyotrophic lateral sclerosis (ALS). Researchers will analyze RNA in the saliva of 653 people, including ALS patients and healthy volunteers. If the test works, it could offer a faster, less invasive way to di…

This study explores whether ultrasound elastography, a non-invasive imaging technique, can accurately diagnose carpal tunnel syndrome (CTS) without the need for painful needle-based nerve tests. Researchers will enroll 80 participants—some with CTS and some healthy volunteers—to …

This study is testing a way to find cardiac amyloidosis—a rare but serious heart condition—earlier in people aged 65 and older. Researchers in Turkey will screen 800 patients at family medicine clinics using simple blood tests and medical history. The goal is to see if this appro…

This study tests a device called AVIGA that tracks eye movements to detect vision problems in people with wet age-related macular degeneration (AMD). Researchers will compare the device's results with standard eye exams to see if it can reliably spot disease activity. The goal is…

This study aims to see if a simple, inexpensive saliva test can accurately diagnose Cushing's syndrome, a condition where the body makes too much cortisol. Researchers will compare two automated saliva test methods in 380 adults who are already being checked for the condition. Th…

This study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …

This study is testing whether artificial intelligence can analyze a person's speech to help diagnose mental health conditions like depression, anxiety, autism, and schizophrenia. Researchers will record 500 people speaking and compare the AI's predictions to standard clinical dia…

This trial tests whether 5-10 minutes of daily mindfulness and compassion training in school can prevent anxiety and depression in children aged 6-16. About 1,750 pupils will take part, with some classes doing the training for 10 weeks and others not. Researchers will track menta…

This trial tests whether a combination of lifestyle interventions—brain training, yoga, music therapy, and a special diet—can slow or reverse early signs of neurological disorders like dementia, stroke, epilepsy, and Parkinson's disease. Participants are healthy adults aged 50 an…

This study tests whether a health promotion program can prevent anxiety and depression in older Latino adults who are at risk. 240 participants will either join a program with group walks and social sessions led by a community health worker, or attend a healthy lifestyles educati…

This study aimed to see if a surgical procedure called scleral buckling could prevent retinal detachment in people with Stickler syndrome, a genetic condition that raises the risk of vision loss. The plan was to treat one eye in patients aged 5 to 35 who had already lost vision i…

This study tests whether a workshop led by fellow firefighters can help prevent PTSD, depression, anxiety, and alcohol problems in new firefighter recruits. About 480 trainees will take part in either an emotion regulation workshop or a standard education session during their aca…

This study tests if a drug called dalargin can prevent serious organ problems like kidney injury, lung failure, or heart damage after major abdominal surgery. About 200 adults having high-risk surgery will get either dalargin or a placebo for 3 days. The goal is to see if dalargi…

Researchers are testing an experimental oral tablet called APSLXR in people with Meniere's disease or vertigo of vestibular origin. The main goal is to check whether the drug is safe when taken once a day for 60 days. A small group will also have blood tests to see how the body p…

This trial tests whether adjusting deep brain stimulation (DBS) settings specifically for sleep can improve sleep quality in people with Parkinson's disease. Participants already use an adaptive DBS system that responds to brain signals. Researchers will compare three different s…

This study tests whether an online problem-solving therapy, matched to a person's specific symptoms using machine learning, can reduce depression and anxiety better than usual care. About 380 adults with moderate symptoms will take part. The goal is to create a precise, scalable …

This pilot study tests whether a home-based program called HOBSCOTCH, originally designed for epilepsy, can be adapted for people with Parkinson's disease. Ten participants will work one-on-one with a coach in nine virtual sessions to learn problem-solving strategies and memory a…

This early-phase study at Johns Hopkins University is testing whether psilocybin, the active compound in magic mushrooms, can safely help people with obsessive-compulsive disorder (OCD). Thirty-five adults with moderate to severe OCD who have already tried other treatments will r…

This study tests whether a 24-week collaborative care program can improve quality of life for adults with epilepsy who also have anxiety or depression. The program includes regular check-in calls and expert recommendations from a psychiatrist and neurologist. Researchers will com…

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 trial tests whether CranioSacral therapy, a gentle manual therapy applied to the feet, diaphragm, neck, and skull, can reduce tremors and improve movement precision in people with Parkinson's disease. Five participants will receive 90-minute sessions twice a month for three …

This study tests whether using advanced brain imaging to guide deep brain stimulation (DBS) can better control tremor in people with Parkinson's disease while avoiding side effects like involuntary movements. Twenty adults who already have DBS implants will have their stimulation…

This study is testing whether hyperbaric oxygen therapy (breathing pure oxygen in a pressurized chamber) can improve symptoms like fatigue and brain fog in people with post-COVID syndrome. About 40 adults with long COVID will receive the treatment. The main goal is to see if a la…

This study compares two low-intensity family treatments for children aged 7-13 with anxiety or obsessive-compulsive disorder (OCD): internet-delivered cognitive behavioral therapy (CBT) and a parent-led reading program. Both are delivered via telehealth and compared to a relaxati…

This study tests whether combining motor skill training with a non-invasive spinal stimulation can improve walking, balance, and strength while reducing muscle spasticity in people with incomplete spinal cord injury. Twenty-eight participants will do a series of standing exercise…

This study tests a communication coaching program for adolescent and young adult cancer survivors (ages 18-35) who have finished treatment at least a year ago. Participants complete online sessions and AI coaching to improve how they talk with peers. The goal is to see if the pro…

This study looked at whether a special exercise program focusing on muscle power (moving quickly) could help people with Parkinson's disease who have slow movements (bradykinesia). Twenty-two adults with Parkinson's were split into two groups: one did standard exercises, and the …

This trial tests whether a 20-minute session of mandala coloring while listening to slow instrumental music can reduce cancer fear and anxiety in women undergoing cancer screening. Women aged 30 to 65 attending a screening center will be randomly assigned to either the coloring a…

This study tests a topical patch called PEACE that uses gentle vibrations to help people feel less stressed or anxious. 150 adults aged 18 to 85 who have stress or anxiety will wear either the active patch or a sham (fake) patch for 14 days. Researchers will measure changes using…

