Neuromuscular disease

This study tests a new oral polio vaccine (nOPV3) in healthy young children, infants, and newborns in Panama. The goal is to see if it is safe and triggers a strong immune response compared to the current vaccine. About 1,500 participants will receive either the new or existing v…

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

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

This phase 3 trial is testing whether the drug pridopidine can slow the progression of amyotrophic lateral sclerosis (ALS) in 500 adults. Participants will take either pridopidine or a placebo daily for 48 weeks, followed by an open-label extension where everyone receives the dru…

This Phase 3 trial tests a product called NTX-001, a set of three solutions applied during surgery to help reconnect severed nerves in the arm. About 98 adults with severe nerve cuts will be randomly assigned to receive either standard nerve stitching plus NTX-001 or standard sti…

This early-phase trial is testing a new treatment called KITE-363 for people with certain autoimmune nerve diseases that have not responded to standard therapies. KITE-363 is a type of CAR T-cell therapy that uses a patient's own immune cells, modified to target and attack faulty…

This study tests two different doses of an intravenous immune globulin (IGIV 10%) as maintenance therapy for adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder causing weakness and numbness. About 161 participants will receive either a higher o…

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

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

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

This trial tests a product called NTX-001, which is applied during surgery to repair completely severed nerves in the arm or face. The product combines three solutions and a device, and is given only once. The study includes people with fresh nerve injuries (within 48 hours) that…

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

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

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

This study tests whether a device called MyokinE100, which sends electrical signals to the thigh muscles, is safe and practical for use in the ICU. It includes 50 critically ill patients with sepsis who are at risk of muscle weakness. Half will receive standard care plus daily 60…

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

This study tests whether starting non-invasive ventilation (a breathing machine) earlier than usual helps people with ALS. Researchers will use a prediction tool to find those at high risk of breathing problems within 6 months. 48 participants will be randomly assigned to early N…

This study tests a new device called the NeuroSpan Bridge for repairing damaged nerves in the arms or legs. It is compared to two standard treatments: a nerve guide and a nerve graft from another part of the body. The trial will enroll 80 people with nerve injuries and measure ho…

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

This study tests the long-term safety of a drug called LY4256984, given via spinal injection, in people with ALS. It includes 32 participants who completed an earlier study and will be followed for about 96 weeks. The main goal is to see how many people have serious side effects …

This expanded access program offers the drug tazbentetol to adults with ALS who cannot join other clinical studies. Participants take one 300 mg pill by mouth each day. The program aims to check the drug's safety and whether it helps control the disease.

This Phase 1 trial tests the safety and tolerability of a new drug called AZD5492 in 72 adults with lupus, myositis, or rheumatoid arthritis. Participants receive one or two doses under the skin and are monitored for side effects and how the drug moves through the body. The study…

This phase 2 trial tests whether tofersen, a drug already approved for a specific type of ALS, can help people with ALS who do not have the SOD1 gene mutation. Thirty adults will receive the drug via spinal injections over 24 weeks. Researchers will measure changes in nerve damag…

This study looks at the long-term safety of an experimental drug called LY4256984 for people with ALS. It enrolls up to 32 participants who have completed a prior ALS trial. The study will follow them for at least 96 weeks to see how well the drug is tolerated over time.

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

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

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

This study tests whether a weekly under-the-skin antibody treatment (XEMBIFY) provides similar blood levels as a standard IV infusion (Gamunex-C) given every 3 weeks in people with CIDP, a chronic nerve disorder. About 40 adults who are stable on IVIG will first receive Gamunex-C…

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

This Phase 2 trial tests a drug called claseprubart (DNTH103) in 36 adults with multifocal motor neuropathy, a rare nerve condition that causes muscle weakness. Participants receive either the drug or a placebo for 15 weeks. The study looks at safety, how the drug works in the bo…

This early-stage study tests a new drug called VTx-002 in 12 people with ALS. The main goal is to check if the drug is safe and tolerable when given as a single injection into the fluid around the brain and spinal cord. Researchers will also look for signs that the drug might slo…

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

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

This study tests an investigational drug called LTX-002 in people with amyotrophic lateral sclerosis (ALS). The drug is designed to target a specific genetic message linked to the disease. The main goal is to check the drug's safety and tolerability, and to see how it moves throu…

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

This study tests an experimental drug called NB-4746 in 80 people with ALS. The goal is to see if it is safe, how it moves through the body, and whether it can reduce a marker of nerve damage (NfL) in the blood. Participants are randomly assigned to receive either NB-4746 or a pl…

This phase 2 trial tests a combination of three drugs—carfilzomib, cyclophosphamide, and dexamethasone—in 20 people newly diagnosed with POEMS syndrome, a rare disorder affecting nerves and blood. The goal is to see if the regimen can reduce abnormal proteins and improve symptoms…

This study tests whether a processed nerve graft from a donor (Avance Nerve Graft) works as well as using a piece of the patient's own leg nerve to repair damaged nerves in the arm. About 178 adults with upper arm nerve injuries will be randomly assigned to receive either the don…

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

This early study tests a new treatment called azercabtagene zapreleucel (azer-cel) for people with B-cell mediated autoimmune disorders like multiple sclerosis and neuromyelitis optica. The treatment uses a patient's own immune cells that are modified in a lab to target and destr…

