Hereditary neurological disease

This Phase 2 trial tests a combination of pembrolizumab (Keytruda) and quavonlimab against other drug combos in 302 people with stage IV colorectal cancer that has a specific genetic feature (MSI-H or dMMR). The goal is to see if the new combo shrinks tumors better and for longer…

This study follows 165 children who previously took dabrafenib and/or trametinib for certain brain tumors. Researchers want to see if these drugs are safe over the long term and how they affect growth. The children continue their treatment and are monitored for side effects and d…

This study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice d…

This study tests the safety of a drug called ASP3021 (avacincaptad pegol) for people in Japan with geographic atrophy, an advanced form of age-related macular degeneration that causes vision loss. Twenty participants will receive monthly injections into the affected eye for 12 mo…

This study investigates whether combining the medication lovastatin with intensive reading tutoring can improve reading abilities in children and young adults (ages 8-20) with Neurofibromatosis Type 1 (NF1) who have reading disabilities. Participants first receive either lovastat…

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

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

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

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

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

This study tests deep brain stimulation (DBS) surgery for people with severe focal hand dystonia, including musician's dystonia, that hasn't improved with botulinum toxin. Five participants will have electrodes placed in brain areas that control movement, connected to a stimulato…

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

This study 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 early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assig…

This study tests whether metformin, a widely used diabetes drug, can safely slow vision loss in people with ABCA4 retinopathy (Stargardt disease). Fifty-five participants aged 12 and older will take metformin by mouth for 24 months, with regular eye exams to track changes. The go…

This early-stage study tests two drugs together—linvoseltamab and dupilumab—in just 6 adults with severe food allergies. The goal is to see if the combination is safe and can lower IgE antibodies that trigger allergic reactions. Participants must have a history of anaphylaxis to …

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

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

This phase 3 trial tests whether a new drug combination (MK-7684A) plus chemoradiotherapy helps people with stage III non-small cell lung cancer live longer than the current standard treatment. About 611 participants will receive either the new combo or durvalumab. The study is a…

This early-stage trial tests an experimental drug called BI 3000202 in 16 adults with rare type 1 interferonopathies, such as Aicardi-Goutières syndrome. Participants take a low dose for 4 weeks, then a higher dose for 36 weeks. The main goal is to see if the drug is safe and how…

This phase 2 trial tests the safety and effectiveness of avelumab, an immunotherapy drug, in people with thymoma or thymic carcinoma that has progressed after platinum-based chemotherapy. About 56 participants will receive avelumab intravenously every two weeks. The study aims to…

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

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is…

This study planned to test whether injecting botulinum toxin (Botox) into the salivary glands could reduce drooling in people with true bulbar palsy, a condition that causes swallowing problems after a brain stem injury. The trial was designed to compare the injections against st…

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

This phase 2 trial tests whether adding a second DNA vaccine to a standard immunotherapy (pembrolizumab) can improve outcomes for men with castration-resistant metastatic prostate cancer. About 60 participants will receive either one or two DNA vaccines along with pembrolizumab f…

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

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

This study compares a targeted drug called selumetinib to standard chemotherapy (carboplatin/vincristine) in children aged 2 to 21 with neurofibromatosis type 1 (NF1) and low-grade glioma, a type of brain tumor. The goal is to see if selumetinib works as well or better at control…

This trial tests a new drug called Cadonilimab in 28 people with advanced colorectal cancer that has not responded to standard immunotherapy. The drug is a bispecific antibody that targets two immune checkpoints (PD-1 and CTLA-4) to potentially reactivate the immune system agains…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This Phase 3 trial tests whether the oral drug remibrutinib works better than teriflunomide at reducing relapses and slowing disability in people with relapsing multiple sclerosis. About 1,000 adults aged 18–55 with active MS are taking part. The study is double-blind, meaning ne…

This study tests a one-time gene therapy called CAP-003 in adults with Parkinson's disease caused by a GBA1 gene mutation. The goal is to see if it is safe and if it can help control the disease. Participants receive a single IV infusion and are monitored for two years.

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

This study tests a single injection of ZVS101e, a gene therapy that delivers a working copy of the CYP4V2 gene directly into the retina. It is for adults with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The trial will check wh…

This early-stage trial tests whether a single infusion of donated mesenchymal stem cells is safe for children with cerebral adrenoleukodystrophy (cALD), a rare and life-threatening brain disease. The stem cells are given as a temporary bridge to buy time before a more definitive …

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

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

This phase 2 trial is testing a combination of two immunotherapy drugs, nivolumab and ipilimumab, in 798 people with over 50 types of rare cancers. The goal is to see if these drugs can shrink tumors by helping the immune system attack cancer cells. Participants receive the drugs…

This early-phase trial tests a gene therapy called MVX-220 in 12 children and adults with Angelman syndrome. The therapy delivers a working copy of the UBE3A gene via a single injection into the fluid around the brain. Researchers are primarily checking safety, but also looking f…

This study tests a single injection of ZVS101e gene therapy for people with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The therapy delivers a working copy of the CYP4V2 gene directly to the retina. Researchers will measure wh…

This program allows eligible patients to receive RGX-202, a gene therapy, on a single-patient basis outside of a clinical trial. It is designed for those with serious conditions who have no other treatment options. Currently, the program is temporarily not available.

This study provides ZVS101e, a gene therapy, to 32 people with Bietti crystalline corneoretinal dystrophy (BCD) who were in the control group of a previous Phase III trial. The therapy uses a harmless virus to deliver a working gene to the eye, aiming to slow vision loss. The mai…

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

This clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, w…

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

This study tests whether a low dose of lithium (20 mg/day) can slow disease-related changes in the brain and blood of people with early-stage Parkinson's disease. Twenty participants will receive either lithium or a placebo for a period, and researchers will use MRI scans and blo…

This early-stage study tests a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with advanced or recurrent solid tumors that have specific genetic changes. The main goal is to find the safest dose with the fewest side effects. The…

This early study at Mayo Clinic tests whether ultra-low, anti-inflammatory doses of radiation can slow Parkinson's disease. Twenty people with early-stage Parkinson's will receive five daily radiation treatments guided by special brain scans. The main goal is to see if the treatm…

This study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check saf…

This early-stage trial is testing a drug called NS-050/NCNP-03 in 20 boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The drug is designed to skip a faulty section of the dystrophin gene, allowing the body to produce a shorter but still functional…

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

This study was designed to test two immunotherapy drugs, nivolumab and ipilimumab, in children and young adults with cancers that returned or didn't respond to treatment and had many genetic mutations. The goal was to see if the drug combination was safe and could shrink tumors. …

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

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

This early study tests a new treatment called UX-DA001 for people with Parkinson's disease. The treatment uses the patient's own cells, which are turned into dopamine-producing cells and implanted into the brain during surgery. The main goal is to check safety, but researchers wi…