This study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…

This trial tests whether spending time in nature can reduce anxiety in children aged 9-12 who have experienced adverse childhood events. Participants are assigned to either group nature outings, family outings, or a waitlist. The goal is to see if these nature-based activities im…

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 trial tests a counseling program called Mpata Yathu ('Our Space') for adolescent girls and young women in Zambia who have experienced gender-based violence and may be living with or at risk for HIV. The program uses problem-solving therapy delivered by trained lay counselors…

This study tests whether mild electrical stimulation of the ear, combined with physical and speech therapy, can improve symptoms like voice problems, swallowing, and balance in people with Parkinson's disease. 46 participants will receive either active or sham stimulation during …

This study looks at whether using the Calm meditation app for 10 minutes a day can help blood cancer patients who have trouble sleeping. Researchers will track sleep, mood, and inflammation in 276 participants over 20 weeks. The goal is to find a simple, drug-free way to improve …

This trial compares two forms of cognitive-behavioral therapy (CBT) for children and teens with anxiety or obsessive-compulsive disorder (OCD). One approach uses internet-delivered modules with therapist guidance; the other trains parents to lead exposure exercises with their chi…

This small early-phase study is testing whether psilocybin, the active ingredient in magic mushrooms, combined with supportive therapy can safely help people with obsessive-compulsive disorder (OCD) that hasn't responded to standard treatments. Ten adults with treatment-resistant…

This pilot study explores whether robot-assisted walking devices can be used in schools to help children who cannot walk independently. The devices attach to a walker and power leg movements, allowing children to stand and walk upright. The study aims to identify the best ways to…

This study compares three treatments for shoulder impingement and carpal tunnel syndrome: ultrasound-guided corticosteroid injections, standard injections guided by touch, and oral pain medication. Researchers want to see which approach provides the best long-term pain relief, im…

This study investigates whether a painless, low-intensity electrical current applied to the scalp can improve cognitive function — specifically the ability to stop an action when needed — in people with Parkinson's disease. The trial includes 50 Parkinson's patients and 40 health…

This study tests a new functional drink made with chicory inulin, B-vitamins, and vitamin D to see if it can improve constipation and overall well-being in people with Parkinson's disease. About 56 participants will drink either the active supplement or a placebo twice daily for …

This study tests whether alternating between high and low frequencies of deep brain stimulation (DBS) can improve walking and balance in people with Parkinson's disease. Twelve participants who already have DBS implants will try different stimulation patterns while their gait and…

This study tests an internet-based cognitive behavioral therapy (CBT) program designed to help autistic adolescents aged 11-17 manage anxiety. The program, called LUNA-Adolescent, is parent-led and delivered online, making it easier for families who cannot access in-person therap…

This study tests whether a unique music therapy, based on Traditional Chinese Medicine's five-element theory, can help men with depression and anxiety when added to their usual medication. One hundred male inpatients will receive either standard drugs alone, drugs plus regular mu…

This study tests a 7-session, peer-led self-management program for 234 cancer survivors who recently finished treatment with curative intent. The program aims to reduce anxiety and depression by teaching symptom management skills in a group setting. Participants are from central …

This study tests whether a new therapy called Emotional Resolution (EmRes) can help reduce anxiety in healthcare workers. About 150 hospital workers with mild to moderate anxiety will either receive EmRes or a mindfulness-based gratitude exercise. The goal is to see if EmRes lead…

This study tests a speech therapy method called Rapid Syllable Transition (ReST) in children aged 5 to 8 with childhood apraxia of speech, a condition that makes it hard to say sounds correctly. The goal is to see if the therapy is practical and helps improve speech clarity and m…

This study tests whether a pain-relief gel (pregabalin) delivered through the skin using ultrasound can reduce pain and improve hand function in people with carpal tunnel syndrome. The treatment is given four times a week for a month, and compared to standard ultrasound therapy w…

This study tests a therapy called RETELL that helps people with severe mental illness explore and reshape their life stories. The goal is to improve personal recovery, quality of life, and identity. Twenty adults with conditions like schizophrenia, bipolar disorder, or PTSD will …

This study looks at whether training parents to help with speech therapy at home improves outcomes for children with childhood apraxia of speech (CAS). Forty children aged 2 to 7 will receive standard speech therapy twice a week for 8 weeks. Some parents will get direct coaching …

This study tests whether a short, intensive course of cognitive behavioral therapy (CBT) over 2 weeks works better than the usual weekly sessions over 6 months for people with anxiety disorders. 120 adults with conditions like panic disorder, OCD, or PTSD will be randomly assigne…

This study was designed to test whether a reusable pouch called DoddleBags could help people with Huntington's disease eat more easily and safely. The pouch holds pureed food and can be used with an anti-choking cap or spoon attachment. The trial was withdrawn before any particip…

This study planned to see if using virtual reality (VR) before a brain stimulation treatment called TMS could help people with OCD feel more comfortable. It was designed for adults already approved for TMS. However, the study was withdrawn before any participants were enrolled, s…

This study tests a digital cognitive behavioral therapy program (SilverCloud) for Spanish-speaking adults with depression or anxiety. 426 participants will either use the program on their own or get extra support from a peer coach via phone or messaging. The goal is to see if add…

This study tests whether 80 sessions of a gentle brain stimulation technique called tDCS, given over 12 months, can improve attention, social skills, and language in 180 children aged 6 to 11 who have autism along with rare diseases, genetic problems, or PANDAS. The treatment is …

This study tests whether a physical activity program can help pregnant people with symptoms of depression or anxiety feel better. 88 participants will be given a step goal and wear an activity tracker. The program includes weekly reminders and motivational coaching every two week…

This study tests whether an internet-based self-help program called VIVIM can help university students with anxiety and depression. About 108 students will be randomly assigned to use VIVIM or receive usual support from their university counseling service. They will fill out ques…

This study tests a program that combines family group sessions with a virtual peer mentoring program for US-born teens (ages 14-17) whose parents resettled as refugees. The goal is to reduce symptoms of PTSD, depression, anxiety, and behavior problems caused by intergenerational …