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

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

This study tests an experimental drug, dazucorilant, in 279 adults with ALS (Lou Gehrig's disease). The goal is to see if the drug can slow the loss of muscle function and improve quality of life over 24 weeks. Participants will receive either the drug or a placebo, and researche…

This study tests a new drug called riliprubart against standard IVIg treatment in 160 adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder causing weakness and numbness. Participants currently on IVIg will be randomly assigned to receive either r…

This phase 3 trial tests whether empasiprubart, given by IV infusion, can help adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder that causes weakness and disability. The study will enroll 160 participants. For the first 6 months, some get the …

This early-stage trial is testing a single injection of a gene therapy called INS1202, given into the spinal fluid, for people with ALS (Lou Gehrig's disease). The study includes 23 adults, some with a specific genetic mutation (SOD1) and some without known genetic causes. The ma…

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

This study tests a drug called riliprubart in 140 adults with chronic inflammatory demyelinating polyneuropathy (CIDP) that hasn't improved with standard care. Participants receive either riliprubart or a placebo for up to 111 weeks. The main goal is to see if the drug reduces di…

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

This study tests a new medicine called empasiprubart against the standard treatment (IVIg) for people with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disease that causes weakness and numbness. About 218 adults who have used IVIg before will receive either t…

This study tests whether zanubrutinib, a drug used for certain blood cancers, can improve nerve function in people with anti-MAG neuropathy—a rare condition causing numbness, weakness, and balance problems. About 50 adults in Italy will take the drug for 12 months. Researchers wi…

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

This Phase 2b study tests whether the drug IMVT-1402 can help adults with CIDP, a nerve disorder causing weakness and numbness. About 162 participants will receive either the drug or a placebo weekly for up to 52 weeks. The main goal is to see if the drug keeps the disease from w…

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

This study tests a brain-computer implant (Stentrode) for people with paralysis from motor neuron disease or similar conditions. The device is placed in a blood vessel near the brain and aims to let users control computers or assistive devices with thought. The trial focuses on s…

This phase 2 trial is testing whether an inhaled drug called cromolyn (PHENOGENE-1A) can help people with mild-to-moderate ALS when added to standard care. About 105 participants will receive either a low dose, high dose, or placebo for 24 weeks. Researchers will measure changes …

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

This study tests a sensory neuroprosthesis—implanted electrodes that send small electrical currents to remaining nerves—to restore sensation of a missing lower limb in people with diabetic peripheral neuropathy and amputation. The 15 participants will also have the option of musc…

This study tests whether a 4-month structured telerehabilitation program, delivered via video calls and an app, can reduce deaths and hospital readmissions in heart patients who have Post-Intensive Care Syndrome (PICS). About 326 adults aged 30-75 who were in the ICU for at least…

This study is testing several experimental treatments at the same time to see if they can slow down ALS, a disease that weakens muscles and gets worse over time. About 1,500 adults with early-stage ALS will take part. The goal is to find treatments that help people stay stronger …

This study tests whether the drug nipocalimab can delay or prevent relapses in adults with CIDP, a rare nerve disorder that causes weakness and numbness. Participants first receive nipocalimab, and those who improve are then randomly assigned to continue the drug or switch to a p…

This study tests a one-time gene therapy called SGT-003 in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it helps them move better, like standing up faster and climbing stairs. Participants will receive either the therapy or a placebo first, t…

This Phase 3 trial tests whether the drug claseprubart (DNTH103) can prevent relapses in adults with CIDP, a nerve disorder that causes weakness and disability. About 256 participants will receive either the drug or a placebo. The main goal is to see how long it takes for symptom…

This study tests the safety and feasibility of the BrainGate2 system, a brain-computer interface. Tiny sensors are placed in the brain area that controls movement, allowing people with tetraplegia (paralysis of all four limbs) to control a computer cursor or other assistive devic…

This study tests a gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1. The therapy is given once through an IV and aims to reduce harmful DMPK RNA and improve muscle function. The trial has two parts: first, finding the safest dose in a small gr…

This Phase 2 trial compares the drug dolutegravir alone versus dolutegravir combined with tenofovir in 146 adults with HTLV-1 infection and related symptoms. The main goal is to see if the combination lowers the virus level more effectively and improves pain, spasticity, and musc…

This study tests a drug called SPI-1005 in 200 adults with Meniere's disease, a condition that causes hearing loss, ringing in the ears, and dizziness. Participants will take the drug twice a day for 6 or 12 months to see if it is safe and tolerable. The goal is to find a treatme…

This study tests a one-time gene therapy called ST-503 for people with chronic pain from small fiber neuropathy that hasn't improved with standard treatments. The therapy aims to block pain signals before they reach the brain. The trial involves 27 adults and compares the gene th…

This study looks at two different doses of a medicine called Privigen in children aged 2 to 17 with a nerve disease called CIDP. The goal is to see which dose better prevents the disease from getting worse. About 30 children will take part, and the study is currently recruiting.

