Hereditary neurological disease

This trial tests a gene therapy called SPVN20, given as a single injection into the eye, for people with advanced rod-cone dystrophy, a genetic condition that causes progressive vision loss. The study includes adults with a confirmed genetic diagnosis and some remaining cone cell…

This phase 2 trial tests whether a single dose of LX2006 gene therapy can improve heart muscle thickening and damage in people with Friedreich ataxia, a genetic disorder that often leads to life-threatening cardiomyopathy. Participants aged 6 and older with confirmed heart enlarg…

This early-stage trial tests whether transplanting lab-grown dopamine-producing cells into the brains of people with Parkinson's disease is safe and might help replace the cells lost to the disease. About 7 adults with Parkinson's who have not responded well to standard medicatio…

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

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

This study tests a new gel made from donated amniotic membrane to treat large macular holes, a condition that blurs central vision. The gel is injected into the eye during surgery to help close the hole. Researchers will compare this method to the standard flap technique in 26 ad…

This study tests a gene therapy called ATSN-201 in males aged 6 and older with X-linked retinoschisis, a genetic eye condition that leads to vision loss. The therapy delivers a working copy of the RS1 gene to the retina. The trial has three parts: first, finding the right dose; s…

This study tests whether using 3-D tractography—a detailed brain imaging method—can make focused ultrasound ablation safer and more effective for people with essential tremor. Twenty-four adults with moderate to severe tremor that hasn't responded to medication will receive the p…

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

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

This study tests a drug called rugonersen, given via spinal injection, to see if it can improve thinking and communication skills in people with Angelman syndrome. The trial includes 165 children and adults aged 1 to 50. Researchers will compare the drug against a sham procedure …

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…

This pilot study tests whether transcranial magnetic stimulation (TMS) is safe and tolerable for teenagers aged 13 to 17 with obsessive-compulsive disorder (OCD) who have not improved enough with standard treatments. Participants receive TMS sessions five days a week for three we…

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

This trial tests whether a daily synbiotic supplement, which combines prebiotics and beneficial bacteria, can improve gut health and mental well-being in people with obesity. Researchers will give 120 adults with a BMI between 30 and 40 either the supplement or a placebo for 12 w…

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

This early-phase trial tests a single injection of gene therapy (ELP-02) into the spinal fluid of 8 children and young adults with CMT4J, a rare genetic nerve disease that causes muscle weakness. The therapy delivers a working copy of the FIG4 gene to nerve cells, potentially slo…

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

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

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

This study tests a combination of two drugs, trametinib and azacitidine, for children newly diagnosed with juvenile myelomonocytic leukemia (JMML), a rare blood cancer. Lower-risk patients get just these two drugs, while higher-risk patients also receive standard chemotherapy. Th…

This trial tests whether a noninvasive technique called transcranial temporal interference stimulation (tTIS) can reduce symptoms in people with obsessive-compulsive disorder (OCD). The stimulation targets the nucleus accumbens, a deep brain region linked to OCD, using electric f…

This study looks at how the body handles the drug nusinersen when given through a new implantable device called ThecaFlex DRx™, compared to the usual method of a lumbar puncture (spinal tap). About 58 people with spinal muscular atrophy (SMA) who are already in another study (PIE…

This trial tests whether using ultrasound to guide botulinum toxin injections into spastic leg muscles works better than the standard method of relying on touch and anatomical landmarks. Researchers will give 40 adults with spastic paraplegia two rounds of injections, one with ea…

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

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

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

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

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

This trial tests an experimental oral medication called VG081821AC for people with early to mid-stage Parkinson's disease. The drug blocks a brain protein called the adenosine A2A receptor, which may improve movement symptoms without using levodopa. About 152 adults in China will…

This study tests two new drug combinations for people with advanced stomach, gastroesophageal junction, or esophageal cancer that has worsened after initial treatment. About 210 participants will receive either an experimental antibody-drug conjugate plus chemotherapy, or a stand…

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

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

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

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

This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…

This study is testing the long-term safety and effectiveness of an experimental drug called CTI-1601 for people with Friedreich's ataxia, a rare genetic disease that affects movement and heart function. It includes about 85 adolescents and adults who have or have not taken the dr…

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

This early-stage study tests a stem cell therapy called ASP7317 for people with geographic atrophy, the advanced form of dry age-related macular degeneration that causes central vision loss. The treatment involves injecting special cells under the retina to replace damaged ones. …

This study is testing whether the drug risdiplam can improve motor skills in children under 2 with spinal muscular atrophy (SMA) who have already received gene therapy. The 28 participants will take risdiplam by mouth, and researchers will measure changes in their gross motor ski…

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

This phase 3 trial is testing whether adding an experimental drug called rinzimetostat to the standard drug darolutamide can slow the progression of metastatic castration-resistant prostate cancer better than current treatments. The study enrolls men whose cancer has worsened aft…

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

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

This study tests whether a daily pill called BHV-8000 can slow the worsening of motor skills in people recently diagnosed with Parkinson's disease. About 550 adults aged 40 to 85 with early Parkinson's will receive either BHV-8000 (10 mg or 20 mg) or a placebo for up to 48 weeks.…

This trial tests whether a 16-week supervised exercise program can reduce symptoms of postural orthostatic tachycardia syndrome (POTS), a condition that causes a sharp rise in heart rate and symptoms like dizziness and fatigue when standing. Researchers at Lund University will en…

This study tests whether efavirenz, a drug used for HIV, can help people with Creutzfeldt-Jakob disease (CJD) live longer. CJD is a rare, rapidly fatal brain disorder. The trial will enroll 246 adults and randomly assign them to receive either efavirenz or a placebo. The main goa…

This early-phase trial tests a new gene therapy called VG801 for Stargardt disease, a genetic condition that causes vision loss. Fifteen people aged 6 and older will receive a single injection under the retina. The main goal is to check safety, but researchers will also measure a…

This trial tests two experimental drugs, donanemab and RG6289, in people who carry a specific genetic mutation that causes an inherited, early-onset form of Alzheimer's disease. The study first gives donanemab to clear amyloid plaques from the brain, then randomly assigns partici…

This study tests a brain implant that records and stimulates brain areas to help adults with severe paralysis (from spinal cord injury, ALS, or stroke) control assistive devices like computers or wheelchairs. The main goal is to check safety, and researchers will also see if part…

This study tests whether photobiomodulation (PBM), a light-based therapy delivered by the Valeda system, can safely improve vision in people with dry age-related macular degeneration (AMD). Researchers will collect data from 500 participants in routine clinical practice across Eu…

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

This pilot study tests whether an antioxidant cocktail called Rett-T can improve motor function in children and adolescents with Rett syndrome, a rare genetic disorder that affects movement and coordination. Participants, aged 2 to 21 and at least partially able to walk, will rec…

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

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

This study tests several experimental drugs in people with NF2-related schwannomatosis, a genetic condition that causes non-cancerous tumors on nerves. The goal is to see if these drugs can shrink tumors and control the disease. About 109 participants will receive one of three dr…

This study tests new experimental drugs combined with standard chemotherapy and immunotherapy for people with advanced stomach, gastroesophageal junction, or esophageal cancer that cannot be removed by surgery or has spread. About 160 participants will receive the combination to …

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

This Phase 2 study tests a drug called apitegromab in 60 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. Participants will receive either the drug or a placebo every 4 weeks for a year. The main goal is to see if the dru…