This study tests whether a 12-week strength and functional exercise program, done at home via video calls with a physiotherapist, can improve walking, balance, and daily life in people with Hereditary Spastic Paraplegia (HSP). Twenty participants aged 16 to 70 will each experienc…

This study tests a 12-session group therapy program (G-BCBT) for active duty military personnel who have recent suicidal thoughts or a recent suicide attempt. The therapy teaches emotion regulation, cognitive flexibility, and relapse prevention skills. Researchers will measure ch…

This study tests whether a gentle electrical current applied to the back of the head (cerebellar tDCS) can improve both movement and mood in people with mild to moderate Parkinson's disease. Twenty-five participants will use a portable device at home for 8 days. Researchers will …

This study looks at whether adding a daily 30-minute muscle stimulation session can improve pain control and quality of life for people who already have a spinal cord stimulator (BurstDR) for chronic back pain. Sixty adults with failed back surgery syndrome or persistent spinal p…

This study tests two simple treatments for mild to moderate carpal tunnel syndrome: pain neuroscience education (learning how the brain processes pain) and kinesio taping (applying elastic tape to the wrist). Sixty adults aged 18 to 60 with symptoms for at least 3 months will rec…

This study compares a new numbing gel (chloroprocaine HCl 3%) to standard eye drops plus a shot for pain relief during eye injections. About 236 adults with conditions like diabetic macular edema or age-related macular degeneration will take part. The goal is to see if the gel wo…

This phase 3 study tests a device that gently stimulates the vagus nerve through the skin (tVNS) to see if it can safely reduce temper outbursts in people with Prader-Willi syndrome. About 102 participants aged 10 to 40 will use either continuous or intermittent stimulation. The …

This study looks at whether a ketogenic diet (high-fat, low-carb) is practical and safe for people with early-stage Alzheimer's disease over one year. The diet may provide an alternative energy source for the brain and reduce inflammation. Researchers will track diet adherence, b…

This study tests whether using a lower-pressure tourniquet during orthopedic surgeries can reduce pain and the need for opioid painkillers afterward. Standard tourniquets use high pressure, which can cause muscle weakness and pain. The experimental device sets pressure based on t…

This study tests whether an 8-week auditory-cognitive training program can improve speech recognition and cognitive function in older adults (60+) who have had a cochlear implant for 3 months to 3 years. Participants will do computer-based listening and thinking exercises at home…

This study tests whether a personalized lifestyle program focusing on stress, exercise, nutrition, sleep, and self-management can improve quality of life for people with Parkinson's disease. 256 participants will be randomly assigned to either work on their goals independently or…

This small, early-stage study tested a high-CBD, low-THC liquid spray for 8 weeks in 12 older adults with mild cognitive impairment or mild-to-moderate Alzheimer's who also had significant anxiety or agitation. The goal was to see if the treatment could reduce these behavioral sy…

This study tests whether a 4-week telerehabilitation program can improve balance and quality of life in 36 people with multiple sclerosis or Parkinson's disease. Participants do balance exercises at home while monitored by a hospital physiotherapist. The results are compared to s…

This study looks at two treatment plans for carpal tunnel syndrome, which causes hand numbness and pain. One plan starts with up to two steroid injections, then offers surgery if needed. The other plan goes straight to surgery. About 258 adults with mild to moderate symptoms will…

This study tests whether mindfulness-based cognitive therapy (MBCT) can reduce psychological distress in people with Parkinson's disease. 174 participants with mild-to-moderate stress symptoms will either receive MBCT or continue usual care. The main goal is to see if MBCT lowers…

This study tests whether a drug called crisugabalin can safely reduce pain in people with Parkinson's disease who have a type of pain called nociplastic pain. About 166 adults will take either the drug or a placebo twice a day for 12 weeks. Researchers will measure pain levels an…

This study tests two types of memory training in 120 people with Parkinson's disease and mild cognitive impairment. Participants complete 8 weekly sessions of either process or strategy training. The goal is to see if these exercises improve remembering to do tasks in daily life,…

This study tests an experimental drug called gemlapodect to see if it can safely reduce tics in people with Tourette syndrome. About 164 adults and teens with moderate to severe tics will take the drug or a placebo for 12 weeks. The main goal is to measure changes in tic severity…

This study looks at whether online community support and telemedicine can improve quality of life for people with Parkinson's disease, Lewy body dementia, and related conditions, as well as their caregivers. About 632 participants will receive palliative care training for their d…

This study tests a set of virtual reality exergames in the Metaverse to help people with neurological conditions like multiple sclerosis, Parkinson's disease, stroke, and traumatic brain injury improve their cognitive skills. 25 participants will try games like virtual cooking an…

This study tests whether a 5-week heart rate variability biofeedback program can lower stress and anxiety in 50 dentistry students moving from classroom to clinic. Participants in the training group practice slow breathing with real-time feedback, while a control group receives n…

This study tests whether personalized computer games can help people with Rett syndrome use their arms more purposefully and reduce repetitive hand movements. Fourteen participants will play games that reward them for separating their hands and reaching for targets. The training …

This study tests whether a mobile app called PTSD Coach, with or without brief clinician support, can reduce PTSD, anxiety, depression, and sleep problems in World Trade Center responders living in Florida. About 120 English- or Spanish-speaking responders will be randomly assign…

This study tests whether a type of brain stimulation called rTMS can reduce depression in people with autism. Twenty-four participants aged 13 to 26 will receive either real or sham (fake) stimulation to see if it helps. The goal is to find a new way to treat depression that hasn…

This study is testing a device called MemorEM, which is a cap that sends low-level electromagnetic waves to the head. The goal is to get feedback from 1,000 patients with neurological diseases like Alzheimer's or Parkinson's, and their caregivers, on how easy the device is to use…

This study tests a special therapy program delivered over the internet for people with mild cognitive impairment or dementia who also feel anxious. The program uses cognitive behavioral therapy (CBT) techniques to help manage worry and fear. Researchers will measure changes in an…