This phase 3 trial tests an experimental drug called AOC 1020 in 200 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo by IV infusion. The study measures muscle…

This study tests an experimental oral drug called ROC-101 in 40 adults with pulmonary arterial hypertension (PAH) or pulmonary hypertension linked to interstitial lung disease (ILD-PH). Participants take the drug alongside their standard care for 24 weeks, with a long-term follow…

This study tests a single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the tre…

This early-phase trial tests a stem cell product called EN001 in 27 adults with Charcot-Marie-Tooth disease type 1A, a genetic nerve disorder that causes muscle weakness. The study has two parts: first, an open-label phase to check safety and find the right dose; second, a placeb…

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

This study tests a new biologic drug called ARGX-119 in children aged 5 to 17 with spinal muscular atrophy (SMA). About 60 participants will receive either ARGX-119 or a placebo by IV infusion for 24 weeks, alongside their usual SMA therapy. The goal is to find the best dose and …

This Phase 2 trial is testing an investigational drug called COYA 302 in 120 adults with ALS. The drug combines two immune-modulating agents to reduce inflammation that may damage nerves. Participants will receive either COYA 302 or a placebo for 24 weeks, with an option to conti…

This study tests a brain implant that records activity from the motor cortex and stimulates the sensory cortex. It aims to help people with severe arm or hand impairment—due to spinal cord injury, stroke, or amputation—control external devices like computers. The trial focuses on…

This study tests an investigational drug called ENTR-601-45 in people with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. About 24 ma…

This study is testing whether a drug called tadalafil (often used for erectile dysfunction) combined with a home cycling program can help boys with Duchenne muscular dystrophy. The drug aims to improve blood flow to muscles, which is often poor in DMD, while exercise builds stren…

This study is testing whether a lower dose of radiation can effectively treat metastatic tumors that have a specific genetic change called an ATM mutation. The goal is to find the smallest effective dose to reduce side effects. About 45 adults with advanced cancer and an ATM muta…

This study tests a new medicine, DYNE-101, in 116 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The main goals are to check if the drug is safe and if it can reduce muscle stiffness and improve muscle function. Participants re…

This early-phase study tests a new treatment called RD06-05, which uses specially engineered immune cells (CAR-T cells) to target and calm the immune system in people with neurological autoimmune diseases like multiple sclerosis, myasthenia gravis, chronic inflammatory demyelinat…

This early study tests a brain-computer implant placed through blood vessels to help people with ALS control external devices using their thoughts. Ten participants with ALS and arm weakness will be followed for safety and device function. The goal is to restore some independence…

This early-phase trial is testing a new cell therapy called QH103 for people with severe autoimmune diseases like multiple sclerosis and myasthenia gravis that haven't responded to standard treatments. The therapy uses specially engineered immune cells to target and attack the fa…

This early-phase trial tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD), aged 2 to 8. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it increases dystrophin protein levels in muscles, which could …

This study tests an experimental drug called TP04HN106 in 60 people with ALS. Half receive the drug, half receive a placebo (saline), and all take riluzole tablets as standard care. The goal is to see if the drug is safe and can slow the decline in physical function measured by t…

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

This study tests whether giving ALS patients 20% more calories than their body needs (via a feeding tube) can slow the disease. Researchers will measure a nerve damage marker in the blood over 6 months. 76 adults with ALS who already use a feeding tube will be randomly assigned t…

This study tests whether an ultra-high-caloric diet made almost entirely of fat can help people with ALS live longer and slow the disease. Researchers will compare the diet to a low-fat placebo in 392 participants. The main goal is to see if the diet extends survival without the …

This study tests a drug called apitegromab in children under 2 years old who have spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The drug works by blocking a protein that limits muscle growth. Researchers want to see if adding apitegromab to stand…

This study compares two treatments for CIDP, a nerve condition that causes weakness and numbness. It involves 20 adults who did not get better with steroids. One group receives a blood-filtering procedure called immunoadsorption, and the other receives standard antibody infusions…

This study tests whether GLP-1 receptor agonists, a class of diabetes medications including semaglutide, can improve heart nerve damage in people with type 2 diabetes. 60 adults will receive either standard care or standard care plus a weekly injection for 6 months. Researchers w…

This study tests an investigational drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a condition causing muscle stiffness and weakness. Participants receive multiple doses of the drug or a placebo by IV to check safety and how the body processes it. The goal is…

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

This early-stage trial tests a gene therapy called scAAV9/JeT-GAN for giant axonal neuropathy (GAN), a rare, fatal nerve disease in children. The therapy is injected directly into the left vagus nerve to target autonomic nervous system symptoms. Only 4 people who have already rec…

This study tests whether functional electrical stimulation (FES) can improve arm and hand function in people with spinal cord or peripheral nerve injuries. Researchers will use the MyndMove device to stimulate muscles during short therapy sessions. The goal is to see if this appr…

This Phase 2 study is testing TAK-411, a specially modified immune globulin, in 36 adults with CIDP, an autoimmune disease that attacks nerve insulation and causes muscle weakness. The main goal is to see if TAK-411 improves physical function over 24 weeks compared to historical …

This early-stage trial tests a new biologic drug called ARGX-119 in 16 adults with a rare genetic muscle weakness condition called DOK7-congenital myasthenic syndrome (CMS). The study aims to see if the drug is safe and how the body processes it. Participants will receive either …

This early-stage trial tests a new drug called TOS-358 in women with advanced HR+ HER2- breast cancer that has a specific mutation in the PIK3CA gene. The study aims to find the safest dose and see if the drug works alone or with standard treatments like fulvestrant and CDK4/6 in…

This phase 3 study tests a drug called salanersen (BIIB115) in people aged 15-60 with spinal muscular atrophy (SMA). The drug is designed to help the body produce more of a protein needed for nerve and muscle function. Participants receive an injection into the spine once a year …