This study follows people with wet age-related macular degeneration who received an injection of SKG0106, an experimental gene therapy, in an earlier trial. Researchers will track their eye health, vision, and any side effects for up to 5 years after the injection. The goal is to…

This early-stage study tests a new drug called LY3962681 in healthy volunteers and people with Parkinson's disease. The goal is to check if the drug is safe and how the body processes it. Participants receive either the drug or a placebo injected into the spinal fluid, and are fo…

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

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

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

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

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

This study looks at how well the drug Foslevodopa/Foscarbidopa works for people with advanced Parkinson's disease who are still in the earlier phases of that stage. About 100 adults will receive the drug as a continuous infusion under the skin for up to 6 months. Researchers will…

This trial tests whether using long-term brain recordings from daily life can improve adaptive deep brain stimulation (aDBS) for people with Parkinson's disease. Unlike standard DBS, which delivers constant stimulation, aDBS adjusts stimulation in real time based on brain activit…

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

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

This trial tests whether a Mediterranean diet, enriched with extra virgin olive oil or walnuts, can lower the chance of stroke and slow cognitive decline in people with cerebral amyloid angiopathy (CAA) or CADASIL, two conditions that affect blood vessels in the brain. Participan…

This phase 1 trial tests whether implanting a small piece of a patient's own nerve tissue into the brain during standard deep brain stimulation (DBS) surgery can support brain cells and improve Parkinson's symptoms. Twenty-four adults with Parkinson's disease who are already sche…

This phase 3 trial is testing whether a weekly injection of nomlabofusp can improve balance and slow the progression of Friedreich's ataxia, a genetic condition that damages the nervous system. The study includes about 150 adults and children with the disease, who will receive ei…

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

This early-stage study tests if implanting a small piece of the participant's own leg nerve into a key brain area is safe and feasible for people with early Parkinson's disease or Multiple System Atrophy. Seven people will take part; four will get the nerve graft and three will h…

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

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

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

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

This trial tests whether repetitive transcranial magnetic stimulation (rTMS) can reduce or delay the involuntary movements, called dyskinesia, that many Parkinson's patients develop after taking levodopa. Researchers will apply targeted magnetic pulses to a brain region involved …

This trial tests whether an internet-based form of cognitive behavior therapy (CBT) can reduce symptoms of common mental disorders like anxiety, depression, and stress-related conditions in adults seeking primary care. About 500 participants will be randomly assigned to either th…

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

This trial tests whether methylphenidate, a standard ADHD medication, can reduce attention and hyperactivity problems in children and adolescents with KBG syndrome, a rare genetic condition often accompanied by ADHD-like symptoms. Participants receive alternating blocks of the dr…

This study tests whether a computerized training program designed to improve response inhibition — the ability to stop an automatic action — can reduce symptoms in people with obsessive-compulsive disorder, trichotillomania (hair-pulling), or skin-picking disorder. Participants a…

This study tests whether the drug pridopidine can slow the worsening of Huntington's disease (HD) in adults. About 400 participants will take either pridopidine or a placebo twice daily for one year, then everyone can receive pridopidine for two more years. The goal is to see if …

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

This study looks at two ways to treat Lennox-Gastaut syndrome (LGS), a severe form of childhood epilepsy: adding more anti-seizure medications or undergoing palliative surgery (like a vagus nerve stimulator or corpus callosotomy). Researchers will track thousands of children acro…

This trial tests a non-invasive brain stimulation technique called temporal interference stimulation (TIS) in people with Parkinson's disease. It compares two different brain targets—the subthalamic nucleus (STN) and the globus pallidus internus (GPi)—to see which one better impr…

This study tests the drug acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart, weakening it. Over 36 months, researchers measure whether acoramidis improves heart function and structure using cardiac …

This trial is testing whether combining two drugs—selumetinib and APG-115—can help people with neurofibromatosis type 1 (NF1) who have nerve sheath tumors that are either pre-cancerous or already cancerous. The study aims to find a safe dose and see if the combination can shrink …

This phase 3 trial tests whether givinostat, an oral drug, can slow muscle decline in boys aged 9 to 18 with Duchenne muscular dystrophy who can no longer walk. Participants receive either givinostat or a placebo for 18 months. The main goal is to see if the drug helps maintain u…

This trial tests whether placing a piece of amniotic membrane soaked in a stem-cell-derived solution (secretome) on the nerve during carpal tunnel release surgery can improve symptom relief and nerve function. Adults with moderate-to-severe carpal tunnel syndrome that hasn't impr…

This study tests whether a 6-week digital mindfulness program, delivered through a mobile app, can help people with obsessive-compulsive disorder (OCD) manage their symptoms. The trial compares the digital therapy alone, standard medication alone, and a combination of both in 150…

This study offers NRX-101, a combination of two drugs, to adults aged 18-65 with treatment-resistant bipolar depression and suicidal thoughts. Participants receive the medication from their own psychiatrist and undergo regular check-ins on mood, suicidal ideation, and side effect…

This phase 3 trial tests whether a combination of two experimental drugs (futibatinib and zimberelimab) plus standard chemotherapy can help people with advanced biliary tract cancer live longer compared to current standard immunotherapy combinations. The study enrolls about 784 a…

This study tests whether combining a leadership alignment program with a champion strategy helps clinics in Malawi better integrate mental health care for anxiety and depression. About 1,080 adult patients who screen positive for these conditions will take part. The goal is to se…

This study tests whether electrical stimulation of a part of the brain called the deep cerebellar nuclei can safely reduce severe tremor. The participants are adults with essential tremor, cerebellar outflow tremor, or MS-related tremor that has not improved with medication or pr…

This study tests whether very low doses of radiation delivered to the whole brain are safe and can help preserve thinking and memory in people with Alzheimer's disease or certain dementias with inflammation. Participants receive an initial course of 10 radiation sessions, followe…

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

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

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

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

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

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

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

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

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

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

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

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

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

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

This study is testing ten different surgical methods to repair full-thickness macular holes, a condition that blurs central vision. 120 adults will be randomly assigned to one technique during vitrectomy surgery. The main goal is to see which method closes the hole most often and…

This study tests a new medicine called DYNE-251 in boys aged 4 to 18 with Duchenne muscular dystrophy (DMD) who can still walk. The goal is to see if it helps them move better and slows muscle damage. Participants will receive either the drug or a placebo every 4 weeks for about …

This study tests two drugs, toripalimab and celecoxib, in people with a specific type of colorectal cancer (dMMR or MSI-H) that has not spread far. The goal is to see if the treatment can make the tumor disappear completely, so some patients might not need surgery. About 105 adul…

This study tests whether a non-invasive brain stimulation technique called repetitive transcranial magnetic stimulation (rTMS) can slow cognitive decline in people with early Alzheimer's disease. The treatment targets a key brain region affected by Alzheimer's. Fifty-five partici…

This study tests whether a purine-rich diet can lower disease markers in people with AICA-ribosiduria, a rare genetic condition causing severe disability and epilepsy. Ten participants will follow a diet with 160 mg of purines per day. Early results in one patient showed promise,…

This study compares two blood-filtering treatments—immunoadsorption and plasma exchange—for people with Guillain-Barré syndrome (GBS), a rare nerve disorder that can cause muscle weakness and paralysis. Researchers will enroll 20 adults to see which method is safer and more effec…

This early-stage study tests a new medicine called FLQ-101 in 18 premature babies born at 26-27 weeks who are at high risk for retinopathy of prematurity (ROP), an eye disease that can cause blindness. The main goal is to check if the drug is safe and how it works in the body. Re…