This study tests whether a dual-task training program—combining physical exercises like walking with mental tasks like counting—can improve both motor and cognitive function in 16 boys aged 6–12 with Duchenne muscular dystrophy. Participants will be randomly assigned to either st…

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study looks at two simple ways to help children feel less anxious and in less pain when getting a tooth pulled. One method uses a fun video to distract them, and the other uses gentle pressure on a specific point on the hand. The study will include 69 healthy children aged 5…

This study tests whether partnered dance aerobic exercise can reduce 'OFF-time' (when Parkinson's medication stops working well) and improve thinking skills. Researchers will enroll 82 people with Parkinson's who experience OFF-time. Participants will take dance classes twice a w…

This pilot study tests whether a Parkinson's-specific Tai Chi program can improve movement and non-movement symptoms like balance, fatigue, and mood. Thirty adults with mild to moderate Parkinson's will either take Tai Chi classes plus usual care or just usual care for 12 weeks. …

This study tests a new, team-based rehabilitation program for 125 working-age adults with persistent physical symptoms and chronic fatigue. The program includes doctors, therapists, and counselors who work together to help patients manage their symptoms and improve daily function…

This pilot study tests whether daily meditation can improve recovery for children and adults having surgery for kidney stones. Participants will be randomly assigned to either a meditation group or a control group. Those in the meditation group will practice a daily 20-minute gui…

This study tests whether a gentle electrical current applied to the scalp (tDCS) can reduce obsessive-compulsive disorder (OCD) symptoms in people who haven't gotten better with standard therapy. 43 adults with severe, long-lasting OCD will receive either real or fake stimulation…

This phase 3 trial tests whether inhaled salbutamol, a drug used for asthma, can help newborns with transient tachypnoea (rapid breathing after birth). About 608 babies born between 32 and 42 weeks will receive either salbutamol or a placebo. The goal is to see if the drug reduce…

This study tests whether a special light therapy device can improve quality of life and movement for people with Parkinson's disease. About 350 participants who are already on standard Parkinson's medication will use the light at home. The goal is to see if adding this light ther…

This study looks at whether two Akashic Records sessions can improve mental health in adults who have felt stressed, anxious, or down in the past year. Participants will attend private video sessions with a therapist and complete surveys about their mood and resilience. The goal …

This pilot study tests a short online program called 'Well-Beans for Caregivers' for adults caring for someone with tuberous sclerosis complex (TSC). The program consists of three weekly 2-hour sessions based on Acceptance and Commitment Therapy to help caregivers cope with diffi…

This study tests whether a gentle electrical brain stimulation device, used at home under remote supervision, can help reduce fatigue in people with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). Twenty participants will use the device for 20 minutes each weekday ov…

This study looks at whether taking vitamin D supplements for six months can improve depression symptoms. About 319 adults with depression are getting either a low or high dose of vitamin D daily, while continuing their usual treatment. Researchers will track their mood and health…

This study tests whether adding sodium bicarbonate (baking soda) to the local anesthetic used during carpal tunnel release surgery reduces the pain of the injection itself. 116 adults with carpal tunnel syndrome will be randomly assigned to receive either buffered or standard ane…

This study compares two common types of stitches used to close the wound after carpal tunnel release surgery: absorbable stitches that dissolve on their own and non-absorbable stitches that need to be removed. About 116 patients will be randomly assigned to one stitch type, and t…

This study tests whether a device called transcutaneous auricular vagus nerve stimulation (TaVNS) can reduce anxiety and distress in healthcare workers. Participants wear special earbuds that send gentle vibrations to stimulate the vagus nerve. Researchers will also use brain sca…

This study is a health check for students at Constructor University Bremen and employees at HAW-Hamburg. Researchers will use surveys and interviews to measure loneliness, anxiety, stress, and health habits. The goal is to understand what affects wellbeing and find ways to suppor…

This study compares stainless steel and titanium-molybdenum alloy wires for moving canine teeth in people with braces. Sixteen participants will have one type of wire on each side of their upper jaw to see which works better. The goal is to find out which wire moves teeth more ef…

This study aims to find blood-based biomarkers that signal the presence and size of plexiform neurofibromas in people with Neurofibromatosis type 1 (NF1). Researchers will collect blood samples and use whole-body MRI to compare biomarker levels in 200 participants with different …

This long-term study looks at children and adults with inherited nerve and muscle disorders that start early in life, like muscular dystrophy. Researchers will track symptoms over time and collect genetic samples from affected individuals, their family members, and healthy volunt…

This study looks at whether the Parkinson's drug Levodopa-Benserazide affects gum health and inflammation. Researchers will measure gum health and saliva markers in 136 people, including Parkinson's patients with and without gum disease, and healthy controls. The goal is to under…

This 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which…

This study is creating a statewide collection of blood samples and medical information from people with various brain blood vessel diseases, such as stroke and dementia. The goal is to build a resource that scientists can use in future research to better understand and potentiall…

Researchers are studying the natural fidgety movements of infants to see if these movements can help predict cerebral palsy and other developmental issues. They will follow 350 babies, including healthy full-term infants and those at higher risk due to medical conditions, trackin…

This study follows 30 people with Parkinson's disease who are scheduled to receive deep brain stimulation (DBS). Researchers will test whether brain scans, thinking tests, and other measures can predict who might experience cognitive decline after surgery. Participants complete t…

This study asks whether exercise can improve how the brain produces and uses energy in people with Parkinson's disease. Researchers will scan participants' brains before and after a six-month program of supervised aerobic cycling, comparing them to a group doing stretching or yog…

Researchers at Baylor College of Medicine are studying how brain activity and visible behaviors, like head movements, facial expressions, and voice tone, change during tasks that trigger OCD symptoms. Adults with OCD and healthy volunteers will complete tasks that provoke distres…

This study is collecting long-term, real-world data from about 6,000 adults being treated with Roche eye medications (Faricimab or the Port Delivery System with Ranibizumab) for conditions like wet age-related macular degeneration and diabetic macular edema. The goal is to unders…