This study tests whether injecting stem cells from umbilical cords into the legs can help people with moderate to severe diabetic nerve damage. 140 adults will get either stem cell shots plus standard care, or standard care alone, and be followed for 24 weeks. The goal is to see …

This study follows 200 adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who are starting treatment with efgartigimod. Researchers will track changes in disability, grip strength, and quality of life over up to 2 years in everyday clinical settings. The…

This early-stage trial tests a new drug called EDK060 in 28 adults with Charcot-Marie-Tooth type 1A, a genetic nerve disorder. Participants receive a single dose of EDK060 or a placebo to check safety and how the drug moves through the body. The goal is to see if EDK060 is safe e…

This phase 3 trial tests a one-time gene therapy called MELPIDA for children with SPG50, a rare genetic disease that causes progressive paralysis and developmental delays. The study will give the treatment via a spinal injection to 24 children aged 4 months to 6 years and compare…

This study tests a small absorbable cap placed over cut nerve endings during finger amputation surgery. The goal is to prevent painful neuromas (nerve balling) that often cause chronic pain. Twenty adults who have had a traumatic finger amputation will be followed for up to a yea…

This study tests whether a special exercise program that combines rhythm and multitasking (called RYMA) can reduce falls, fractures, and heart problems in people aged 65 and older with type 1 or type 2 diabetes. Participants will be screened for fall risk, bone health, nerve func…

This study tests a brain-computer implant (BCI) that lets people with ALS or motor neuron disease control digital devices using only their thoughts. Ten participants will receive the implant, and researchers will monitor safety and how well the device works over one year. The goa…

This phase 2 trial tests whether anakinra (Kineret), an anti-inflammatory drug, can improve hearing in people with Meniere's disease or autoimmune inner ear disease who cannot take steroids. About 57 participants will receive daily injections of anakinra or a placebo for 42 days,…

This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…

This early-stage trial tests a new medicine, AMX0114, in 48 adults with ALS. The drug aims to reduce a protein called calpain-2, which may damage nerves. The main goal is to check safety and how the body handles the drug, not yet to prove it works.

This early study tests a brain-computer implant that reads brain signals to help people with severe paralysis (from ALS, stroke, or spinal cord injury) communicate. The device aims to decode imagined speech and allow control of computers. Only 2 participants will be enrolled to c…

This trial tests a new implantable device called ThecaFlex DRx in 90 people with spinal muscular atrophy (SMA) who cannot receive standard spinal injections due to spine problems or other risks. The device is placed under the skin and delivers the medication nusinersen directly i…

This pilot trial tests whether transplanting gut bacteria from healthy donors (MTP-101C) is safe and can help slow ALS. Twenty people with fast-progressing ALS will receive the treatment after a bowel cleanse. Researchers will track changes in muscle function, quality of life, an…

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

This early-stage trial tests the safety of siplizumab, a drug that may calm the immune system, in 48 adults newly diagnosed with ALS. Researchers will monitor side effects and how the drug moves through the body. The goal is to see if it is safe enough for larger studies.

This study tests a combination of two targeted drugs, dabrafenib and trametinib, in people with rare solid tumors that have a specific genetic change called BRAF V600E. The goal is to see how well the drugs shrink or control the tumors. The study includes both children (age 1 and…

This study tests whether a drug called glycerol phenylbutyrate (RAVICTI®) can reduce fatigue and improve daily life for people with pyruvate dehydrogenase (PDH) deficiency, a rare genetic condition that affects energy production. About 15 children and young adults (ages 2 to 25) …

This study is testing the long-term safety of an experimental drug called PGN-EDODM1 in 48 adults with myotonic dystrophy type 1 (DM1) who have already taken the drug in a previous study. Participants will receive the drug by IV infusion and be monitored for side effects. The goa…

This observational study follows up to 2,000 adults with ATTR-CM, a serious heart condition, who are starting treatment with acoramidis as part of their normal care. Researchers will track patient characteristics, how the drug is used, and its effects on heart function and qualit…

This study tests a single dose of NTLA-2001, a gene-editing therapy, in 1200 adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart. The goal is to see if it reduces heart-related deaths and events compared t…

This program offers efgartigimod PH20 SC to adults with CIDP who cannot join clinical trials and have not been helped by existing treatments. The drug is injected under the skin and aims to control the disease. The program continues until the patient stops treatment or the drug b…

This study tests whether a 3-week intensive summer camp combining motor skill training and strength exercises can improve movement in children with spinal muscular atrophy (SMA). Twenty children aged 5-17 who are already on stable disease-modifying therapy will attend 6-hour sess…

This study tests a new type of shoe, called the LEEF-shoe, designed to prevent foot ulcers in people with diabetes and nerve damage. About 30 adults will wear both the LEEF-shoe and standard orthopedic shoes for several weeks to compare how well they relieve pressure on the feet.…

This early-stage trial tests an experimental drug called ACR-2316 in 100 people with advanced solid tumors that have spread or come back. The main goals are to check the drug's safety, find the best dose, and see if it can shrink tumors. It is for patients who have had up to 3 pr…

This study tests whether giving botulinum toxin (Botox) injections early can prevent shoulder bone deformities in babies with obstetrical brachial plexus palsy, a nerve injury at birth. About 62 babies aged 10-11 months will receive either the injection or a sham procedure. The g…