This study looks at a blood-filtering procedure called immunoadsorption (IA) in 50 people with ME/CFS or Post-COVID syndrome who have certain autoantibodies. The goal is to see if removing these antibodies improves symptoms like severe fatigue and brain fog. Researchers will trac…

This study looks at thinking and mental health issues in people with Velocardiofacial, Williams, and Fragile X syndromes. Researchers will test how well medications like methylphenidate, fluoxetine, and risperidone improve these symptoms. The goal is to better understand and trea…

This study tests whether a radioactive tracer called 18F-S16/T807 can help diagnose tau-related brain diseases. Tau is a protein that builds up abnormally in conditions like Alzheimer's and other neurodegenerative disorders. Researchers will give 500 adults a single injection of …

This study aims to create an objective way to diagnose and monitor light sensitivity by measuring facial features, pupil reactions, and nerve responses to light. Researchers will compare 120 people with and without light sensitivity (due to traumatic brain injury or migraine) to …

This study tests a radioactive tracer called 18F-AIPF-1026 that binds to monoamine oxidase A (MAO-A), an enzyme linked to depression, anxiety, and heart injury. About 200 adults with these conditions will receive a single injection of the tracer and then undergo PET/CT or MRI sca…

This study tests whether a special imaging agent called 18F-FPyCO/SMBT-1 can help diagnose Parkinson's disease and related conditions by detecting a brain enzyme called MAO-B. About 200 adults with suspected or confirmed Parkinson's will receive a single injection of the agent an…

This study is testing three new radioactive tracers that light up alpha-synuclein clumps in the brain during PET/CT scans. Researchers want to see if these scans can accurately diagnose Parkinson's disease and related disorders. 500 adults with suspected or confirmed Parkinson's …

This study tests whether a cardiac imaging technique called a PYP scan can detect transthyretin amyloidosis in people aged 65 and older who have heart failure with preserved ejection fraction, also known as diastolic heart failure. The researchers will scan 515 participants to se…

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

This study aims to improve how doctors diagnose Cushing's syndrome, a condition caused by too much cortisol. Researchers will test the accuracy of the desmopressin stimulation test in 140 people aged 18-70 who have or may have Cushing's, plus healthy volunteers. Participants will…

This early-phase trial is testing whether a single dose of a hormone pill can help diagnose oxytocin deficiency in young people with hypopituitarism. Researchers will give the pill to 20 participants and measure oxytocin-related substances in blood and saliva. The goal is to deve…

This study aims to find the missing genetic causes of Parkinson's disease in people with early-onset or familial forms who haven't received a molecular diagnosis. Researchers will use a combination of whole genome and RNA sequencing to look for hidden genetic variants. The goal i…

This study is testing a new, noninvasive imaging device called HH-SECTR to see inside the eyes of premature infants with retinopathy of prematurity (ROP), a condition that can affect vision. The goal is to see if this technology can provide clearer, more detailed images of the re…

This early-phase trial tests a new radioactive tracer called [11C]MODAG-005 that binds to abnormal protein clumps in the brain linked to Parkinson's disease and multiple system atrophy. Nine participants (patients with Parkinson's, MSA, and healthy volunteers) will receive the tr…

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

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

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

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

This study is testing whether a new, more detailed DNA reading technique (long-read genome sequencing) can find genetic causes of dystonia that standard tests miss. Researchers will analyze blood samples from 150 people with dystonia and their family members. If it works, this co…

This study tests a new way to check for bleeding after stroke surgery using a special ultrasound through a clear skull implant. It aims to see if this method is as accurate as a CT scan, which is the current standard but is expensive and time-consuming. About 25 adults who had a …

This trial tests whether a smartphone app can help people with REM sleep behavior disorder, a condition that often precedes Parkinson's, become more physically active over two years. Participants receive personalized activity goals through the app and track their steps and exerci…

This trial tests whether a smartphone app can help 600 people with Parkinson's-related gene mutations increase their daily physical activity over two years. Participants set their own exercise goals and get feedback through the app. Researchers will measure changes in step counts…

This study tests whether eating goji berries five days a week for six months can lower the risk of age-related macular degeneration (AMD) in people aged 65 to 95. Participants will eat 14 grams of goji berries or a fiber supplement and have their eye health, gut bacteria, and oth…

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

This study tests whether an oral blood pressure drug, propranolol, can prevent severe eye disease (retinopathy of prematurity) in extremely premature infants. About 276 babies born before 28 weeks and weighing under 1250 grams will receive either propranolol or a placebo. The goa…

This study is testing a mind-body program called DNRS for people with ME/CFS (chronic fatigue syndrome) who may also have Long COVID. The program uses brain retraining to help calm overactive nerves and improve symptoms like fatigue, pain, and mood. Researchers will track quality…

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement…

This trial tests whether adding personalized feedback makes an ultra-brief online program more effective for adults with depression and anxiety. The program, called Things You Do, teaches skills to increase healthy actions like meaningful activities, healthy thinking, and social …

This study tests whether an 8-week mindfulness program can reduce anxiety and depression in adults with mild emotional distress. Participants are randomly assigned to either mindfulness training or progressive muscle relaxation. Researchers use brain scans to see if mindfulness c…

This Phase 2 trial tests whether adding Lu AF28996 to standard Parkinson's medications can extend the time patients feel their movement symptoms are well-controlled (called 'good ON time'). About 150 adults with Parkinson's who still experience motor fluctuations despite optimize…

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

This study tests a speech therapy called DTTC-Connect for children aged 3 to 12 with childhood apraxia of speech, a disorder that makes it hard to say words clearly. The therapy focuses on helping children move from saying single words to speaking in phrases. Sixty-eight children…

This study tests an online, therapist-guided program to help cancer survivors who have anxiety, depression, or fear of cancer returning. About 400 adults in Sweden who finished cancer treatment at least 6 months ago will take part. The goal is to see which parts of the 10-week on…

This study tests a four-day intensive therapy called Bergen Four-Day Treatment (B4DT) for people with obsessive-compulsive disorder (OCD). About 35 patients will receive the therapy, which involves exposure and response prevention exercises over four days, followed by three month…

This study tests whether using a stress ball for 15 minutes before retrograde intrarenal surgery (a type of kidney stone procedure) can lower patients' anxiety and fear, and improve heart rate and blood pressure. Sixty-six adults scheduled for elective surgery will be randomly as…

This study tests a short, three-session online program designed to help caregivers of people with advanced prostate cancer manage their fear that the cancer will progress. The program teaches techniques like detached mindfulness to help caregivers relate to worrying thoughts in a…

This trial tests whether a four-week improv comedy program can reduce anxiety and improve mental well-being in Chinese young adults in Hong Kong who have mild-to-moderate anxiety but no diagnosed mental disorder. Participants attend weekly three-hour improv workshops and complete…

This pilot study tests whether the Voiceitt app can help people with speech impairments caused by conditions like cerebral palsy, stroke, ALS, or Parkinson's disease communicate more effectively. Participants use the app to see if it improves speech clarity and daily communicatio…

This study tests an 8-week program called MH-POWER designed to help autistic college students manage stress, anxiety, and depression. The program combines cognitive behavioral therapy, acceptance and commitment therapy, and habit formation through weekly group sessions and daily …

This study tests an experimental drug called IRL757 in 75 people with Parkinson's disease who also have apathy (loss of interest or motivation). The main goal is to see if the drug is safe and tolerable over 12 weeks. Researchers will also check if it helps improve apathy symptom…