This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whe…

This early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly in…

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

This study looks at how safe and effective the drug everolimus is for Taiwanese adults with tuberous sclerosis complex (TSC) who have kidney tumors called angiomyolipomas. Four participants will take the drug and be monitored for side effects and tumor shrinkage. The goal is to c…

This study follows 75 Japanese babies with retinopathy of prematurity (ROP) who are being treated with Eylea, a drug injected into the eye. ROP causes abnormal blood vessel growth in the retina and can lead to blindness. The study collects real-world data on side effects and how …

This trial tests a personalized treatment plan for people with advanced colorectal cancer that has spread to the liver and stopped responding to standard treatments. The approach combines a liver-directed chemotherapy (HAIC), a targeted therapy (fruquintinib or cetuximab), and an…

This study tests a drug called ACER-001 (sodium phenylbutyrate) in a single patient with combined D,L-2-hydroxyglutaric aciduria, a rare genetic disorder that causes brain damage and seizures. The goal is to see if the drug is safe and can lower harmful acid levels in the body. R…

This study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …

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

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

This study tests whether adding the immunotherapy drug pembrolizumab to standard chemotherapy (with or without radiation) after surgery can help prevent high-risk endometrial cancer from coming back. About 990 participants who had surgery to remove their uterus and ovaries will r…

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

This study is a follow-up to a Phase 3 trial testing a gene therapy called AAV5-hRKp.RPGR for people with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. The treatment aims to slow or stop vision decline by delivering a working copy of the RPGR gene …

This study tests whether the drug tafamidis can safely stop the progression of ATTR amyloidosis in people who have already received a heart transplant. About 25 stable patients will take tafamidis daily for 12 months. Researchers will measure changes in blood protein levels, nerv…

This study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and …

This study tests a personalized deep brain stimulation (DBS) approach for people with severe obsessive-compulsive disorder (OCD) that hasn't improved with standard treatments. Ten adults will first undergo brain mapping to find the best stimulation targets, then receive a DBS dev…

This study looks at a rare disease called KSHV-associated multicentric Castleman's disease (MCD), caused by a virus. Researchers are testing several drug combinations, including chemotherapy and antivirals, to see if they can control the disease. The study also tracks how the dis…

This Phase 3 study tests a gene therapy called AAV5-hRKp.RPGR in 4 Japanese people with X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The therapy is injected into the eye to deliver a working copy of the RPGR gene. The main goal is to che…

This study tests new drugs, alone or combined with pembrolizumab, in people with extensive-stage small cell lung cancer that has stopped responding to initial therapy. About 110 participants will receive experimental treatments to see if they are safe and can shrink tumors. The g…

This early-stage trial is testing a new drug called IDE161, alone or with the immunotherapy pembrolizumab, in adults with advanced solid tumors (like breast, ovarian, prostate, or lung cancer). The main goals are to check safety, find the right dose, and get a first look at wheth…

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

This Phase 2 trial tests two experimental drug combinations in 63 women with a specific type of ovarian cancer (BRCA-mutant or HRD-positive) that has worsened after prior PARP inhibitor treatment. Participants receive either tuvusertib plus niraparib or tuvusertib plus larteserti…

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

This study tests whether intravenous immunoglobulin (IVIG) can reduce symptoms of autonomic dysfunction—such as dizziness, rapid heartbeat, and fatigue—in people with long COVID. Two hundred participants will receive either IVIG or a placebo (saline) for 9 months, plus coordinate…

This phase 2 study tests a personalized immune cell therapy called tumor-infiltrating lymphocytes (TIL) in 20 people with advanced solid cancers (like stomach, colon, or pancreatic cancer) that have not responded to standard treatments. Patients first receive chemotherapy to prep…

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

This trial will test whether deep brain stimulation (DBS) surgery improves movement, balance, and daily function in 40 people with Parkinson's disease. Participants will have their walking speed, balance, muscle strength, and activity levels measured before and after surgery. The…

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

This study tests an oral drug called SKY-0515 in 500 adults with Huntington's disease who have already completed a prior study of the same drug. The goal is to see if the drug can safely lower harmful proteins and improve symptoms over the long term. Participants must be 25 or ol…

This phase 3 trial tests a single injection of 4D-150 gene therapy in 480 adults with wet age-related macular degeneration (AMD). The goal is to see if one treatment can control the disease as well as or better than standard aflibercept injections given every few weeks. Participa…

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

This early-stage study tests a new long-acting injection called HNC364, which turns into the Parkinson's drug rasagiline in the body. Researchers want to see how much of the drug gets into the bloodstream compared to taking rasagiline pills daily. The study involves 30 healthy ad…

This study looks at how safe and tolerable the drug pegcetacoplan (Syfovre) is for people with geographic atrophy, a form of advanced age-related macular degeneration that causes vision loss. About 300 patients who are already prescribed the drug by their doctor will be followed …

This early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …

This early-stage study tests the safety of acamprosate, a drug used for alcohol dependence, in 30 adults with ALS who have a specific mutation in the C9orf72 gene. Participants take the pill three times daily for 24 weeks and undergo regular checkups, breathing tests, and spinal …

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

This Phase 3 trial tests remibrutinib, an oral tablet, against teriflunomide in 1000 adults with relapsing multiple sclerosis. The goal is to see if remibrutinib better reduces yearly relapse rates and delays disability. Participants will then have the option to continue remibrut…

This study was designed to test whether the drug govorestat could improve symptoms of Charcot-Marie-Tooth disease caused by sorbitol dehydrogenase deficiency (CMT-SORD), a rare nerve condition. It planned to enroll people aged 16 to 65 and compare govorestat to a placebo over 36 …

This study tests whether a two-drug combination can help the immune system attack ovarian cancer that has returned after standard treatments. The drugs are E7777, which removes certain immune cells that block the body's defenses, and pembrolizumab, which reactivates cancer-fighti…

This early-phase trial tests a single injection of AAV-MECP2 gene therapy into the spinal fluid of 8 girls aged 4-10 with Rett syndrome, a severe neurodevelopmental disorder. The goal is to see if the treatment is safe and can reduce disease severity. The study is not yet recruit…

This early-stage study tests a new drug called EYE201, given as three eye injections over 12 weeks, to see if it is safe and can help reduce swelling in the back of the eye. It includes people with three different eye conditions that cause vision loss: branch retinal vein occlusi…

This study tests a new drug, Tuvusertib, combined with an immunotherapy called Cemiplimab in people with a specific type of advanced lung cancer (non-squamous non-small cell) that has worsened after prior treatments. The trial aims to see if the combination can shrink tumors and …

This study tests a drug called trametinib in children with a rare blood cancer (juvenile myelomonocytic leukemia) that has returned or not responded to treatment. The drug works by blocking certain enzymes that help cancer cells grow. The goal is to see if it can shrink or contro…