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

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

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

This study aims to translate an English questionnaire used to diagnose carpal tunnel syndrome into Thai and check if it works well. Researchers will enroll 80 adults at Siriraj Hospital who have been diagnosed with carpal tunnel syndrome. Participants will answer the new Thai que…

This study is working on a new blood test for pregnant women that can check for serious genetic conditions like cystic fibrosis, spinal muscular atrophy, and sickle cell disease. Researchers will collect blood samples from 4,000 pregnant women who are at higher risk of passing on…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study tests a radioactive imaging agent called Iodine-124 evuzumitide (AT-01) in 150 people with systemic amyloidosis. The goal is to see how well it detects amyloid deposits in the heart and other organs using PET/MRI or PET/CT scans. If accurate, it could become a new tool…

This study is testing whether a new approach—using ultrasound to guide a tiny needle to collect fluid near the ulnar nerve, then analyzing it for leprosy bacteria DNA—can more accurately diagnose leprosy and confirm when it's cured. Researchers will follow 108 participants for on…

This study is testing a handheld device called mScan that uses a tiny, painless electrical current to measure muscle health. Researchers want to see if it can give similar results to an MRI, but faster and more conveniently. The study involves 150 adults with and without muscle d…

This study tests whether a wearable device called SleepImage can accurately detect respiratory failure in people with ALS (Lou Gehrig's disease). Researchers will compare the device's readings with standard overnight sleep tests in 15 patients. The goal is to find a simpler way t…

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

This study tests a special cooling and compression sleeve (Paxman Limb Cryocompression System) worn during chemotherapy to prevent nerve damage, a common side effect. About 200 cancer patients receiving weekly paclitaxel will use the device for up to 4 hours per session. Research…

About 30% of people who have surgery for a herniated disc still suffer from nerve pain afterward. This study tests whether applying a gel made from the patient's own blood (platelet-rich fibrin) around the nerve during surgery can prevent that chronic pain. The trial will enroll …

This study tests if special sensor-equipped insoles can help prevent foot ulcers from coming back in people with diabetes and nerve damage. About 400 high-risk participants will use the insoles alongside standard care or just standard care alone. The goal is to see if the insoles…

This study tests whether desloratadine, a common allergy medication, can prevent nerve damage caused by taxane chemotherapy in breast cancer patients. About 116 participants will receive either desloratadine or a placebo for 12 weeks while on chemotherapy. The goal is to see if t…

This study tests whether taking fish oil supplements can prevent nerve damage in the hands and feet caused by oxaliplatin chemotherapy in colorectal cancer patients. 120 participants will receive either fish oil or corn oil (placebo) during their treatment. The goal is to see if …

This study tracks how nerve pain from chemotherapy develops in breast cancer patients. It also tests a brain-stimulating device called a closed-loop brain-computer interface to see if it can prevent that pain. About 45 participants will be observed or receive the experimental sti…

This study tests whether cooling and compressing the arms and legs during taxane chemotherapy can prevent peripheral neuropathy—a common side effect causing numbness, tingling, or pain. About 777 cancer patients will be randomly assigned to one of three approaches: cryocompressio…

This study tests whether taking mecobalamin (a form of vitamin B12) can prevent or reduce nerve damage caused by taxane chemotherapy. About 326 adults with various solid tumors will take mecobalamin tablets or receive no preventive treatment during their chemo. The goal is to see…

This study tests whether wearing tight-fitting surgical gloves and compression knee socks during chemotherapy can prevent nerve damage (neuropathy) in breast cancer patients. The idea is that compression narrows blood vessels, reducing the amount of chemo drugs reaching the hands…

This study tests whether soaking feet in cold salt water can prevent nerve damage caused by the chemotherapy drug paclitaxel in breast cancer patients. Thirty women will be split into three groups: cold salt water foot baths, cold unsalted water foot baths, or standard care. The …

This study tests whether the oral medication venlafaxine, typically used for depression, can prevent postherpetic neuralgia—a persistent nerve pain that often follows a shingles rash. Researchers will enroll 832 adults who have had a shingles rash for less than 30 days and are ex…

This study tests a new treatment called ACTIVE that combines counseling (Acceptance and Commitment Therapy) with balance exercises for people with mild traumatic brain injury. Researchers want to see if it helps reduce dizziness and anxiety better than standard balance therapy al…

This study is testing if a non-invasive brain stimulation method called HD-tDCS can help people with ALS. The goal is to see if it can improve muscle control and breathing by calming overactive brain cells. The study will involve 80 adults with ALS and will measure changes in bra…

This study tests whether people can use a small, portable device at home to stimulate the vagus nerve through the ear. It aims to see if this approach is practical and acceptable for managing nerve pain caused by chemotherapy. The study includes 36 adults who have had platinum or…

This early-phase study is testing whether a capsaicin patch (made from chili peppers) can relieve nerve pain in the feet and legs caused by chemotherapy. Twenty adults who finished chemo within the past year and have lasting pain will try the patch. Researchers will track side ef…

This study tests whether a non-invasive brain stimulation technique called rTMS can reduce nerve pain, depression, and sleep problems in people with spinal cord injury. Researchers will compare two different rTMS protocols against a placebo in 63 adults. Participants continue the…

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

This study looks at whether a high-dose capsaicin patch (made from chili pepper extract) can relieve long-lasting tailbone pain that hasn't responded to other treatments. Researchers will review medical records from 106 patients treated over six years to see how well the patch wo…