This study explores whether combining three eye-imaging techniques can reveal how structural damage in the retina relates to vision loss in people with geographic atrophy, a form of age-related macular degeneration. Researchers will use OCT scans, ultra-high-resolution AOSLO imag…

This study follows 68 people aged 12 and older who have ABCA4 gene mutations, which can cause vision loss from diseases like Stargardt disease. Over 10 years, researchers will collect blood and skin samples and perform detailed eye exams to understand how the disease progresses. …

This natural history study follows 259 children, adolescents, and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers will track tumor growth, monitor quality of life, and perform genetic testing to better understand how the disease changes over time. The g…

This study tests whether wearable biosensors can accurately track symptoms like heart rate, breathing, sleep, and movement in people with Rett syndrome. Up to 30 participants will use the devices at home for 9 weeks and also undergo one overnight sleep study in the clinic. The go…

This 5-year study follows 250 people with von Hippel-Lindau disease who have brain or spinal cord tumors. Researchers use regular MRI scans and blood tests to see how fast tumors grow and what factors like hormones might affect growth. The goal is to better understand which tumor…

This study aims to identify genetic, biochemical, and clinical factors linked to disease severity in people with Gaucher disease and other lysosomal storage disorders. Researchers will evaluate up to 1,000 participants, including patients, carriers, and healthy controls, to bette…

This study is testing whether a blood test (liquid biopsy) can detect a rare but serious nerve tumor called MPNST earlier than current methods in adults with Neurofibromatosis Type 1 (NF1). Researchers will compare the timing of tumor detection by the blood test versus standard i…

This study follows 1,500 volunteers with normal memory, mild cognitive impairment, or Alzheimer's over several years. Researchers use brain scans, memory tests, and blood samples to track how the disease progresses. The goal is to find better ways to diagnose and treat Alzheimer'…

This study aims to find early markers of Parkinson's disease, multiple system atrophy, and Lewy body dementia by analyzing samples from the nose, blood, and urine. Researchers will compare results from 180 people with these conditions and healthy volunteers. The goal is to improv…

This study aims to find biological markers (biosignatures) in people with ALS and FTD to make diagnosis more precise. Researchers will analyze samples like spinal fluid, skin, and tears from 230 participants using advanced techniques. The goal is to create a 'disease fingerprint'…

This study will use wearable devices like heart rate monitors and sleep trackers to see how financial stress and news affect your body. Researchers will follow 2,500 adults remotely, collecting data on heart rate, sleep, and stress levels. The goal is to create AI tools that can …

This observational study aims to measure how many people in Portugal have Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare and severe forms of epilepsy. Researchers will review existing medical records from about three public hospitals to estimate the percentage of…

This study follows 46 people with gyrate atrophy, a rare genetic condition that causes vision loss, over 4 years. Researchers measure ornithine levels in the blood and track changes in the retina using eye scans and photos. The goal is to learn how the disease progresses under st…

This study aims to describe the pattern of muscle involvement in people with limb girdle muscular dystrophy caused by mutations in the anoctamin 5 gene. Researchers will collect and analyze MRI scans from 200 participants to see which muscles are affected and how much fat replace…

This study investigates whether people with neurodegenerative diseases—like Alzheimer's, Parkinson's, or ALS—show changes in behavior, social circumstances, or health before and after their diagnosis. Researchers will compare the records of over 61,000 patients with those of matc…

This study follows 850 people aged 40-64 with early-onset cognitive impairment to understand how Alzheimer's disease progresses. Participants undergo cognitive tests, brain scans, and genetic analysis, but no experimental treatment is given. The goal is to gather data that could …

This study follows people treated for anal cancer with radiotherapy to see how their sexual quality of life changes over time. Participants complete questionnaires during treatment, three months later, and two years after diagnosis. The goal is to understand the long-term impact …

This study investigates whether a combination of brain MRI scans, sleep recordings, and cognitive tests can track early brain changes in people with idiopathic REM sleep behavior disorder (iRBD), a condition that often precedes Parkinson's disease. Researchers will follow 50 peop…

This study uses health records from about 100,000 people in Israel to see if past viral infections (like Epstein-Barr, COVID-19, or herpes) increase the risk of developing mental illnesses such as depression, anxiety, bipolar disorder, or schizophrenia. It also looks at whether i…

This study follows 127 people with a genetic eye condition called USH2A-related retinal degeneration, which can cause vision loss and hearing problems. Researchers measure changes in vision, retinal sensitivity, and mobility over several years. The goal is to understand how the d…

This study follows children aged 6 to 21 who have a CNKSR2 gene mutation, which can cause epilepsy, language difficulties, and intellectual disability. Researchers will use brain scans, EEGs, and developmental tests to track how the condition changes over time. The goal is to cre…

This study looks at how Friedreich's ataxia, a genetic nerve disease, affects thinking skills like memory and problem-solving. Researchers will compare cognitive test results between people with the condition and healthy volunteers, and see if these results relate to the number o…

This study looks at how myotonic dystrophy types 1 and 2 affect the brain. About 100 adults aged 30-65 will have MRI scans, thinking and movement tests, and blood draws. Some will also have a spinal tap. The goal is to find brain changes that could be used as markers in future tr…

This study looks at 50 adults with sleep bruxism (teeth grinding during sleep) to understand how it affects sleep, pain, anxiety, and overall health. Researchers will use sleep tests, blood samples, and questionnaires to find links between bruxism and other conditions. The goal i…

This observational study follows people with early Parkinson's disease, those at genetic risk, and healthy volunteers over time. Researchers collect clinical exams, brain scans, and biological samples (blood, spinal fluid, skin, stool) to identify markers that predict how the dis…

This study follows 20 people with a rare nerve disease called CMT4J for up to 2 years. Researchers will track symptoms and health changes to better understand how the disease progresses. The goal is to find better ways to measure the disease, which could help design future treatm…

This study looks at how silicone oil, used to repair detached retinas, affects the cells on the inner surface of the cornea. Researchers will measure cell density and shape changes in 40 patients at 1 and 3 months after surgery. They also want to see if any changes go away after …