This phase 3 trial tests whether adding pembrolizumab (an immunotherapy) and olaparib (a targeted drug) to standard chemotherapy and radiation can help people with limited-stage small cell lung cancer live longer. About 672 participants will receive either the drug combo or a pla…

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

This study tests an experimental drug called BIIB122 in 50 people who have a specific genetic form of Parkinson's disease (LRRK2-PD). The main goal is to check the drug's safety and how it affects certain markers in the blood and urine over 12 weeks. Participants are randomly ass…

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

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

This phase 2 trial tests an experimental drug called tominersen in 301 people with early-stage Huntington's disease. The drug aims to lower levels of the harmful mutant protein that causes the disease. Researchers will compare tominersen against a placebo to see if it can slow do…

This study compares the immunotherapy drug pembrolizumab with standard chemotherapy in about 100 Chinese patients whose advanced colorectal cancer has a specific genetic feature (MSI-H or dMMR). The goal is to see if pembrolizumab can slow cancer growth or improve survival better…

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

This pilot study aimed to test whether a mixture of dexamethasone, hyaluronic acid, and a green dye could be safely injected into the ear to treat certain types of hearing loss that don't improve with standard treatments. The plan was to enroll 26 adults with sudden hearing loss,…

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

This study looks at whether taking selumetinib with a low-fat meal reduces stomach problems in teenagers with neurofibromatosis type 1 (NF1) who have tumors that cannot be removed by surgery. About 24 teens will take the drug under both fed and fasted conditions to compare drug l…

This early-stage trial tests a new oral drug called OCT-980 for people with a specific inherited eye disease (autosomal dominant retinitis pigmentosa) that causes gradual vision loss and blindness. The study first gives a single dose to healthy volunteers to check safety, then mu…

This early-phase trial tests an experimental drug called nL-FLVC-001 in a single person with posterior column ataxia with retinitis pigmentosa (PCARP), a rare genetic condition that causes vision loss and coordination problems. The drug is an antisense oligonucleotide injected in…

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

This study follows about 200 people with Prader-Willi syndrome who are taking or starting VYKAT XR. Researchers will track side effects and how the drug affects their health over time. The goal is to gather more safety information, not to test if the drug cures the condition.

This phase 2 trial tests a daily oral drug called sevasemten (EDG-5506) in 244 adults and adolescents with Becker muscular dystrophy, a condition that causes progressive muscle weakness. The study compares the drug to a placebo to see if it can reduce muscle damage, improve walki…

This clinical trial is testing several new combinations of drugs for people with advanced kidney cancer that has spread. The study includes about 400 participants who have not had any prior treatment for their advanced disease. The goal is to see if these combinations are safe an…

This study planned to test a small telescope implant inside the eye for people with severe vision loss from advanced age-related macular degeneration (AMD). The device was meant to improve central vision by magnifying images onto healthy parts of the retina. However, the trial wa…

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

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

This early-stage trial is testing the safety and potential benefit of pembrolizumab, an immunotherapy drug, in 71 children whose brain tumors (like DIPG, high-grade gliomas, or medulloblastoma) have come back or not responded to standard treatments. The drug works by helping the …

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

This early-stage study tests a gene therapy called AB-1001 in 5 adults with early Huntington's disease. The treatment is injected directly into the brain to potentially slow the disease. The main goal is to check safety, but researchers will also look at brain changes on MRI and …

This phase 3 trial tests whether adding belzutifan to standard pembrolizumab after kidney cancer surgery can delay or prevent the cancer from coming back. About 1800 participants with clear cell renal cell carcinoma are randomly assigned to receive either belzutifan plus pembroli…

This study tests a drug called selumetinib in 32 Chinese children and adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be surgically removed. The goal is to check the drug's safety and how well it works at shrinking these tumors. Participants take the …

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

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

This study tests whether a cochlear implant can safely and effectively restore hearing in people who become deaf in one ear after surgery to remove a benign tumor (vestibular schwannoma) or to treat Meniere's disease. Fifteen participants will receive the implant during their sur…

This phase 3 trial tests whether adding the immunotherapy drug pembrolizumab (Keytruda) to standard chemoradiotherapy helps people with esophageal cancer live longer and delay recurrence. About 700 participants who cannot have surgery will receive either pembrolizumab or a placeb…

This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrol…

This study tests whether adding the drugs pembrolizumab and olaparib to standard chemoradiation helps people with stage III non-small cell lung cancer live longer without their cancer growing. About 870 participants will receive one of three treatments: pembrolizumab with chemora…

This study tests a drug called selumetinib in children and young adults with low grade glioma that has come back or not responded to other treatments. The drug works by blocking certain enzymes that help tumor cells grow. The goal is to find the best dose and see if it can shrink…

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

This study follows 250 adults with advanced Parkinson's disease who are prescribed ABBV-951 by their doctors. The goal is to see how safe and effective the drug is in everyday use over 52 weeks. Participants continue their normal care with no extra visits required.

This study tests a custom-made drug for one child with a rare, severe brain disease called CONDBA, caused by a specific gene mutation. The drug aims to slow or stop brain damage by targeting the faulty gene. Researchers will track changes in movement, coordination, and quality of…

This study tests whether adding olaparib to the standard chemotherapy temozolomide helps control advanced pheochromocytoma or paraganglioma—rare neuroendocrine tumors that have spread or can't be removed surgically. About 46 adults with these cancers will be randomly assigned to …

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

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

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

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

This phase 3 trial tests whether adding the immunotherapy drug pembrolizumab before and after surgery can help people with advanced head and neck cancer stay cancer-free longer. About 714 participants receive either pembrolizumab plus standard radiation (with or without chemo) or…

This study tests whether a brain shunt—a device that drains extra fluid—can improve walking and thinking in older adults with idiopathic normal pressure hydrocephalus (iNPH). One hundred participants will either receive an active shunt or a placebo procedure. The main goal is to …

This early-stage study is testing whether combining standard psychiatric medications (like antidepressants and anti-anxiety drugs) with individual cognitive behavioral therapy can improve symptoms and daily functioning in people with anxiety, depression, PTSD, OCD, bipolar disord…

This early-stage study tests the safety of an experimental drug called RO7669330, given as an injection into the eye, for people with geographic atrophy (GA) due to age-related macular degeneration (AMD). The study involves 27 participants and will monitor side effects and eye he…

This phase 3 trial tests whether a refillable eye implant delivering ranibizumab can be safely used every 36 weeks instead of every 24 weeks for people with wet age-related macular degeneration. About 451 participants who have already responded to standard injections will receive…

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

This Phase 3 trial compares the immunotherapy drug pembrolizumab (Keytruda) to standard chemotherapy in 280 women with advanced or recurrent endometrial cancer that has a specific genetic feature (dMMR). The goal is to see if pembrolizumab can better delay cancer growth and impro…