This study is testing a new type of spinal cord stimulator that automatically adjusts its signal based on how your nerves respond. It aims to improve pain relief and quality of life for 25 adults with long-term, hard-to-treat pain in the trunk or limbs. The device is already appr…

This study tests a device called Walkasins, worn like an ankle strap and insole, that gives gentle vibrations to help people with peripheral neuropathy feel their feet better. The goal is to see if it improves walking, balance, and daily physical activity. Eighteen adults aged 60…

This study tests a small, TENS-like device that sends a mild electric signal to improve balance and walking in older adults. Researchers will measure walking speed and stability in 120 participants aged 60 and older, as well as younger adults for comparison. Participants will wal…

This study tests whether a diet low in added sugar, high in fiber, and rich in omega-3s (called LASO-3) can help reduce nerve pain, numbness, and tingling caused by chemotherapy. Fifty cancer survivors who finished chemo at least three months ago will receive virtual nutrition co…

This study tests a device called Walkasins, worn on the lower legs, that vibrates to give sensory feedback about foot pressure. The goal is to see if using it daily for six months improves balance and walking in 200 adults aged 55+ with peripheral neuropathy. Participants will do…

This study tests whether describing a common pain shot (epidural steroid injection) in a very positive way helps reduce leg pain more than a neutral description. Researchers will compare three groups: positive framing of the shot, neutral framing of the shot, and neutral framing …

This Phase 2 trial is testing four experimental drugs (taken orally or by IV) against placebo in 10,000 people with chronic pain from knee osteoarthritis, diabetic nerve damage, or low-back pain. The goal is to see if any of these drugs can safely reduce pain. The study uses a fl…

This trial tests a wearable robot called ENMS-BSF that combines soft muscles, electrical stimulation, and balance feedback to correct foot drop and foot inversion after a stroke. Sixty people who had a stroke at least six months ago and have mild-to-moderate walking problems will…

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

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

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

This study tests whether a spinal cord stimulator can reduce pain and improve nerve function in people with diabetic peripheral neuropathy. Sixty adults with pain that hasn't responded to standard treatments will be randomly assigned to receive the implant or standard care. Resea…

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

This study tests whether shortwave therapy can relieve symptoms of diabetic peripheral neuropathy, a common complication causing pain and numbness. About 202 adults with diabetes will receive either real shortwave treatment or a sham device for 20 minutes daily over five days. Re…

This study tests a new medicine called suzetrigine for people with nerve pain from diabetes. About 1,100 adults with diabetic peripheral neuropathy will receive either suzetrigine, a placebo, or an active comparator to see if it safely reduces pain over 12 weeks. The goal is to f…

This early-stage study tests whether a new drug called ASCT-83 is safe for people with nerve pain. It involves 72 healthy adults who will receive either the drug or a placebo. The study will also measure how the drug moves through the body. Results will help decide safe doses for…

This study tests a newer way to give epidural steroid injections for people with long-term leg pain caused by nerve issues. It compares the new 'transgrade' method to the standard 'interlaminar' method. The goal is to see which approach provides better pain relief three months af…

This study looks at whether electroacupuncture (a type of acupuncture with mild electric pulses) can help relieve pain from shingles and reduce the chance of long-term nerve pain. About 228 adults with shingles will be split into three groups: one gets electroacupuncture plus sta…

This study tests whether a capsaicin 8% patch (made from chili pepper extract) can reduce chronic nerve pain in teenagers aged 12 to 17 who have had pain for over 3 months after surgery or injury. Half the teens get the real patch, the other half get a placebo dressing. Pain leve…

This study tests whether a six-week at-home light therapy program is practical and helpful for childhood cancer survivors who have nerve pain or numbness from chemotherapy. Twenty survivors aged 5 and older will use a light therapy device on their symptoms for 15 minutes, three d…

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

This study tests special gloves and boots that gently squeeze the hands and feet during chemotherapy. The pressure may reduce how much chemo reaches the nerves, possibly preventing pain and numbness. About 142 adults with cancer will try the devices to see if they help avoid nerv…

This early-stage study tests whether an injection of RP-008 followed by a daily varenicline pill is safe and can reduce pain for people with trigeminal neuralgia, a condition causing severe facial pain attacks. About 24 adults aged 18-80 with classic or idiopathic trigeminal neur…

This study tests a new type of breathing machine (volume-targeted ventilation) for people with slowly worsening muscle diseases who already use a standard breathing machine at night. The goal is to see if it can better control carbon dioxide levels and improve sleep comfort. Abou…

This study tests if ranolazine, a heart medication, can safely reduce muscle cramps and improve quality of life in people with ALS. About 72 adults with ALS who have frequent cramps will receive either the drug or a placebo for 28 weeks. The main goals are to check safety and see…

This study compares two types of nerve-stretching exercises (tensioner and slider) for people with sciatica. Eighty participants with leg pain lasting 3 to 12 months will receive one of the techniques plus standard physical therapy. Researchers will measure pain, disability, and …

This study tests whether shining a special laser light on painful areas can reduce nerve pain caused by chemotherapy. About 70 cancer survivors with lasting nerve pain will receive either real or placebo laser treatments. The goal is to see if this safe, non-drug approach can imp…

This study tests whether scrambler therapy, a device that sends electrical signals to the skin, can reduce pain, numbness, and tingling from chemotherapy-induced peripheral neuropathy. About 90 adults with persistent nerve pain will receive either real or sham treatment for 10 se…