This study aimed to see if how well a person can fix their gaze relates to signs of active disease on eye scans in patients with age-related macular degeneration (ARMD) treated with antiVEGF injections. Researchers planned to follow patients for two years, measuring fixation qual…

This study was designed to look back at the health of people with Cushing's disease in Western France between 1990 and 2015. The goal was to find out how many patients went into remission and how many had their disease come back after their first surgery. However, the study was w…

This study aimed to see if Alzheimer's disease affects the nerves in the gut, similar to what happens in Parkinson's disease. Researchers planned to take extra colon biopsies during routine colonoscopies from people with Alzheimer's, Parkinson's, and healthy individuals to compar…

This study tests a short guide called the 'Jumpstart Guide' to help doctors have better conversations with dementia patients and their families about healthcare goals. Over 1,800 patients and their caregivers are taking part. Researchers will compare whether those whose doctors u…

This long-running study follows nearly 100,000 French women, mostly teachers, since 1990 to understand what factors influence their risk of cancer, heart disease, diabetes, and other major illnesses. Participants fill out questionnaires every few years about their lifestyle, medi…

This study follows people with advanced dry age-related macular degeneration who previously received a one-time injection of the experimental treatment VOY-101. Researchers are monitoring participants over the long term to check for any side effects related to the treatment. The …

This study follows 400 people with intermediate age-related macular degeneration (AMD) for about 3 years. Researchers watch how the disease advances to more severe stages and test if measuring the loss of light-sensing cells in the eye can be a useful way to track the disease. No…

This study aims to identify biomarkers—biological clues in the body—that can help distinguish between Parkinson's disease and similar conditions like progressive supranuclear palsy and multiple system atrophy. Researchers will follow people with early-stage parkinsonism and healt…

This study follows 214 men treated for prostate cancer to see how many develop lower limb lymphedema (leg swelling) and who is most at risk. Participants attend check-ups every three months for 18 months. The goal is to better understand this side effect and its impact on quality…

This study is observing 217 people with ALS and related conditions like frontotemporal dementia and hereditary spastic paraplegia. Researchers aim to connect each person's genetic makeup with their symptoms and find biological markers in blood and spinal fluid. No new treatments …

This study investigates how genetic, lifestyle, and biological factors relate to memory and thinking problems in people over 60 with Alzheimer's disease, Parkinson's disease, or mild cognitive impairment. Researchers will use brain scans, spinal fluid and blood tests, and data fr…

This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…

This Johns Hopkins study looks at whether a leaky blood-brain barrier and immune changes contribute to cognitive problems in people with ME/CFS (chronic fatigue syndrome), both those who got sick before the pandemic and those who developed it after COVID-19. Researchers will use …

This study was designed to see if a new method of measuring the visual field—by projecting vision zones on the ground—helps people with retinitis pigmentosa understand their remaining sight and improve their walking rehabilitation. The trial was withdrawn before enrolling any par…

This study tests a new radioactive compound called F 18 T807 that lights up tau tangles in the brain during a PET scan. Researchers want to see if the amount of tau relates to memory and thinking problems in people with a rare, inherited form of Alzheimer's disease. About 130 adu…

This study uses a radioactive tracer called [18F]MK-6240 to take detailed pictures of tau protein clumps in the brain, a hallmark of Alzheimer's disease. Researchers will scan 200 people from families with a rare genetic form of Alzheimer's to see how tau buildup changes over tim…

This study follows 1000 Chinese patients with myotonic dystrophy type 1 (DM1) to understand how the disease affects multiple body systems and leads to disability. Researchers will collect health data and blood samples every 3 to 6 months for several years. The goal is to identify…

This pilot study tests whether older adults with intermediate age-related macular degeneration (AMD) can eat 2 or 4 whole eggs daily for 5 months. The main goal is to see if people can stick with the diet, not to prove eggs improve vision. Researchers will also measure changes in…

This study looks at whether low blood pressure during major noncardiac surgery harms organs like the heart and kidneys. Researchers are using a non-invasive brain monitor to see if a personalized blood pressure target can be set. 650 patients at cardiovascular risk are being obse…

This study examines whether a low pulse pressure (the difference between top and bottom blood pressure numbers) in the first hours after a cardiac arrest is linked to early death. Researchers will analyze data from 3500 patients who had a cardiac arrest outside the hospital and a…

This study looks at whether diabetes or blood sugar problems can worsen thinking and memory issues in people with myotonic dystrophy type 1. Researchers will use MRI brain scans and thinking tests over 4 years to track changes. The goal is to understand why some patients have mor…

This study observes 35 people in residential treatment for substance use disorders to measure behavioral rigidity—the tendency to get stuck in inflexible thinking patterns. Participants complete weekly questionnaires and have their therapy sessions analyzed for language patterns.…

This study enrolls 500 adults with a sleep condition called REM sleep behavior disorder (RBD), which often leads to Parkinson's disease or related brain disorders. Researchers will track symptoms like thinking, movement, and smell to develop a rating scale. The goal is to prepare…

This study aims to understand why kidney tumors in people with VHL syndrome can be different from each other. Researchers will use advanced imaging, multiple biopsies, and lab-grown tumor models to map these differences. The goal is to learn more about the disease, not to test a …

This study asks 200 adults with von Hippel-Lindau syndrome to fill out an anonymous online questionnaire about their quality of life, psychological well-being, and difficulties accessing care. No treatment or medical tests are involved — the goal is simply to learn what patients …

This study is testing a virtual reality game designed specifically for people with Duchenne muscular dystrophy (DMD). Six participants aged 7 and older will play the game once using a VR headset and hand tracking. Researchers will measure how easy and enjoyable the game is, and w…

This study checks whether a French version of a scale called PLSFRS works well for people with primary lateral sclerosis (PLS), a rare nerve disease. Researchers will ask 80 patients to fill out the questionnaire during routine check-ups every 3 to 6 months. The goal is to see if…

This study looks at whether anxiety and depression are connected to carpal tunnel syndrome in medical students. Researchers will measure the median nerve in the wrist and check students' anxiety and depression levels using questionnaires. The goal is to see if there is a link bet…