This pilot study tests whether hippotherapy, a type of physical therapy using the natural movement of a horse, can improve mobility, gait, balance, and quality of life in people with Parkinson's disease. Participants will be randomly assigned to receive either hippotherapy sessio…

This trial tests a program called Teachers Leading the Frontlines (Tealeaf) that trains elementary and middle school teachers in North Carolina to support students with mental health needs, such as depression, anxiety, and behavior challenges. Teachers learn to identify students …

This study at the University of Pennsylvania is testing whether personalized brain stimulation can reduce negative emotions in people with depression and anxiety. Researchers will use brain scans to map each participant's negative emotion patterns, then apply different types of t…

This pilot study will test whether a mobile phone app can help reduce depression symptoms in African American adults. Eight participants will attend six weekly 90-minute classes through the app and complete surveys over four months. The goal is to see if this approach is effectiv…

This study tests a gel called DYNAVISC placed around the nerve during repeat carpal tunnel surgery to see if it reduces pain and tingling better than standard surgery alone. About 50 adults with ongoing hand pain after a prior carpal tunnel release will take part. Results are mea…

This study tests a group program to help teens (ages 13-17.5) with Prader-Willi Syndrome and their families learn practical ways to handle irritability, meltdowns, and anxiety. Ten participants will attend weekly 90-minute sessions. The goal is to see if the program improves emot…

This study tests whether a gentle brain stimulation technique (tDCS) combined with standard speech therapy can reduce stuttering severity and its negative impact on daily life. Sixteen adults who stutter will receive both treatments, and researchers will measure changes in speech…

This study tests a non-invasive device that delivers mild electrical stimulation to the cerebellum, timed precisely to each patient's hand tremor rhythm. Ten people with Parkinson's and moderate-to-severe hand tremor will receive both real and sham stimulation to see if the real …

This study aims to help families with food insecurity and mothers with depression by providing resources like food, rent help, and mental health support. Researchers will enroll 2,000 caregivers of young children in the hospital to see if these services improve mother-child bondi…

This trial tests a program that screens cancer survivors for fear of recurrence and refers those who need help to psychological services. The program is being rolled out in cancer clinics in Hong Kong, with nurses trained to identify at-risk patients. Researchers will measure how…

This trial tests a stepped-care program to help cancer survivors manage fear of their cancer returning. The program starts with an online intervention called eConquerFear, which teaches strategies to control worry and accept uncertainty. If needed, participants receive additional…

This study tests a digital music device called MyMusiQ that delivers personalized music cues to help people with Parkinson's disease walk more easily and automatically. Participants wear foot sensors and headphones; the device adjusts the music's rhythm in real time based on thei…

This study tests whether breathing pure oxygen in a pressurized chamber (hyperbaric oxygen therapy) can help people with mild to moderate chronic fatigue syndrome. 74 participants will receive either real treatment or a sham (placebo) for 60 sessions over 3 months. Researchers wi…

This trial tests whether a program built around small, deliberate choices — called micro-choices — can help people with persistent fatigue, including those with long COVID. Participants attend a four-day group workshop and receive three weeks of digital support to build new habit…

This study tests whether wearing light therapy glasses for one hour each morning can reduce obsessive-compulsive disorder (OCD) symptoms in young adults (ages 18–35) who go to bed at 1 a.m. or later. Participants wear the glasses daily for five weeks, track their sleep, and compl…

This study tests a program called Grow with Grit, where school nurses screen children aged 8-12 in rural Montana for anxiety using a standard questionnaire. Children who show signs of anxiety receive a brief, 3-5 minute coping-skills session during a regular nurse visit, along wi…

This small pilot study tests a special standing desk that gently moves, prompting people with Parkinson's to take small steps while standing. Five participants will use the desk for up to 12 sessions over 6 weeks. Researchers will measure walking speed, motor symptoms, and thinki…

This clinical trial tests whether safinamide, an add-on medication, can reduce chronic pain in people with Parkinson's disease who also experience motor fluctuations. About 60 adults with Parkinson's and pain lasting over three months will receive either safinamide or a placebo f…

This study tests whether a specialized speech therapy that focuses on word meanings can help people with mild-to-moderate semantic aphasia—a condition where brain damage impairs understanding and use of words. Participants first undergo a control period of standard language exerc…

This study tests whether a single dose of psilocybin, given with psychological support, can improve depression in people with Parkinson's disease. Sixty adults aged 40 to 80 with mild to moderate Parkinson's and depressive symptoms will receive either a low or high dose of psiloc…

This study tests whether a new form of exposure therapy, called Expectancy Focused Exposure (EFE), can reduce anxiety symptoms and prevent fear from returning more effectively than traditional exposure therapy. Adults with anxiety disorders—such as social anxiety, panic disorder,…

This trial tests whether drinking hydrogen-rich water for 16 weeks can reduce fatigue and improve physical functioning in people with ME/CFS (chronic fatigue syndrome). Half of the 80 participants will receive the active supplement, while the other half gets a placebo. The study …

This trial tests whether adding focal tendon vibration to a two-week intensive rehabilitation program can improve balance in adults with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A causes progressive muscle weakness and poor balance, often leading to falls. Participants wi…

This study tests whether theatrical improvisation can help children and adolescents aged 8 to 17 who have elevated anxiety or depression. Participants are randomly assigned to one of three groups: improv combined with wellness techniques (like cognitive-behavioral skills), improv…

This trial tests whether a therapy that blends Exposure and Response Prevention (ERP) with Habit Reversal Training (HbRT) can reduce both obsessive-compulsive and tic symptoms in adults with Tourettic OCD — a subtype where OCD and tic disorders overlap. Standard treatments usuall…

This trial tests whether sertraline, a widely used antidepressant, can reduce anxiety in children and teens aged 8 to 17 with neurodevelopmental disorders such as autism, ADHD, Fragile X syndrome, and Tourette syndrome. Participants receive either sertraline or a placebo for seve…

This trial tests whether a culturally adapted, nurse-led program called COPE can improve the psychological wellbeing of primary caregivers of children with autism spectrum disorder in Pakistan. The program includes four weekly sessions focused on coping skills, emotional regulati…

This study tests whether two non-prescription therapies—inhaled essential oils and a wearable TENS device—can help adults with chronic nausea and anxiety. Participants use the essential oil products as needed for 12 weeks, then switch to the TENS device for another 12 weeks, whil…

This phase 2 trial tests whether a single dose of psilocybin, the active ingredient in magic mushrooms, can improve depression in people with Parkinson's disease. Forty participants will receive a low to high dose with psychological support. The study measures changes in depressi…

This trial tests a 12-session telecare program for children aged 7 to 17 with limb-girdle muscular dystrophy. The group-based intervention focuses on social cognition, emotional management, and coping strategies to improve quality of life and reduce symptoms. Participants attend …

This study investigates whether complex rhythmic movement training, like dancing with new partners, can improve mobility and learning in people with Parkinson's disease or mild cognitive impairment. Researchers will compare how participants with Parkinson's, those with memory pro…

This study tests whether inhaling DMT, a psychedelic drug, can quickly reduce symptoms of major depression and suicidal thoughts. 140 adults with moderate-to-severe depression will receive either a high or low dose of DMT in two puffs. Those not improved after a week can get an e…

This study explores whether virtual reality can help people with Parkinson's disease exercise harder and enjoy it more while cycling. Participants ride a stationary bike in different conditions—with and without virtual reality, and with different cycling styles—to measure intensi…