This study is tracking 1,000 people who have received or will receive the Allay Hydrogel Cap, a gel device placed during nerve surgery to protect the nerve ending. Researchers are collecting real-world data on pain levels, medication use, and complications to see how safe and hel…

This study tests whether a 10-week virtual exercise program can reduce nerve pain and improve daily activities in cancer survivors who have persistent nerve damage from chemotherapy. About 240 participants will either receive the program or usual care. The program includes person…

This study compares a targeted nerve block injection to usual pain treatment for people with sudden sciatica in the emergency department. About 100 adults will be randomly assigned to receive either the nerve block or standard care. Researchers will measure pain levels and walkin…

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

This Phase 2 trial tests an experimental oral drug called VX-993 for pain caused by diabetic peripheral neuropathy, a common complication of diabetes. About 300 adults with type 1 or type 2 diabetes and nerve pain in both legs for at least a year will receive VX-993, pregabalin (…

This study tests whether a single injection of RE104 can reduce symptoms of depression or mixed depression and anxiety in people with adjustment disorder caused by a serious medical illness like cancer, ALS, multiple sclerosis, Parkinson's disease, or idiopathic pulmonary fibrosi…

This study tests whether using cold wraps plus compression (cryocompression) on hands and feet during chemotherapy can reduce nerve damage (peripheral neuropathy) in people with gynecologic cancer. About 190 participants will be randomly assigned to receive either cryocompression…

This study tests whether a non-invasive brain stimulation technique called repetitive transcranial magnetic stimulation (rTMS) can reduce nerve pain caused by chemotherapy in breast and gynecologic cancer survivors. About 19 women who have persistent pain after chemo will receive…

This small study tests whether a gentle, non-invasive brain stimulation technique called tACS can reduce nerve pain in people with spinal cord injury. Fourteen participants will receive either real or sham stimulation for 20 minutes. Researchers will measure pain levels and heart…

This study tests an injected drug called Halneuron to see if it can safely reduce nerve pain caused by chemotherapy. About 240 adults who have nerve pain from platinum or taxane chemo will receive either Halneuron or a placebo shot. The goal is to measure pain relief, sleep, fati…

This small pilot study tests whether using a hand-held vibration device for just 3 minutes each day can safely reduce nerve pain and numbness in the hands caused by chemotherapy. Sixteen adults who finished chemotherapy at least 60 days ago will try the therapy at home. The main …

This study tests a home-based exercise program for people who were hospitalized with severe COVID-19. Participants use a smartphone app to guide them through high-intensity interval training and other exercises. The goal is to see if this approach improves walking ability, fitnes…

This study will give a steroid and numbing injection to 60 people with carpal tunnel syndrome, half of whom also have type 2 diabetes. Researchers will track pain, nerve function, and hand strength for 12 weeks to see if the diabetes group recovers as well as those without diabet…

This study tests whether balance rehabilitation that changes how patients see (closing eyes, using a mask, or projecting moving dots) can improve walking stability better than standard rehab with eyes open. It includes 40 adults with chronic nerve damage who have trouble with bal…

This study tests whether a single 30-minute session of transcutaneous electrical nerve stimulation (TENS) on the face can reduce jaw and facial pain, improve jaw movement, and make chewing easier for people with ALS or PLS. Ten participants will receive the treatment during one c…

This study tests whether a common back operation (microdiscectomy) actually relieves sciatica leg pain better than a pretend surgery. 122 adults with a herniated disc and persistent leg pain will be randomly assigned to get either the real operation or a sham procedure. Neither t…

This study tests two stretching techniques—post-isometric relaxation and active isolated stretching—in 40 adults with piriformis syndrome, a condition that causes buttock and hip pain. Over two weeks, participants will receive six sessions of one stretch type plus standard physio…

This study tests whether a gel containing pregabalin, applied through the skin using ultrasound (phonophoresis), combined with calf muscle stretching, can reduce nerve pain and improve walking in people with grade III knee osteoarthritis. The trial includes 35 adults aged 40-70 w…

This phase 3 trial tests suzetrigine, an experimental oral drug, for pain caused by diabetic peripheral neuropathy. About 734 adults with diabetes and nerve pain in their legs for at least a year will receive either the drug or a placebo. The main goal is to see if suzetrigine re…

This study tests whether playing virtual reality games can help people with Duchenne muscular dystrophy improve their arm and hand function. 36 participants will either receive standard physiotherapy or the same therapy plus VR games using a Meta Quest 3 headset. The games are de…

This study tests a device called the Sana Pain Reliever, a mask with earbuds that delivers pulses of light and sound for 15 minutes at a time. Researchers want to see if it can reduce chronic pain and improve sleep in people with post-treatment Lyme disease syndrome. The trial in…

This study is testing whether combining two types of electrical stimulation can help people who have had a stroke lift their toes while walking. Participants will receive mild electrical currents on their scalp and leg while doing walking exercises. The goal is to see if this com…

This study tests whether a new tablet called Y-4, which combines two drugs (pregabalin and riluzole), can better relieve postherpetic neuralgia—persistent nerve pain after shingles—compared to pregabalin alone. About 160 adults in China will take either Y-4 or pregabalin for 14 w…