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

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

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

This study tests a device that delivers specific light and sound patterns to the brain, aiming to slow the progression of mild-to-moderate Alzheimer's disease. About 670 participants will use the device at home for an hour daily over a year, with half receiving a sham (inactive) …

This pilot study is testing whether adults with serious mental health issues like psychosis, anxiety, or depression can receive hospital-level care in their own homes. Researchers will see how many people are eligible and willing to try this approach. The goal is to offer a safe,…

This study tests a procedure called focused ultrasound (Exablate) that uses sound waves to target a small area in the brain involved in Parkinson's disease. It involves 67 people with early-stage Parkinson's who will either receive the ultrasound treatment or continue with standa…

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

This study tests if adding another drug (belzutifan or quavonlimab) to a standard two-drug treatment can help people with advanced clear cell kidney cancer live longer or stop their cancer from growing. About 1,650 adults who have not had prior treatment for advanced disease will…

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

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

This study is testing experimental combinations of drugs for people with advanced kidney cancer (clear cell type) that has gotten worse after initial treatment. The goal is to see if these combinations are safe and can shrink tumors. About 370 participants will be enrolled in thi…

This early-stage trial tests an experimental drug called OCU-10-C-110, given as an eye injection, in 24 people with wet age-related macular degeneration (nAMD). The main goal is to check safety and tolerability at different doses. Researchers will monitor for side effects over ti…

This study tests whether adding the immunotherapy drug pembrolizumab to standard BCG treatment helps people with high-risk non-muscle invasive bladder cancer stay cancer-free longer. About 1,400 participants will receive either BCG alone or BCG plus pembrolizumab. The goal is to …

This early-phase study tests a new type of deep brain stimulation that adapts in real time to a person's movements, aiming to improve walking and motor skills in Parkinson's disease. Ten patients already scheduled for standard DBS surgery will receive this adaptive stimulation an…

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

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

This study tests whether injecting a replacement enzyme directly into the eye can slow or stop vision loss in children with CLN2 Batten disease, a rare genetic disorder that causes blindness and brain damage. Five children aged 2 to 6 who already receive brain infusions of the en…

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

This study tests a new way to use deep brain stimulation (DBS) for Parkinson's disease. Instead of constant stimulation, the device automatically switches settings when the person starts walking, aiming to improve gait and balance. Six people who already have a special DBS implan…

This study tests a stem cell therapy called OpRegen for people with advanced dry age-related macular degeneration (geographic atrophy). Researchers inject retinal pigment epithelium cells derived from human embryonic stem cells into the eye to see if they can safely replace damag…

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

This study follows 200 children in Japan with growth hormone deficiency who are taking Sogroya® (somapacitan) as part of their normal care. Researchers will monitor side effects and growth over 1 to 3 years to see how safe and effective the drug is in real-world use. Participants…

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

This expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed…

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

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

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

This study tests whether leucovorin calcium, a vitamin-like supplement, can improve language and other symptoms in young children with autism spectrum disorder. Researchers will enroll 80 children aged 2.5 to 5 years with language delays. The trial lasts about 26 weeks and compar…

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

This phase 2 trial tests the drug trametinib in children and young adults (ages 1 month to 25 years) with low-grade glioma or plexiform neurofibroma that has not responded to prior treatment. Participants take a daily oral dose for up to 18 cycles. The study aims to see if the dr…

This phase 2a trial tests whether the drug TB006 can safely improve motor function in people with early-stage Parkinson's disease. About 62 participants will receive either TB006 or a placebo by IV infusion. The study is double-blind, meaning neither patients nor doctors know who…

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

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

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

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

This study looks at the long-term safety and effects of a drug called SAT-3247 in 10 people with Duchenne muscular dystrophy who were in a previous trial. Participants take the drug by mouth five days a week for about 11 months. Researchers will check for side effects and measure…

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

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

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

This Phase 3 study tests an experimental drug called CVN424 in 330 people with Parkinson's disease who experience motor complications. Participants take either 75 mg, 150 mg, or a placebo pill once daily for 12 weeks. The main goal is to see if CVN424 can reduce the daily hours o…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

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

This study uses special brain scans (PET imaging) to measure amyloid buildup—a protein linked to Alzheimer's—in healthy older adults, people with mild memory problems, and those with dementia. Participants receive scans every 2-3 years for up to 20 years to see how amyloid change…

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

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

This study tests whether a new type of PET/CT scan, using a radioactive tracer called 68Ga-DOTATOC, can find neuroendocrine tumors (NETs) and similar tumors more safely and accurately than current imaging methods. About 800 people with known or suspected NETs will receive the sca…

This study is developing a web-based app that analyzes how you speak and remember things to quickly detect early cognitive problems like dementia or Alzheimer's. Researchers will enroll 500 people (healthy adults over 45 and those already diagnosed with mild cognitive impairment …

Li-Fraumeni syndrome is a rare genetic condition that greatly increases the risk of developing many types of cancer. This pilot study will test three advanced MRI techniques—whole body STIR MRI, DW-MRI, and PET-MRI—in 30 people with the condition to see how well they can detect v…

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

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

This study tests a special bed called SuPerSense that senses how a person lies on it and gives feedback to help improve posture and balance. People with Parkinson's disease who have trouble with balance or posture will either do standard rehab exercises or use the smart bed plus …

This study looks at whether pramipexole, a drug used for restless leg syndrome, can ease leg symptoms in women with X-linked adrenoleukodystrophy (ALD). About 24 women will take the drug or a placebo for two months. The goal is to see if it improves sleep, walking, and quality of…

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

This study tests whether smelling essential oils during skin surgery can reduce the unpleasant smell of burnt flesh from electrocautery and lower patient anxiety. About 111 adults having same-day skin procedures will be randomly assigned to receive either a real essential oil inh…

This study explores whether a new type of group therapy, based on Eastern cultural practices, can help people with anxiety disorders. Researchers will enroll 60 participants aged 15 to 55 to test the therapy's effects on anxiety symptoms. The goal is to see if this approach is bo…

This study investigates whether whole-body vibration (WBV) therapy, added to standard exercise, can improve balance, walking, thinking, and the ability to do two things at once in people with Parkinson's disease. Participants will be randomly assigned to receive either low-freque…

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

This study tests whether a smartphone app called AIM+ can help juvenile justice officers and other professionals who work with youth in the legal system manage chronic stress, anxiety, and burnout. Over 30 days, participants use the app for short guided meditations and videos. Th…

This phase 2 trial tests whether fasudil, a drug already used in Japan for blood vessel spasms, is safe and can help symptoms in people with early Parkinson's disease. 75 participants will take either fasudil or a placebo for 3 weeks. The study focuses on safety and tolerability,…

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

This study looks at two ways to lower anxiety in children aged 3 to 10 who need surgical pins removed from an elbow fracture. One approach removes the pins right after the cast comes off, before an X-ray, instead of after. The other uses a virtual reality headset during the pin r…