This study looks at how common Charles Bonnet Syndrome (CBS) is in people with vision loss from conditions like macular degeneration or glaucoma. CBS causes complex visual hallucinations without mental illness. Researchers will survey 640 participants to measure how often it happ…

This study is testing whether taking a tiny sample of fluid from the front of the eye is safe for people with dry age-related macular degeneration (AMD). Researchers will analyze proteins in the fluid to see if they change over time. The study includes 50 adults aged 50 to 89, so…

This study is testing a new way to care for people with rare diseases. It will use advanced genetic testing and a team of specialists to help diagnose patients faster and coordinate their care better. The study involves 136 participants with certain rare diseases and aims to redu…

This study follows 450 older adults to understand why many develop a slow, unsteady walk. Researchers think it may be linked to a treatable brain condition called Normal Pressure Hydrocephalus. They will use brain scans and track health outcomes over six years to find better ways…

This study tests an adaptive deep brain stimulation (aDBS) system in just 3 people with severe, treatment-resistant OCD. The device is implanted in the brain and chest, and it aims to detect brain signals linked to OCD distress and adjust stimulation automatically. The main goal …

This study looks at how things like social support, income, and health habits are linked to mental health and brain diseases such as depression, Alzheimer's, and Parkinson's. Researchers will collect surveys, blood samples, and optional brain scans from over 1,300 adults in Europ…

This study was designed to test whether scheduled video calls with a nurse could help family members who make medical decisions for hospitalized older adults with Alzheimer's or similar cognitive issues. The goal was to improve communication and reduce anxiety and depression. How…

This study is creating a simple test to check if people with Alzheimer's or similar conditions can understand and agree to medical decisions. Researchers will work with 100-150 participants to compare the new test with existing methods and look at brain scans and blood markers. T…

This study is building an online platform called DBS MatchMaker to connect doctors around the world who treat rare movement disorders with deep brain stimulation (DBS). The goal is to help doctors share knowledge and find the best ways to treat patients. Up to 500 people of all a…

This study involves 324 adults with facioscapulohumeral muscular dystrophy (FSHD) across 14 international sites. Researchers are testing two new measurement tools—a functional test called FSHD-COM and a muscle-impedance device—to better track disease progression. The goal is to i…

This study tests whether a culturally tailored mobile health program can improve mental health and pregnancy outcomes in Black pregnant women. Half of the 150 participants will receive the program, which builds communication and self-advocacy skills and provides support. The goal…

This observational study follows 36 people with neurologic conditions like traumatic brain injury, cerebral palsy, multiple sclerosis, or Alzheimer's who are already receiving intranasal MuSE stem cell or exosome therapy as part of their routine care. Researchers track safety and…

This pilot study looks at whether light, self-paced swimming is a tolerable activity for people with ME/CFS and related conditions like Long COVID and fibromyalgia. Participants will swim for 15-30 minutes at their own pace and track symptoms and heart rate variability using an a…

This study looks at whether treating obstructive sleep apnea (OSA) with a breathing device (CPAP) lowers blood pressure more in people who are very sleepy compared to those who are not. Researchers will analyze data from about 10,000 adults with OSA from previous studies. The goa…

This study looks at how tiredness affects daily life and muscle function in older adults (65+) and people with Parkinson's disease. Researchers will use questionnaires and simple exercises like handgrip and leg movements to measure fatigue. The goal is to better understand fatigu…

This study was designed to test a wearable sensor system that tracks how people walk on different surfaces like grass, pavement, and stairs. It aimed to include healthy volunteers and people with conditions such as Parkinson's disease, dementia, stroke, multiple sclerosis, or low…

This study looks at data from 1 million adults worldwide to see if eating nuts is linked to a lower risk of diseases like diabetes, heart disease, cancer, and dementia. Researchers will combine information from many past studies to get clearer answers. The goal is to understand i…

This observational study will enroll 100,000 people over age 45 to track early signs of Alzheimer's and Parkinson's using smartphone-based tests, genetic data, and brain scans. Researchers aim to develop better ways to identify who is at risk before symptoms worsen. Participants …

This study looks at whether having low eye pressure before surgery changes how well doctors can predict your final glasses prescription after a combined cataract and retinal detachment repair. Researchers will compare two groups of people: those with low eye pressure and those wi…

This study is a national registry collecting data from 1500 people with familial adenomatous polyposis (FAP) and related conditions across 28 Italian centers. It aims to better understand how these diseases progress, how they are currently managed, and what factors influence outc…

This study brings together patient registries from 10 countries to analyze data from 700 people with spinal and bulbar muscular atrophy (SBMA). Researchers will look at changes in muscle function, walking ability, and age of symptom onset. The goal is to better understand how the…

This long-term study uses yearly brain scans, genetic tests, and thinking/memory assessments to track changes in nearly 1,800 people with various dementias (like Alzheimer's and vascular dementia) and over 140 healthy older adults. The goal is to better diagnose, monitor, and eve…

This study is analyzing blood samples from 1,799 people with Alzheimer's, dementia, depression, bipolar disorder, schizophrenia, or Parkinson's disease. Researchers want to see if certain substances in the blood can help doctors diagnose and manage these conditions more accuratel…

This study uses a small skin sample to measure levels of a protein linked to Parkinson's disease and REM sleep behavior disorder. Researchers will follow 100 participants for 18 months to see if changes in the protein match disease progression. The goal is to develop a simple bio…

This study looks at what happens in the body to cause POTS and dysautonomia after viral infections, including COVID-19. About 100 women will give a blood sample and share medical records. Researchers will study immune system markers to find clues that could lead to better diagnos…

This study aims to uncover the genetic roots of aortic aneurysms and valve disease by analyzing tissue and blood samples from 3,000 participants. Researchers will look for new disease-causing genes and factors that affect disease severity. The goal is to build a biorepository to …

This study follows about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in an earlier study. The goal is to monitor their safety and muscle function over time. No new treatment is given in this study.