This study tests whether a 30-minute virtual reality session with nature scenes and music can reduce pain, anxiety, and improve comfort in people who just had hip replacement surgery. Participants choose from forest, beach, underwater, or outdoor footage. The goal is to see if VR…

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

This study tests whether giving children a sugary drink (maltodextrin) two hours before surgery can reduce their anxiety and improve recovery. Children aged 3 to 17 having day surgery will receive either the sugary drink or a plain drink like water or tea. Researchers will measur…

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

This study compares group-based Acceptance and Commitment Therapy (ACT) to standard individual therapy for young people aged 16-25 with depression, anxiety, or stress. About 212 participants will be randomly assigned to either group ACT or individual therapy. The goal is to see i…

This study tests whether standard carpal tunnel surgery—cutting a ligament in the wrist—actually relieves symptoms like numbness and pain better than a pretend surgery where the ligament is not cut. About 180 adults with carpal tunnel syndrome will be randomly assigned to real or…

This study looks at whether a visual rehabilitation program can ease anxiety and depression in people with advanced age-related macular degeneration (AMD). Twenty-two participants will attend five group sessions over 60 days, learning skills like coin recognition, using voice ass…

This study tests two types of speech therapy for adults with mild-to-moderate primary progressive aphasia, a condition that slowly impairs language. Researchers will compare the therapies in 200 participants to see which better improves communication and reduces caregiver burden.…

This study tests an online program called My Wellbeing Guide for cancer patients who feel depressed or anxious. The program uses videos, activities, and written tips based on cognitive behavioral therapy over 7 weeks. About 6,500 participants will be randomly assigned to the prog…

This study tests whether a special kind of talk therapy, called Emotion-Focused Acceptance and Commitment Therapy (ACT), can reduce symptoms of Obsessive-Compulsive Disorder (OCD). About 36 adults with OCD will be randomly assigned to either ACT group therapy or a supportive grou…

This study tests whether L-dopa, a drug used for Parkinson's, can improve movement and communication in children with a rare genetic disorder called CTNNB1 syndrome. The condition causes developmental delays, muscle stiffness, and trouble walking. Seven children aged 1 to 15 will…

This small pilot study tests whether a special rocking bed (Somnomat Casa) can improve sleep quality in people with Parkinson's disease. Fifteen participants will use the bed at home for two months, and researchers will measure how acceptable and helpful the treatment is. The goa…

This study tests whether watching augmented reality (AR) health education videos can help reduce anxiety and depression in women after breast cancer surgery. 60 female patients will be randomly assigned to receive either standard education alone or standard education plus AR vide…

This study tests two extra therapies added to standard exercise rehab for people with Parkinson's disease: robot-assisted walking training and electrical stimulation of the thigh muscle. Forty participants will be randomly assigned to one of the two groups and undergo treatment f…

This study tests whether bright light therapy can help reduce excessive daytime sleepiness in people with Prader-Willi syndrome. About 50 participants will receive either bright light or a dim placebo light. The goal is to see if the therapy improves alertness, mood, and behavior…

This study tests whether a structured emotional intelligence (EI) training program can improve mental health and reduce anxiety and depression among secondary school teachers and students in Dhaka, Bangladesh. The program teaches skills like self-awareness, self-management, socia…

This study tests whether a mindfulness-based program can help adults with Tourette syndrome or persistent tic disorders reduce their tics. Participants will either learn mindfulness techniques to manage urges or receive standard relaxation and coping support. The goal is to see w…

This study tests an accelerated form of transcranial magnetic stimulation (TMS) for people with obsessive-compulsive disorder (OCD) who haven't gotten better with standard treatments. Over 7 days, 30 participants will receive 28 sessions of brain stimulation targeting areas linke…

This study tests whether psilocybin (the active ingredient in magic mushrooms) can safely reduce depression in adults with COPD, ALS, MS, or atypical Parkinson disease who have at least 6 months to live. Participants receive either two moderate-to-high doses (15mg then 25mg) or t…

This study looks at whether eating more salt can help people with POTS, a condition that causes a fast heart rate and dizziness when standing. Researchers will compare a high-salt diet to a normal-salt diet over 3 months in 50 adults. The goal is to see if extra salt lowers heart…

This study looks at whether wearing compression garments (like tight waist-high stockings or abdominal shapewear) can help adults with POTS feel better and lower their heart rate during normal daily activities. About 30 participants will wear the garments at home and have their h…

This study tests an online psychological program called MY-CHOICE for adults with ischemic heart disease who also have anxiety or depression. The program offers personalized online modules and therapist calls over 6-12 weeks. Researchers want to see if patients find it acceptable…

This study tests whether a device that gently stimulates a nerve in the ear (transcutaneous vagus nerve stimulation) can help people with chronic fatigue from fibromyalgia or long COVID get more benefit from exercise. 60 adults who still feel very tired after a standard exercise …

This study looks at whether doing resistance exercises (like leg presses) can reduce swelling in the lower legs of people with lymphedema. It also checks if wearing compression garments during exercise makes a difference. Thirty-six adults with lower limb lymphedema will exercise…

This study tests whether a gentle electrical brain stimulation technique called tDCS can improve cognitive problems in people with Parkinson's disease who have mild memory or thinking issues. About 36 participants will receive the stimulation while their brain activity and cognit…

This study tests whether low or moderate doses of LSD can reduce anxiety, pain, and depression in people with advanced or terminal illnesses. Sixty participants will receive either two moderate doses or two very low (placebo-like) doses of LSD in a double-blind setup. The goal is…

This Stanford study tests how ketamine quickly reduces OCD symptoms by blocking opioid receptors with naltrexone. 150 adults with OCD who haven't responded to standard treatments will receive ketamine plus either naltrexone or a placebo. The goal is to see if the opioid system is…

This study tests whether a non-invasive brain stimulation technique called tDCS, combined with cognitive training, can improve thinking skills and mood in people with mild cognitive impairment or early dementia. Fifty participants will receive both real and placebo stimulation in…

This study tests whether simple physical maneuvers like leg crossing, bending, or arm tensing can prevent fainting in children aged 6 to 18. Participants who visit the emergency department after a fainting episode will either receive standard care or learn these counterpressure m…

This study tests whether a single 25 mg dose of psilocybin can relieve major depression in 100 adults with cancer, compared to a very low 1 mg dose. Participants must be 20–80 years old, have had depression for at least a month after their cancer diagnosis, and stop other antidep…

This study looks at whether art therapy, delivered through virtual reality glasses or in person, can help reduce anxiety and improve sleep and well-being in people with colorectal cancer. 78 patients receiving chemotherapy will be randomly assigned to one of three groups: virtual…

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

This study offers genetic testing and counseling to people with Parkinson's disease. Researchers aim to identify genetic mutations linked to the condition and build a large database for future research. Participants learn their genetic status, which may empower them to join clini…

This study collects blood or brain tissue from families with two or more members diagnosed with Alzheimer's or dementia. The goal is to build a large DNA bank that researchers can use to find genes linked to the disease. No individual results are returned, but the data may lead t…

This study tests whether immune cells called monocytes from people with the atrophic form of age-related macular degeneration (AMD) survive longer on retinal cells than monocytes from healthy people. Researchers will collect blood samples from 80 participants, extract monocytes, …

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

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

This study is testing a new non-invasive device that uses laser speckle to measure blood flow and structure inside the eye. Researchers will compare these images with standard vision tests in 500 people with various retinal conditions. The goal is to see if this technology can be…