This study looks at whether a peer support program is acceptable and helpful for people with mild depression or anxiety who are starting or receiving hormone care. One hundred participants are randomly assigned to either the peer support group or usual care. The program includes …

This study tests whether a simple ear acupressure technique, called auricular point sticking therapy, can reduce anxiety and improve sleep in women undergoing IVF. 178 women aged 20-45 with anxiety or insomnia will either receive standard IVF care or standard care plus ear acupre…

This study is testing whether a 6-month program of online exercise classes, guided meditation, and nutrition advice can improve thinking, sleep, and overall health in people with Parkinson's disease. 60 participants aged 40 to 85 will take part, with some starting the program rig…

This study looks at the long-term safety of valbenazine for people with Huntington disease who have involuntary jerking movements (chorea). It includes 7 participants from Canada who were in a previous valbenazine study. Everyone gets the drug, and researchers track side effects …

This study tests whether a safe, painless brain stimulation technique called tDCS can improve verbal learning in people with Alzheimer's disease. Researchers will compare active stimulation to a sham (fake) version in 90 participants with typical or language-based Alzheimer's. Th…

This study tests a single dose of an experimental drug called REGN7544 in 82 people with Postural Orthostatic Tachycardia Syndrome (POTS). The goal is to see if the drug safely reduces the rapid heart rate that happens when standing up. Researchers will also check for side effect…

This study looks at whether auto-captioning glasses are easy and helpful for adults with NF2-related schwannomatosis who have hearing loss. Eighteen participants will use the glasses for 12 weeks and report their experience through surveys and interviews. The goal is to see if th…

This study tests whether adding cognitive training to balance and gait exercises helps people with Parkinson's disease improve their balance, walking, and thinking. Fourteen participants will undergo 12 sessions of combined training. The goal is to see if this approach eases move…

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

This study aimed to see if a web-based self-help program called Deprexis could help orthopedic patients with major depressive disorder. The plan was to offer the 8-week online program to patients with depression and see if they would use it and if their mood and physical function…

This study compares two different remote support programs to see which one helps women feel better after surgery for suspected gynecological cancer. About 160 women will take part, and researchers will measure pain, mood, and sleep quality. The goal is to find simple, at-home way…

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

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

This study tests a specially adapted stress management program called 3RP-VHL for adults with von Hippel-Lindau disease (VHL), a genetic condition that causes tumors. The program teaches relaxation and resilience skills through 8 video sessions. Researchers want to see if it is f…

This study tests whether tolcapone, a drug used for Parkinson's disease, can reduce symptoms of obsessive-compulsive disorder (OCD) in adults. About 49 people with moderate to severe OCD will receive either tolcapone or a placebo. The main goal is to see if the drug safely lowers…

This study tests whether a new type of group therapy, called Group Cognitive Evolutionary Therapy, can help reduce symptoms of depression and anxiety. Sixty-four adults with mild to severe depression will either receive two full days of therapy or be placed on a waiting list. Res…

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

This study tests whether group psychotherapy can help parents of children with Leber congenital amaurosis (LCA) feel better emotionally and handle stress. Forty parents will take part, with half joining therapy groups and the other half serving as a comparison. Researchers will m…

This study tests a single online session that teaches a simple coping skill to youth ages 9-17 who are on a waitlist for therapy. The goal is to see if this quick digital tool can help ease symptoms of anxiety, depression, and stress while they wait. About 226 youth and their par…

This study tests whether combining music-based movement cues (patterned sensory enhancement) with mild brain stimulation can improve arm and hand function in people with corticobasal syndrome, a rare brain disorder. Twenty participants will be split into two groups: one receiving…

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

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

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

This study tests whether a community-based music mindfulness program can reduce anxiety and depression in children aged 8-12 with ADHD or anxiety. Participants attend bi-weekly sessions for 8 weeks, wear fitness trackers, and complete surveys. The goal is to see if this approach …

This study looks at whether adding resistance (strength) exercises to standard aerobic and balance training can improve quality of life, daily activities, thinking skills, and muscle health in people with early-stage Parkinson's disease. About 34 adults aged 40-70 with mild to mo…

This study tests whether a 6-week, caregiver-assisted breathing program done at home with remote coaching is practical and helpful for children with Duchenne muscular dystrophy. Twelve children will do breathing exercises, airway clearance, and relaxation techniques at least 5 ti…

This study tests whether monthly alexandrite laser treatments can safely shrink or improve the look of skin tumors in adults with neurofibromatosis type 1 (NF1). Ten participants will receive up to six monthly laser sessions, with some areas treated with cooling and others withou…

This early-stage study is testing whether repeated doses of psilocybin, the active ingredient in magic mushrooms, can reduce symptoms of obsessive-compulsive disorder (OCD). Twenty-eight adults with moderate-to-severe OCD who have not responded to standard treatments will receive…

This study tests a remote speech therapy program for 32 Korean-speaking people with Parkinson's disease and their families. The therapy includes 16 sessions over four weeks, plus daily home practice, to improve speech clarity. The goal is to see if this culturally adapted approac…

This study tests whether a 'social prescription' program can reduce loneliness in people with Parkinson's disease who already attend group exercise classes. About 40 participants who feel lonely will work with an occupational therapist to choose from 11 social activities, like sp…

This study tests an 8-week program called Step-CBT that combines physical activity goals with cognitive behavioral therapy for veterans with COPD who also have depression or anxiety. The program is delivered via video calls to make it accessible. Researchers want to see if it is …

This study tests whether adding motor imagery—mentally practicing movements without actually moving—to standard physical therapy can improve balance and reduce fear of movement in people with Parkinson's disease. Forty-two participants will be randomly assigned to receive either …

This study tests whether taking a special olive oil (high in a compound called oleocanthal) along with curcumin (the active part of turmeric) is safe and might help shrink skin tumors in adults with neurofibromatosis type 1 (NF1). About 23 participants take the supplements twice …

This study tests a therapy called Dynamic Temporal and Tactile Cuing (DTTC) to help children with childhood apraxia of speech, a disorder that makes it hard to plan and coordinate speech movements. The trial involves 72 children and measures how well they can say treated and new …

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

This study tests whether a device that sends mild electrical pulses to facial muscles can help treat major depression. Twenty adults with depression will use the device at home for 45 minutes daily over four weeks. The main goal is to see if the approach is feasible and safe, wit…

This study tests two medications, dexmedetomidine and tramadol, given before hemorrhoid surgery to see which one better reduces anxiety and pain. Ninety adults will receive one of the drugs or a placebo before spinal anesthesia. Researchers will measure anxiety levels and pain sc…

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

This study tests a new way to match dementia caregivers with each other for emotional support using a computer algorithm. Researchers want to see if matching based on personal preferences leads to better satisfaction and less stress than random matching. About 355 caregivers of p…

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

This study tests whether a single injection of ketamine can quickly reduce suicidal thoughts in people with depression who come to the emergency room. About 90 participants will receive either ketamine or a sedative (midazolam) and be monitored for 24 hours. The trial is currentl…

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

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

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

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

This study looks at whether low-dose rapamycin can reduce symptoms and improve quality of life in people with ME/CFS or Long-COVID. About 150 participants will take the drug as prescribed by their doctor and complete surveys and blood tests over time. The goal is to see if the dr…

This pilot study tests whether an 8-week mindfulness program (MBSR) can reduce anxiety, depression, and stress in people with COPD who are already in pulmonary rehab. About 44 participants will be followed for 12 months. The goal is to see if this mind-body approach improves emot…

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

This study looks at whether changing the sound of your own voice (like adding echo or whisper) can help adults who stutter speak more smoothly. About 100 people will use a special app for one month. The goal is to see if this simple technique improves fluency over time.