This study is tracking 2000 adults with carpal tunnel syndrome who are having surgery to release the nerve. The surgery uses a special device called UltraGuideCTR, which is guided by ultrasound. Researchers are collecting real-world data on symptoms and hand function for up to 24…

This study follows 44 people with a rare genetic form of Usher syndrome caused by PCDH15 mutations. Over 48 months, researchers measure how their vision changes using eye exams and imaging. The goal is to identify the best ways to track disease progression, which will help design…

This study uses advanced MRI scans to measure blood flow in the brains of 100 adults with moyamoya disease, a condition that narrows major brain arteries and raises stroke risk. Participants will have scans and cognitive tests before and after their clinically needed surgery. The…

This study follows 25 people with limb-girdle muscular dystrophy type 2A (LGMD2A), a rare genetic disease that causes progressive muscle weakness. Researchers will measure how muscle strength changes over time and how it affects quality of life. The goal is to better understand t…

This study looks at how repeated seizures affect breathing and heart function in people with Dravet syndrome, a severe form of epilepsy. Researchers will monitor 92 children and adults for 24-48 hours using video, brain wave, heart, and breathing recordings. The goal is to find c…

The Brain Health Registry is an online project that collects information about memory, mood, sleep, diet, and exercise from adults over 18. Participants also take simple cognitive tests online. The goal is to build a large database to speed up research into brain diseases like Al…

This study follows 432 adults who were in the ICU after a traumatic injury or burn. Researchers want to see if and why patients develop long-term problems with memory, thinking, and daily function. By tracking cognitive skills and inflammation over time, they hope to uncover link…

This study follows 20 adults with hereditary transthyretin amyloidosis (ATTRv) who have both nerve and heart involvement. They are treated with patisiran or vutrisiran for two years. Researchers use MRI scans and questionnaires to see how the drugs affect heart structure and func…

This study looks at whether a new care pathway can shorten wait times for people with carpal tunnel syndrome. Researchers will compare wait times before and after the pathway is put in place. About 200 adults will take part and fill out a short satisfaction survey.

This study follows 96 people with moderate Parkinson's disease for up to 24 months. Participants take their usual medications, and doctors may adjust them as needed. Researchers will track motor symptoms using standard rating scales to better understand how the disease progresses…

This study is testing whether rehabilitation that combines physical and mental exercises (called dual-task training) can help people with stroke, multiple sclerosis, Parkinson's disease, or long COVID. Researchers will design and try out different dual-task strategies in small gr…

This study is working to create better tools for measuring muscle function in people with facioscapulohumeral muscular dystrophy (FSHD). Researchers will follow 100 adults with FSHD for up to 24 months, testing new ways to track disease progression. The goal is to make future cli…

This study was designed to track heart muscle changes in people with Duchenne muscular dystrophy using two cardiac MRIs taken two years apart, along with blood tests for heart failure markers. It planned to enroll participants aged 6 and older with a confirmed genetic diagnosis. …

This study aims to learn more about diseases that affect the brain, such as Parkinson's disease and different types of dementia. Researchers will follow 500 adults with symptoms of these conditions over time, using brain scans and genetic tests to see how the diseases progress. T…

This study follows 500 people with neurofibromatosis type 1 (NF1) to see how their skin tumors (called cutaneous neurofibromas) grow and change over time. Researchers use 3D whole-body photography to track tumor number and size each year. The goal is to better understand these tu…

This study aims to find better ways to measure disease progression in people with advanced facioscapulohumeral muscular dystrophy (FSHD) who use wheelchairs. Researchers will test various muscle strength and function tests over two years in 30 adults. The goal is to develop relia…

This study explores a personalized form of brain stimulation called resonant frequency rTMS for people with major depression. Researchers will test different stimulation frequencies to see which one best engages brain circuits. The goal is to develop a more targeted treatment app…

This study is looking at how genetics, diet, lifestyle, and medications work together to influence the risk of age-related macular degeneration (AMD), a leading cause of vision loss. Researchers will collect blood samples and health information from 1,800 adults in Portugal. The …

This study looks at why people with Postural Tachycardia Syndrome (POTS) often have trouble thinking clearly, known as 'brain fog.' Researchers will compare brain scans of 21 POTS patients and healthy volunteers while they do mental tasks, both sitting and under simulated standin…

This study planned to use brain scans to measure how a meditation technique called OM affects dopamine and stress markers in people with Parkinson's disease. It was withdrawn before enrolling any participants, so no results are available. The goal was to better understand meditat…

This study follows 35 infants with spinal muscular atrophy (SMA) who are receiving gene therapy or other advanced treatments. Researchers use small wearable motion sensors to measure how well the babies move their arms and legs over two years. The goal is to create a more precise…

This study looks at whether yearly MRI scans can find new head and neck or brain cancers early in people who had hereditary retinoblastoma and were treated with radiation. Researchers will follow 88 participants for 10 years to see if early detection leads to better surgery outco…

This study is testing a special eye test called microperimetry to see if it can detect early signs of dry age-related macular degeneration and diabetic retinopathy. Researchers will measure how sensitive the central retina is to light and compare it with standard scans. The goal …

This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over…

This study uses monthly MRI scans to closely watch brain changes in 75 people with cerebral amyloid angiopathy (CAA), a condition common in older adults that can cause bleeding and strokes. Researchers aim to understand how small bleeds and silent strokes develop over time. Parti…

This observational study will enroll 40 adults with facioscapulohumeral muscular dystrophy (FSHD) to understand how muscle degeneration affects movement. Researchers will use MRI to assess muscle health and 3D motion capture to analyze walking and posture. The goal is to create a…

This study looks at whether prolactin (a hormone) and certain inflammation markers in the blood are linked to how severe Parkinson's disease is. Researchers will measure these in 300 people with Parkinson's and compare them to standard disease severity scores. The goal is to find…

This study tests whether a wrist-worn movement recorder, called the Personal KinetiGraph (PKG), helps doctors make better treatment decisions for people with Parkinson's disease. About 231 participants will be split into two groups: one where the doctor sees the PKG data during t…

if hula can help quality of life, well being, balance and overall motor symptoms for individuals with Parkinson's disease.