This study uses special MRI scans to measure the flow of cerebrospinal fluid and blood in the brain and spine of people with Chiari malformation. The goal is to see how these flows differ between people with symptoms and those without. Researchers will compare these measurements …

This study is looking for simple ways to detect early signs of multiple sclerosis and Parkinson's disease in people who drive. Researchers will test 200 drivers with these conditions using computer-based attention and reaction tests. The goal is to find quick, easy tests that cou…

This study creates a registry of pregnancies where the fetus received a genetic diagnosis of spinal muscular atrophy (SMA) and was treated with a disease-modifying therapy while in the womb, such as risdiplam. Researchers will collect information from medical records and question…

This study follows 300 people with CLN3 Batten disease, a rare genetic disorder that causes vision loss, seizures, and decline in thinking and movement. Researchers collect samples like blood and spinal fluid to find biological markers that could be used in future treatment trial…

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

This study monitors pregnancy outcomes in women who took patisiran-LNP for hereditary ATTR amyloidosis. Researchers will track birth defects, pregnancy complications, and infant health. The goal is to better understand the drug's safety during pregnancy, not to test a new treatme…

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

This observational study will follow 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, for many years. Researchers will track how the disease progresses, how patients are treated in real-world settings, and the safety of two approved drugs, pat…

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

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

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

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

This observational study follows 500 people with atypical parkinsonian syndromes, including multiple system atrophy, dementia with Lewy bodies, progressive supranuclear palsy, and corticobasal degeneration. Researchers track motor and non-motor symptoms and imaging changes every …

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

This observational study collects health information from up to 500,000 adults with and without Parkinson's disease. Researchers aim to identify risk factors and early signs that predict who may develop the disease and how it progresses. Participants report their symptoms and med…

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

This study creates a database and biobank for people with Li-Fraumeni syndrome or related TP53 gene changes, conditions that raise the lifetime risk of cancer to as high as 90%. Researchers will collect clinical data, blood samples, and tumor tissue from up to 300 participants to…

This study investigates how deep brain stimulation (DBS) affects cognitive and psychiatric symptoms in people with Parkinson's disease. Researchers will compare the effects of stimulating the left versus the right hemisphere of the brain on tasks like verbal fluency and impulsivi…

This study follows 1,000 adults with myotonic dystrophy type 1 (DM1) over time to measure how the disease progresses. Researchers will track walking speed, hand muscle relaxation, grip strength, heart and lung function, and daily activity. The goal is to establish reliable biomar…

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

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

Researchers are building an international registry to collect health information from people with congenital central hypoventilation syndrome (CCHS), a rare condition that affects breathing control. The study asks patients with a confirmed PHOX2B gene mutation to fill out surveys…

Researchers are investigating whether narrowing of the ophthalmic artery, which supplies blood to the eye, contributes to geographic atrophy, an advanced form of dry age-related macular degeneration. The study will use a noninvasive imaging technique called PC-CTA to measure arte…

This study looks at how deep brain stimulation (DBS) affects the ability to stop actions in people with Parkinson's disease. Researchers will measure brain waves and behavior during impulse control tasks before, during, and after DBS surgery. The goal is to understand how stimula…

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

This study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.

This study follows 50 adults with facioscapulohumeral muscular dystrophy type 2 (FSHD2), a rare inherited muscle disease, for 18 months. Researchers will measure muscle function and severity using standard clinical tests and collect blood samples to look for markers that might pr…

This study measures thinking, memory, and attention in people with congenital central hypoventilation syndrome (CCHS), a rare condition that can reduce oxygen to the brain. Participants complete a 45-minute app-based cognitive assessment on an iPad, and parents or adult participa…

This study builds a global online registry to collect long-term health information from children and adults with Angelman Syndrome. Parents and caregivers report on diagnosis, symptoms, and daily functioning over time. The goal is to better understand the condition's natural cour…

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

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

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

This pilot study tests whether different volumes of aerobic exercise produce different changes in a blood marker called BDNF, which supports brain health. Researchers will enroll 30 adults with Parkinson's disease and assign them to either a standard rehabilitation program (180 M…

This study follows 392 people with stage I-III colon cancer who have had surgery to remove their tumor. Researchers will take blood samples at regular intervals over three years to look for tiny pieces of tumor DNA, called molecular residual disease (MRD). The goal is to see if a…

This study follows 600 people with neurodegenerative diseases such as Parkinson's, Alzheimer's, and related conditions over 1 to 10 years. Researchers collect data from routine clinical exams, brain imaging, movement analysis, and blood tests. They use statistical tools and artif…

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

This study looks at how children who stutter process speech sounds compared to children who do not stutter. Researchers will use hearing tests, brain wave recordings (EEG), and brain scans (fMRI) to measure brain activity. The goal is to understand the differences in speech proce…

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

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

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

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

This study follows 3,000 people with Friedreich ataxia, a rare inherited disease that affects movement and coordination, to map how the condition changes over time. Researchers will measure symptoms yearly using standard rating scales for up to 25 years. The goal is to build a de…

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

This study uses ultrasound, a safe imaging technique that uses sound waves, to get detailed pictures and measurements of nerves and muscles. Researchers aim to collect normal values from healthy adults and compare them with data from people who have neuromuscular disorders. The g…

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

Researchers at the University of Pennsylvania are building a large repository of blood, DNA, and family history data from people with age-related brain diseases like Alzheimer's, Parkinson's, and ALS, as well as their family members. The goal is to support studies into the geneti…

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

This study follows thousands of people with ataxia, hereditary spastic paraplegia, and spastic ataxia, rare conditions that cause progressive problems with walking, balance, and coordination. Researchers will track how symptoms evolve using clinical exams, patient reports, digita…

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

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

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

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

This study is testing whether a simple vision screening program in general practice can help detect vision loss early in adults over 70 who have at least one chronic condition. Participants will have three vision tests done by their GP or nurse, then visit a collaborating opticia…

This study looks at adults who had a traumatic brain injury at least a year ago. Researchers want to see whether those with growth hormone deficiency have worse quality of life, fatigue, mood, and sleep than those without it. Participants complete questionnaires and a test for gr…

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

This study looks at how well people with retinal diseases like retinitis pigmentosa and Stargardt's disease see in the dark. Researchers want to find out if measuring dark adaptation can help detect and track these conditions. Healthy volunteers and people with retinal disease ag…

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

This small study tests a smartphone app that creates a 'digital twin' of the patient to help manage health after a stroke, moyamoya disease, or cerebral amyloid angiopathy. Twenty adults will either use the app plus standard care, or standard care alone. The goal is to see if pat…

This study looks at people with REM sleep behavior disorder (RBD), a condition where people act out their dreams and which often leads to Parkinson's disease. Researchers will use advanced brain imaging, sleep tests, and physical exams to compare 50 people with RBD to 50 healthy …

This study looks at women who carry the gene for X-linked adrenoleukodystrophy (X-ALD), a rare inherited condition that mainly affects the nervous system. Although the disease is linked to the X chromosome, up to 80% of female carriers may develop symptoms like leg weakness and b…

This study aims to create a network of biobanks across Italy that collect and store biological samples and health data from people with rare diseases and their families. The goal is to standardize how samples are collected and shared, so researchers can access high-quality materi…

This study aims to build a comprehensive 'physiological brain atlas' by collecting electrical and other data from patients undergoing stereotactic brain surgery for conditions like Parkinson's disease, OCD, and epilepsy. The goal is to map this information onto a standard brain t…