This study tests whether an 8-week aquatic high-intensity interval training (HIIT) program can improve balance, physical function, and muscle health in people with Parkinson's disease. Fifty-six participants with mild to moderate Parkinson's will do Tabata-style exercises in a po…

This study tests whether an online cognitive behavior therapy (CBT) program can help children aged 8 to 17 with asthma who also feel anxious or worried about their condition. The therapy is designed to improve quality of life, reduce anxiety, and help manage asthma better. The st…

This study tests a mindfulness program (called GEM) delivered through an app and weekly group video calls. It aims to see if young adults (ages 18-24) who have faced early life hardships find the program helpful and easy to use. The study also checks if the program reduces sympto…

This study tests whether adding balance training, education, and vestibular rehab to the standard Epley maneuver helps people with benign paroxysmal positional vertigo (BPPV) more than the maneuver alone. Forty-six adults with BPPV will receive either the combined approach or sta…

This study tests an online cognitive behavioral therapy (CBT) program for university students with mild depression or anxiety. Two hundred students who previously took a mental health survey will receive a tailored internet treatment. The goal is to see if this e-health approach …

This study tests whether a gentle, low-level electric current applied to specific brain areas can improve speech and language difficulties in people with primary progressive aphasia (PPA) and progressive apraxia of speech (PAOS). The treatment is called high-definition transcrani…

This study tests whether listening to specific sound frequencies for just 9 minutes a week can improve mental health. 110 adults with mild anxiety, stress, or depression will either sit in a special sound chamber or use headphones. The goal is to see if this simple, drug-free app…

This study tests whether using a virtual reality headset during PICC line placement can reduce anxiety and pain in cancer patients. About 120 adults will be randomly assigned to either receive VR plus standard care or standard care alone. The goal is to see if this non-drug appro…

This study tests whether a non-invasive magnetic brain stimulation technique, called rTMS, can help ease depression in people with Parkinson's disease. Fifty adults with mild to moderate depression will be randomly assigned to receive either real rTMS or a sham (fake) treatment f…

This study looks at whether cancer patients can use a self-guided meditation program at home to reduce anxiety and depression. About 35 patients will try techniques like deep breathing or gentle movement for two weeks. The goal is to see if this simple, low-cost approach is pract…

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

This study tests whether a non-invasive device that gently stimulates the vagus nerve through the ear can improve speech recovery in people with aphasia after stroke or brain injury. Twelve adults with apraxia of speech will receive either active or sham stimulation during 16 wee…

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

This study tests a biodegradable tube that is placed over a cut nerve during a leg biopsy. The goal is to see if it is safe and reduces common side effects like pain, redness, and swelling. Twenty adults with various nerve conditions are taking part, and half will get the tube wh…

This study planned to test whether a lidocaine nerve block (SPG block) could reduce anxiety in adults at electronic dance music festivals, compared to a saline placebo. The trial was withdrawn before enrolling any participants, so no data were collected. The approach was experime…

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

This study tests a brief digital mental health program for people on waiting lists for psychological treatment. About 80 participants will be randomly assigned to one of four groups: a short program (30-40 minutes), a longer version, or control conditions. The main goal is to see…

This study tests whether an immersive virtual reality program can improve balance in people with Parkinson's disease. Fifty participants in Spain will either receive the VR program or standard physical therapy. The goal is to see if VR can help with balance and reduce fall risk.

This study tests whether adaptive deep brain stimulation (DBS) can reduce or prevent freezing of gait in 20 people with Parkinson's disease. Participants already have DBS implants, and the device will adjust stimulation in real time based on brain signals. Freezing episodes will …

This study compares a 12-week tele-rehabilitation program using mindful and conscious movement (like adapted yoga and breathing exercises) to conventional low-intensity exercise and usual care for people with chronic fatigue syndrome or post-COVID syndrome. The goal is to see if …

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

This study tests whether combining a mild electrical brain stimulation (tDCS) with virtual reality meditation can help ease anxiety. 52 adults with generalized anxiety disorder or related conditions will receive 12 sessions over two weeks. Researchers will measure anxiety levels …

This study tests a drug called bezisterim (NE3107) in 60 people with early Parkinson's disease who have not yet started standard treatment. The goal is to see if it can improve movement symptoms like stiffness and slowness. Participants take the drug or a placebo twice daily for …

This study tests special gloves that vibrate in a pattern to help people with moderate Parkinson's disease move more easily. Thirty participants wear the gloves at home for months. Researchers measure changes in movement scores to see if the vibration therapy works.

This study tests a wearable device that delivers gentle vibrations to the skin to improve motor function in people with Parkinson's disease. The treatment can be used alongside regular medication and aims to delay the need for higher doses. Thirty participants with moderate-stage…

This study looks at whether using a mindfulness app on a smartphone for 8 weeks can improve mental health in Korean immigrants aged 60 and older. Participants use the app for about 10-15 minutes daily and have weekly check-ins. The goal is to see if the app is easy to use and hel…

This study aimed to see if having a parent appear on a video call during anesthesia induction could reduce children's anxiety as effectively as the sedative midazolam. It was designed for children aged 4 to 8 undergoing outpatient surgery. However, the study was withdrawn before …

This study tests a device called NervioX-2400 that uses weak electrical currents to stimulate specific brain areas without surgery. Researchers want to see if it can improve movement problems like tremors and stiffness in 100 people with early-to-mid stage Parkinson's disease. Ea…

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

This study tests a 12-week online program for mothers of infants (12-36 months old) with SMA Type 1. The program aims to teach mothers how to support their baby's development and improve their own knowledge. Thirteen mothers will take part, with 10 receiving the program and 3 rec…

This early-stage trial tests whether monthly injections of Kybella (a drug that dissolves fat) can safely shrink skin tumors in people with Neurofibromatosis Type 1. Up to 15 adults will receive up to 6 monthly treatments, and researchers will compare treated and untreated tumors…

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

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

This study tests if a therapy called FIRST, which uses five key principles to help youth manage negative emotions, works better than standard therapy. About 212 youth aged 7 to 15 with anxiety, depression, trauma, or behavior problems will take part. The goal is to see if FIRST i…

This study tests a special needle with a tiny tracking device inside, like a mini GPS, to help doctors find tumors that are hard to see on CT scans. About 3,900 adults with certain cancers or growths will have their biopsy or ablation procedure done using this smart needle. The g…

This study aims to better understand fatigue by comparing people who have felt tired for more than a month with those who are not fatigued. Researchers will use questionnaires, physical tests, and optional brain scans to identify different types of fatigue. The goal is to gather …

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

This study follows 110 people who previously took part in the AREDS or AREDS2 trials to see how age-related macular degeneration (AMD) and cataracts change over time. Participants get a yearly eye exam, including vision tests and eye photos, and may give a blood sample. The goal …

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

This study investigates whether advanced eye imaging and artificial intelligence can predict proliferative vitreoretinopathy (PVR), a condition that can cause retinal detachment surgery to fail. Researchers will analyze scans from 100 patients with retinal detachment, some with P…

This study follows 25 people with spinocerebellar ataxia type 7 (SCA7) for up to 5 years to learn how the disease changes vision, coordination, and thinking. Participants will have yearly eye exams, brain scans, and neurological tests. No treatment is given; the goal is to gather…

This study follows 125 people with Parkinson's disease or related conditions over two years. Researchers use special brain scans to measure changes in a chemical called acetylcholine, which is linked to walking problems, falls, and thinking skills. The goal is to understand how t…

This study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement…

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

This observational study will follow 2,500 adults with vision loss from nAMD or DME who are already prescribed aflibercept 8 mg by their doctors. Researchers will collect data from routine visits over up to 24 months to see how vision changes. The goal is to understand how well t…

This study looked at how sounds played during sleep affect brain waves and memory in children with Rolandic epilepsy. Researchers used brain scans (MRI, MEG/EEG) to understand which brain circuits are involved. The goal was to find new treatment ideas for this type of childhood e…

This study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…

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

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

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

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

This study investigates how the brain's emotion network works by recording brain activity directly from the surface of the brain in people with epilepsy who are already undergoing surgery for seizure monitoring. Participants watch short video clips designed to trigger emotions li…

This study aims to improve how doctors test for autonomic failure, a condition where the body's automatic functions like blood pressure control don't work properly. Researchers will use advanced imaging, sweat tests, and skin biopsies in up to 89 adults with Parkinson's disease, …

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

This study looks at how adults at risk for Alzheimer's disease react after learning their amyloid and tau biomarker test results. Researchers will track changes in distress, stigma, quality of life, and future planning over time. The goal is to understand the psychological and be…

This study was designed to see if giving a short, mild electrical stimulation to compressed nerves during surgery could speed up recovery of strength and function. It planned to include people with nerve compression in the spine or limbs who were already scheduled for decompressi…

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

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

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

This study aims to find the genes that cause inherited hearing loss by analyzing DNA from 24,000 people in large Pakistani families. Researchers will compare affected and unaffected family members to identify new deafness genes. The goal is to improve genetic testing and counseli…

This study tracks a protein called neurofilament light chain in the blood of 342 people who carry genetic mutations linked to frontotemporal dementia (FTD). Participants provide blood samples every three months for three years, often from home. The goal is to see if this biomarke…

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

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

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

This study looks at X-linked juvenile retinoschisis (XLRS), a rare inherited eye disease that causes vision loss in young males. Researchers will examine 351 participants, including affected males and female carriers, using eye exams, retinal photos, and genetic tests. The goal i…

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

This study looks at whether taking short walks throughout the day is better for kids at risk for type 2 diabetes than one longer exercise session. Researchers will measure blood sugar, insulin, and thinking skills in 188 children aged 8-11 with overweight or obesity. The goal is …

This study follows 80 people aged 8 to 50 with Stargardt disease, a genetic eye condition that causes vision loss. Researchers will monitor how the disease changes over time using eye imaging. The goal is to better understand the disease and prepare for future clinical trials.

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

This study looks at people with ALS and similar diseases like frontotemporal dementia and hereditary spastic paraplegia. Researchers want to understand how genes affect the disease and find biological markers (biomarkers) to help develop future treatments. About 708 participants,…

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

This study enrolls people with ALS and related disorders, as well as healthy volunteers, to track biological markers (biomarkers) in urine, blood, and spinal fluid. The goal is to better understand how these markers change over time, which could help design more effective future …

This study looks at people and families who have a high risk of cancer to learn what genes and environmental factors might increase that risk. Over 5,000 participants are followed over time with questionnaires, medical records, and optional genetic testing. No treatment is given,…

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

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

This study tracks brain activity in 160 Parkinson's patients who have or will receive deep brain stimulation (DBS). Researchers record signals from the brain during memory tasks, with the stimulator on and off, to find patterns linked to cognitive side effects. The goal is to ide…

This study tests new radio frequency coils for MRI scanners to see if they can produce better images in children, including those with Duchenne muscular dystrophy. Researchers will check for side effects like heating and discomfort, and measure image quality. The goal is to impro…

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

This study tests a new type of brain implant that can both sense brain activity and deliver stimulation in real time. It involves 25 people with Parkinson's disease or dystonia who already need deep brain stimulation. The goal is to see if closed-loop stimulation (which adjusts a…

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

This study uses special brain scans (MRS) to measure two natural chemicals, GABA and glutathione, in people with Friedreich's Ataxia (FRDA). Researchers want to see if these chemicals can serve as reliable markers of the disease and how they change after taking the drug Omaveloxo…

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

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

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

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

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

This study collects information from about 889 people with inherited retinal diseases (IRDs) to understand how vision changes over time. Participants have their eyesight and visual field tested regularly. The goal is to learn more about the natural course of these rare eye condit…

This study tests a new imaging method to see if white blood cells enter the brain in people with multiple sclerosis, fibromyalgia, or chronic fatigue syndrome. Participants receive their own white blood cells tagged with a radioactive tracer, then undergo a brain PET/MRI scan. Th…

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

This study follows 300 Hispanic and Latino caregivers of relatives with memory problems or dementia. Participants complete daily surveys for three weeks at a time, plus follow-ups at six and twelve months, to track how their mood and stress change day-to-day. The goal is to ident…

This study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study tracks the exercise habits of 288 people with Parkinson's disease over one year using activity trackers and a phone app. Researchers want to see if different types of exercise advice—like standard care, written guidance from a neurologist, or physical therapy—help peop…

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

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

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

This study follows nearly 600 people with Smith-Magenis syndrome (SMS), a rare genetic condition, to track how their health, behavior, and development change over time. Researchers will perform detailed medical exams, genetic tests, and surveys to better understand the syndrome's…