This observational study aims to paint a detailed picture of the physical, cognitive, and clinical characteristics of people with Parkinson's disease in Galicia, Spain. Researchers will also test whether a short physical performance battery (SPPB) is a reliable and valid tool for…

This study explores whether methane gas produced by gut microbes is linked to the progression of Parkinson's disease and related conditions like dementia. Researchers will measure breath methane levels in 200 adults—including those with Parkinson's, those at high risk (REM sleep …

This study investigates whether specialized visual function tests can detect early changes in people with early or intermediate age-related macular degeneration (AMD). Researchers will follow 120 adults with good visual acuity but early signs of AMD, using tests like microperimet…

This study looks at how the nerves connecting the gut and brain affect Parkinson's disease. Researchers will compare people with Parkinson's, those at risk, and healthy volunteers. Participants will give blood, stool, and saliva samples, have X-rays and colon exams, and answer qu…

This study investigates why some people with early-onset Alzheimer's disease or a variant called posterior cortical atrophy show different patterns of brain damage. Using advanced PET and MRI scans, researchers will map tau protein deposits and brain network activity to identify …

This study follows a large group of people with Parkinson's disease and related conditions in France to map how the disease unfolds over time. Researchers collect detailed information on symptoms, treatments, and genetic samples at regular visits. The goal is to identify distinct…

This study looks at whether a standard dose of the antibiotic cefazolin, given before surgery to place a ventricular shunt, reaches high enough levels in the cerebrospinal fluid to prevent infection. Researchers will measure cefazolin in the fluid and blood of 15 adults undergoin…

This study looks at whether liver function and blood clotting tests can help doctors better understand and monitor cardiac transthyretin amyloidosis (ATTR-CA), a condition where abnormal protein builds up in the heart. Researchers will compare these tests in people with ATTR-CA a…

This study aims to understand brain changes in people with mild cognitive impairment (MCI) and Alzheimer's disease. Researchers will use MRI, EEG, cognitive tests, and blood and stool samples to find differences between healthy individuals and those with cognitive impairment. The…

This study investigates the biological mechanisms behind young-onset Parkinson's disease, which begins before age 50. Researchers will analyze genetic tests and protein markers in spinal fluid and skin biopsies from 250 participants to identify distinct disease subtypes. The goal…

This study follows children with congenital or childhood myotonic dystrophy over time to see how their symptoms change. Researchers will test a range of assessments, from walking speed and hand strength to language and quality of life, to find the best ways to measure the disease…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study is a large registry that collects health and genetic information from people with a personal or family history of pancreatic tumors. The goal is to find the genes and other factors that cause pancreatic cancer to run in some families. By studying thousands of participa…

This study follows people with SPLIS, a rare inherited metabolic condition, to map its natural history—how the disease unfolds over time. Researchers will track growth, organ function, quality of life, and survival in about 28 participants. The goal is to understand the full rang…

This study looks at whether levels of a protein called HMGB1 are different in people with LAMA2-related muscular dystrophy, a condition that causes severe muscle weakness. Researchers will measure HMGB1 in blood and muscle samples already collected from about 25 patients. The goa…

This study follows people with a genetic condition called OPA1-associated optic atrophy, which causes gradual vision loss. Researchers will use a battery of eye exams—including vision charts, contrast sensitivity, and retinal imaging—to see how the disease changes over time. The …

This study follows adults with a suspected bone or soft tissue tumor to see how their stress and anxiety levels change at three critical moments: before biopsy, after learning the diagnosis, and when treatment begins. Participants fill out standard questionnaires about anxiety, d…

This large observational study is investigating whether people with Parkinson's disease who carry a mutation in the GBA1 gene have a higher chance of developing cancer. Researchers will compare cancer rates in this group to those in Parkinson's patients without the mutation and t…

This study is testing whether a non-invasive eye scan called optical coherence tomography (OCT) can help track neurological diseases like multiple sclerosis, Alzheimer's, Parkinson's, and migraine. Over two years, researchers will measure the thickness of retinal layers in 840 pa…

This study investigates post-exertional malaise (PEM), the hallmark symptom of ME/CFS, where even minor activity triggers severe symptom worsening. Researchers will compare 50 ME/CFS patients with 50 healthy volunteers matched by age, sex, and activity level. Participants undergo…

This study uses advanced PET scans and blood tests to examine brain inflammation and white matter damage in people with corticobasal syndrome, a rare movement and thinking disorder. Researchers will compare these brain changes across different subtypes of the condition and agains…

This study looks at children in Vietnam who have movement disorders—like tremors, jerks, or coordination problems—with no known cause. Researchers will collect medical information and blood samples to search for genetic changes that might explain these conditions. The goal is to …

This study uses advanced PET/MRI scanning to examine the noradrenaline system in the brains of 165 healthy adults and people with Parkinson's disease. Participants receive a radioactive tracer that binds to specific receptors, allowing researchers to see how this system changes w…

This study investigates how a small brain region called the subthalamic nucleus controls movement in both healthy people and those with Parkinson's disease. Researchers will measure brain cell activity and electrical signals during arm movements and walking to understand why move…

This study uses special PET scans to measure brain inflammation and tau protein buildup in people with different forms of Alzheimer's disease, including those with memory, vision, or language problems. Researchers aim to understand how inflammation relates to other brain changes …

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

This study follows 50 children and adults with Baker Gordon Syndrome, a rare genetic disorder, to track how the condition changes over time. Participants will undergo brain scans, EEGs, and genetic testing, and complete developmental assessments. The goal is to create better tool…

This study looks at why people with Alzheimer's disease may move more slowly. Researchers will use brain MRI scans and simple reaction time tests in 155 participants with mild cognitive impairment or early dementia. The goal is to link brain lesions to attention-related motor slo…

This study looks at how silicone oil, used to hold the retina in place after a retinal detachment, affects the retina's electrical activity. Researchers will measure these signals in 50 people who had surgery for a detached retina. The goal is to better understand how the oil imp…

This study looks at how epileptic brain activity during sleep can cause awakenings and change dream recall or content. Researchers will use tiny electrical pulses in the brain during sleep to trigger mild epileptic activity and observe the effects. The goal is to better understan…

This study follows 20 people from families with a genetic risk for frontotemporal dementia (FTD), including those already diagnosed and those at risk. Over five years, researchers will track changes in health and brain function to better understand how the disease develops. The g…

This study tests whether computer analysis of speech can reliably detect motor, emotional, and cognitive changes in people with Parkinson's disease. Researchers will record speech from 80 patients while they are on and off medication or deep brain stimulation. The goal is to find…

This study explores whether the gut's nervous system can reveal early signs of Parkinson's disease. Researchers will analyze gut tissue and microbiome samples from 60 people aged 45-75 during routine colonoscopies. The goal is to find biomarkers that reflect brain changes, not to…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study follows 35 men in residential rehab for severe substance use. They write weekly stories about their feelings and challenges, and fill out questionnaires every two weeks. Researchers use natural language processing to analyze the stories and look for patterns that might…

This study looks at whether special brain cells called tanycytes play a role in Alzheimer's disease and frontotemporal dementia. Researchers will measure hormone levels in blood and spinal fluid from 102 participants to see if these cells help control metabolism and hormone signa…

This study tests a structured program to help adolescents with epilepsy transition from pediatric to adult healthcare. Researchers will compare how many patients attend their first adult clinic appointment and whether they return to pediatric care. The goal is to improve self-man…