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

This observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …

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

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

This study looks back at the medical records of 150 people diagnosed with Creutzfeldt-Jakob disease (CJD), a rare and fast-moving brain disorder. Researchers will examine symptoms, blood tests, spinal fluid, brain scans, and EEG results to find patterns that could help doctors di…

This study looks at how thinking, memory, and movement change in older adults with conditions like dementia or Parkinson's disease. Researchers will use tests, brain scans, and lab samples to find clues for earlier diagnosis and better care. About 260 people, including healthy vo…

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

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

This study follows 1,000 patients in China who are already receiving faricimab for diabetic macular edema, retinal vein occlusion, or wet age-related macular degeneration. Researchers will collect data from routine clinic visits over one year to see how well the drug works and ho…

This observational study examines how aflibercept 8 mg, an injection into the eye, is used in routine care for patients in Japan with age-related macular degeneration or diabetic macular edema. Researchers will analyze claims data from about 3000 patients to understand patient ch…

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

This study uses a special brain scan called PIB PET to look at different types of dementia in people with Parkinson's disease. The goal is to find out who might develop dementia and what causes it. Researchers will study 320 participants to better understand these brain changes.

This study collects movement data from 250 people with stroke, Parkinson's disease, or leg amputations using wearable sensors and video. The goal is to build an online database that researchers can use to study how people move and recover. No treatments are being tested—this is p…

This study was designed to see if a smartphone app called iBlink can measure how well the brain learns new movements in people with spinocerebellar ataxia, a rare and serious movement disorder. Researchers planned to compare aerobic exercise with balance training to see which bet…

This study looks at whether specific brain wave patterns on an EEG can predict how well the drug fenfluramine works for people with Lennox-Gastaut Syndrome (LGS), a severe form of epilepsy. Researchers will follow 20 children and adults aged 2 to 35 who are already taking fenflur…

This study was designed to test whether a technique called self-distancing—where children refer to themselves in the third person (e.g., 'Emily can touch the spider')—helps them approach feared objects more than using first-person self-talk ('I can touch the spider'). The trial p…

This study looks at whether having a licensed counselor available can improve the mental well-being of medical residents (doctors in training). The researchers will measure changes in well-being, depression, and anxiety. 21 internal medicine residents from the University of Nevad…

This study looks at why adults with cancer may fear taking opioid pain medication after surgery. Researchers will use questionnaires to measure barriers, fears, and medication adherence in 50 patients. The goal is to learn more about these concerns to improve pain management afte…

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

This study uses advanced MRI scans and genetic tests to track how neurodegenerative diseases like frontotemporal dementia and ALS spread across the brain's network. Researchers will follow 645 participants, including patients, family members, and healthy controls, for up to 2 yea…

This study looks at 40 people with early-stage Huntington's disease and healthy volunteers to see how well two types of brain scans can detect changes over 2 years. Participants get PET and MRI scans plus clinical exams at the start and after 2 years. The goal is to find better w…

This study aims to understand how diet affects the eyes and skin by measuring natural pigments called carotenoids. Researchers will use non-invasive methods to check levels in the eye and skin, and also test blood in some participants. Anyone age 7 or older having a routine eye e…

This study is observing up to 198 people with rapid-onset dystonia-parkinsonism (RDP) or related genetic mutations. Researchers aim to identify affected individuals, document how common the disease is, and track its progression over time. Participants undergo assessments of sympt…

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

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

This study aims to find better ways to measure how retinitis pigmentosa (a genetic eye disease that causes vision loss) progresses over time. Researchers will use advanced imaging and vision tests in 40 people with specific genetic forms of the disease. The goal is to identify re…

This early-phase study tests whether a new type of PET scan can measure inflammation in the brains of people with Parkinson's disease. Researchers will compare scans from 11 participants (both with Parkinson's and healthy volunteers) to see if the imaging drug 18F-NOS can help di…

This study explores whether a non-invasive brain stimulation technique called repetitive transcranial magnetic stimulation (rTMS) can reduce compulsive behaviors in people with anorexia nervosa. Twenty participants will receive multiple sessions of rTMS targeting a brain region l…

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

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

This observational study follows 37 children and young adults with juvenile-onset Huntington's disease (JoHD) to understand how their brains develop differently from adult-onset cases. Participants undergo cognitive tests, behavioral assessments, and MRI scans over time. The goal…

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

This study looks at gene activity in brain tissue removed during surgery from 10 people with temporal lobe epilepsy and hippocampal sclerosis. The goal is to find which genes are linked to the disease and may help predict outcomes. No new treatment is being tested; instead, resea…

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

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

This study was designed to test the safety and feasibility of a focused ultrasound device called Exablate Neuro with a new Echo-Focusing feature for people with treatment-resistant brain disorders like essential tremor or depression. However, the trial was withdrawn before enroll…

This early study tests an adaptive deep brain stimulation (aDBS) system in 2 people with severe, treatment-resistant OCD. The device records brain signals to identify patterns linked to OCD distress and stimulation side effects. The goal is not to treat OCD now, but to gather dat…

This study aimed to understand how blood pressure changes when standing up affect thinking abilities and brain blood flow in people with Parkinson's disease. Researchers planned to compare participants with and without orthostatic hypotension (low blood pressure upon standing) us…

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

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

This study tests whether giving patients with hand problems like carpal tunnel syndrome or trigger finger a decision aid—a booklet with clear information about their condition and treatment options—helps them feel more satisfied and less conflicted about their choices. About 126 …

This study follows 100 people aged 60-85 with mild memory or movement problems to see if losing smell, hearing, or balance speeds up cognitive decline. Researchers will test these senses and track thinking skills and quality of life for 5 years. The goal is to understand which se…

This study creates a registry for up to 500 people with CDKL5 Deficiency Disorder (CDD), a rare genetic condition that causes seizures and developmental delays. Patients and their caregivers will provide information about symptoms, treatments, and quality of life over several yea…

This study is checking whether a questionnaire called the MAP-Hand scale is a valid and reliable way to measure hand activity in people with carpal tunnel syndrome. Researchers will enroll 180 adults with mild or moderate carpal tunnel syndrome and compare their scale results to …

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

This study tests a video-based mobile app designed to reduce mental health stigma and medical mistrust among Black adults with moderate to severe depression or anxiety. Participants watch personal stories from others with similar experiences. The goal is to see if this approach i…

This study follows 200 people with Parkinson's disease or multiple sclerosis over one year to see how active they are. Participants use a smartphone app called Datos Health to track steps, workouts, and heart rate. A physical therapist sets personalized exercise goals. The goal i…

This study tests whether a special type of X-ray called DXA, taken while lying down, can measure spine curves as well as standard standing X-rays in people with scoliosis. About 20 participants will have both types of images taken, and researchers will compare the results and ask…