This study aims to find genes that increase the risk of developing Alzheimer's disease. Researchers are recruiting 10,000 people, including those with Alzheimer's and their family members, from all ethnic backgrounds. By analyzing DNA, they hope to discover new genetic clues that…

This study follows 450 people with Parkinson's disease for four years to learn why some become less active and what helps them stay active. Participants wear activity monitors and complete tests and questionnaires. The goal is to identify early warning signs of declining activity…

This study looks at whether new, quick tests that measure how fast people can move can help doctors diagnose brain diseases like Alzheimer's and predict recovery after a stroke. Researchers will test 129 adults aged 40-85 with stroke or memory problems. The goal is to see if thes…

This study looks at whether a child's genes can predict if they will have certain side effects from common antidepressants (SSRIs). Researchers will collect saliva samples from 120 children and young adults aged 6 to 24 with depression, anxiety, or OCD. The goal is to find geneti…

This study tests an artificial intelligence algorithm that uses wearable sensors to detect freezing of gait—a sudden inability to walk—in people with Parkinson's disease. Researchers want to see if the AI works accurately in home environments, not just in the lab. About 126 parti…

This study aims to understand why some people with Parkinson's disease experience central pain. Researchers will use brain scans (PET and MRI) to examine the serotonin system in 34 participants. The goal is to see how serotonin relates to pain, not to test a new treatment.

This study follows 450 people with FSHD for at least 3 years to measure how the disease affects walking, arm use, breathing, and other daily activities. The goal is to improve patient care and help researchers design better clinical trials by understanding what changes in movemen…

This study looks at how deep brain stimulation (DBS) affects brain activity in people with essential tremor. Researchers will use EEG and MEG to record brain signals with the stimulator on and off, and also test other tremor-relief methods like cooling or medication. The goal is …

This study uses brain imaging (PET scans) and memory tests to understand why some people with Parkinson's disease develop dementia. Researchers will follow 320 participants over several years, using scans to look for abnormal proteins and track thinking changes. The goal is to id…

This study looks at tremor (shaking) in people with Charcot-Marie-Tooth (CMT) disease, a nerve condition. Researchers will use muscle and movement tests to better understand why tremor happens and how it affects daily life. About 75 adults with CMT will take part, including some …

This study follows 140 adults with Tourette syndrome for two years to see how stress from life events impacts tic severity, depression, anxiety, and quality of life. Participants complete interviews and questionnaires about their tics, mood, and daily functioning. The goal is to …

This study looks at whether giving low-income adults aged 40 and older free meal kits and nutrition education can improve their food security, diet quality, mental health, and heart health. About 120 participants will be split into two groups: one gets meal kits plus nutrition cl…

This study looks at how muscles grow and change in children with cerebral palsy (CP) by examining tiny samples from leg muscles. Researchers will compare these microscopic findings with muscle images and movement tests. The goal is to better understand why muscles in CP develop d…

This study uses a special camera called an adaptive optics scanning laser ophthalmoscope to take incredibly detailed pictures of the retina, the light-sensitive layer at the back of the eye. Researchers will compare images from healthy people and those with eye diseases like Star…

Researchers at the University of Pittsburgh are testing whether a short, non-invasive brain stimulation technique called continuous theta burst stimulation (cTBS) can alter emotional and physical responses to stress. Fifty-five healthy adults will receive both real and sham stimu…

This study is testing a new way to measure fatigue in people with spinal muscular atrophy (SMA), a condition that causes muscle weakness. The test is designed to be easy enough for patients with different levels of ability. Researchers want to see if the test is safe, doable, and…

This early study tests whether a brain implant can help people with severe paralysis from conditions like ALS, spinal cord injury, or stroke control devices using their thoughts. The implant records brain signals to decode text or synthesized speech. Only 3 adults will participat…

This study looks at how often people with Charcot-Marie-Tooth disease (CMT) have problems with their autonomic nervous system, which controls things like heart rate and sweating. Researchers will use questionnaires and simple, non-invasive tests to measure these symptoms in 50 ad…

This study at Mayo Clinic looks at two different types of Progressive Apraxia of Speech, a condition that makes it hard to speak clearly. Researchers will use brain scans, speech assessments, and thinking tests in 47 adults to better tell these types apart. The goal is to improve…

This study looks at how social and emotional thinking skills relate to everyday behavior in people with Alzheimer's disease and similar conditions. Researchers will give 120 participants tests of social cognition and ask their study partners to complete behavior checklists. They …

This study looks at whether taking a hot bath before or after muscle-damaging exercise helps muscles recover faster. Researchers will measure muscle function and a protein called HSP70, which helps protect and repair cells. The study involves 96 healthy young male athletes who ex…

This study follows 140 people with early signs of normal pressure hydrocephalus (NPH) to see if brain scans and fluid tests can predict who will get worse and need shunt surgery. Participants include those with mild symptoms, those with clear NPH, and healthy volunteers. The goal…

This study looks at how the heart and blood vessels communicate when blood pressure drops, like when standing up quickly. Researchers will test young and older men and women using a vacuum chamber around the legs to safely mimic low blood pressure. They will also see if squeezing…

This study is collecting medical information and biological samples (like blood, urine, and kidney tissue) from 300 people with FSGS or MCD who have had or will have a kidney transplant. The goal is to build a research biobank called RESOLVE to help scientists study why these dis…

This study looks at how a focal seizure (a seizure starting in one part of the brain) temporarily changes a person's ability to pay attention. Researchers will test 100 adults with epilepsy before and after a seizure using attention tasks and brain wave recordings. The goal is to…

This study tests two advanced eye imaging techniques—central microperimetry and radial OCT—to see if they can more accurately measure the progression of geographic atrophy, a severe form of age-related macular degeneration that can lead to blindness. Researchers will follow 80 ad…

This study looks at 300 people with Lynch syndrome, a genetic condition that raises the risk of colorectal cancer. Researchers want to understand why some patients still develop cancer even with regular check-ups. They will analyze blood, gut bacteria, and hair samples to find cl…

This study looks at how sleep apnea changes pressure in the brain and belly during sleep in people with normal-pressure hydrocephalus. Researchers want to see if these pressure changes affect how well a surgically placed shunt (a tube that drains fluid from the brain) works. The …

CAPTURE ALS is a long-term study that collects health data and biological samples from 150 people with ALS and related conditions. The goal is to create a shared resource for researchers worldwide to better understand the disease and develop new treatments. No experimental drugs …

This study aims to validate a questionnaire that measures anxiety in cancer patients before surgery. Researchers will test the Italian version of the Surgical Anxiety Questionnaire (SAQ) with 180 adult patients. The goal is to better understand patients' fears and concerns so tha…

This study enrolls 25 people with severe retinal diseases like retinitis pigmentosa and Stargardt disease to test new ways of measuring visual function. Researchers will evaluate how well participants can perform various vision tests, such as navigating obstacles or detecting lig…

This study looks at 100 older adults with normal pressure hydrocephalus (NPH) to find out why some get better after a shunt surgery and others do not. Researchers will use brain scans, blood tests, and wearable monitors to check for nerve damage and signs of dementia. The goal is…

The goal of this clinical trial is to compare benzhexol and procyclidine for improving tremor in adults with tremor-predominant Parkinson's disease who are taking levodopa. Researchers will compare the two medicines to determine whether benzhexol provides similar improvement in t…

The UPGRADE Study will examine how food-processing level and nutritional quality affect energy intake, eating rate, satiety, and glycemic response. In a randomized-order crossover design, each participant will consume three laboratory test meals: low processing/high nutritional q…