This study tests whether a single 30-minute session of electrical nerve stimulation (PENS) can quickly reduce sciatica pain and improve daily function. 88 adults with long-term sciatica will be randomly assigned to receive PENS, TENS (skin patches), dry needling, or a placebo. Re…

This study is testing whether different types of NSAIDs (like ibuprofen, diclofenac, and meloxicam) work better for specific pain types in people with frozen shoulder. 120 adults with shoulder pain and stiffness will take one of these drugs along with home exercises for two weeks…

This study tests whether giving calcitonin early after a spinal cord injury can lower the chance or severity of nerve pain. About 126 adults with a recent spinal injury will receive either calcitonin or a placebo. Researchers will track pain levels and side effects for up to a ye…

This study looks at whether adding ultrasound-guided shoulder injections to a home exercise program can reduce pain and improve movement in people with frozen shoulder. About 68 adults with chronic shoulder pain will receive either one type of injection or a combination, and be f…

This study tests whether a treatment called Percutaneous Electrical Nerve Stimulation (PENS), combined with exercise, can reduce pain and improve quality of life in people with long-term sciatica. 88 adults with chronic leg pain from sciatica will be randomly assigned to receive …

This pilot study tests a mechanical in-exsufflator (MIE) device that helps ALS patients cough more effectively by pushing air in and then pulling it out. The goal is to see if daily use for four weeks can maintain or improve lung function. The study will enroll 36 adults with ALS…

This study tests a powered leg brace called the Nomad in 36 people who have trouble walking due to conditions like stroke, multiple sclerosis, or spinal cord injury. Participants will use the brace at home for three months to see if it improves their walking speed, balance, and q…

This pilot study is testing whether a commercially available CBG/CBD oil is safe and can help reduce nerve pain, numbness, and tingling caused by platinum-based chemotherapy. Twelve adults with gastrointestinal cancers who have completed chemotherapy will take the oil under the t…

This study tests a five-week tongue exercise program for 20 people with ALS to see if it can help with swallowing and speech. Participants press their tongue against a device at home five days a week, with weekly check-ins via telehealth. Researchers will measure tongue strength …

This study tests whether a modified version of the muscle relaxant metaxalone (640 mg) can reduce low back and leg pain in people with acute sciatica or spinal stenosis. Fifty adults aged 18 to 80 with new-onset pain will take either the drug or a placebo for 7 days. Researchers …

This study tests whether a simple head-and-body movement called the Epley maneuver can quickly relieve dizziness from benign paroxysmal positional vertigo (BPPV) in the emergency department. About 118 adults with BPPV will either get the real maneuver or a fake one. Researchers w…

This study compares two numbing drug recipes for a nerve block given before leg surgery. One group gets a mix of lidocaine and bupivacaine, while the other gets bupivacaine alone. Researchers want to see which combination works faster and provides better pain control after the op…

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

This study tests two types of foot and ankle exercises to see which one better helps balance, walking, and fall risk in people with diabetic peripheral neuropathy (nerve damage in the feet). Researchers will compare exercises for the small muscles in the foot with 3D ankle-foot e…

This study tests whether combining brain stimulation (rTMS) with virtual reality mirror therapy can reduce hard-to-treat nerve pain. 64 adults with chronic neuropathic pain that hasn't responded to drugs will receive either real or sham mirror therapy alongside rTMS. The goal is …

This study compares two exercise approaches for dead butt syndrome, a condition where the gluteal muscles become inactive, causing pain and discomfort. Forty participants will be randomly assigned to either gluteal activation exercises alone or combined with core strengthening. T…

This study tests whether adding a specific leg-rotation technique to standard physical therapy can better relieve sciatica caused by disc problems in the lower back. Researchers will measure pain, flexibility, and daily function in 70 adults aged 18 to 50. The goal is to see if t…

This study looks at whether a custom mouthguard (occlusal splint) can reduce pain and improve muscle function in people with teeth grinding (bruxism) and related jaw and neck muscle pain. About 40 adults will be split into two groups: one gets the mouthguard right away, the other…

This study tests whether a mobile app can teach family caregivers safe massage techniques to help cancer survivors with long-term nerve pain and numbness caused by chemotherapy. About 300 participants (survivors and their caregivers) will use the app at home. The goal is to reduc…

This study tests whether low-dose naltrexone (LDN) can safely reduce pain from diabetic neuropathy, a common complication of diabetes. Thirty-five adults who have not found relief from standard treatments will receive LDN and a placebo in random order. The goal is to see if LDN o…

This study tests whether combining spinal cord stimulation with a rehabilitation program can reduce chronic nerve pain in the legs and improve physical function. Ten adults aged 18-60 with nerve pain lasting over 6 months will receive the treatment bundle. The goal is to see if t…

This study tests a new type of deep brain stimulation called 'Burst-DBS' for people with long-term facial pain or pain after a stroke. Twenty adults will have electrodes placed in the thalamus (a pain-related brain area) and try both standard and burst stimulation patterns. The g…

This study tests whether eye drops made from cord blood plasma can reduce pain in people with long-term nerve-related eye pain. Twenty adults with persistent eye pain and abnormal corneal nerves will receive the drops. Researchers will measure pain levels and nerve health to see …

This study compares three methods to reduce pain during corticosteroid injections for arthritis, tendinopathy, or nerve compression in the hand, wrist, or elbow: no anesthesia, a vibrating device, or a cooling spray. Each participant will receive two of the three methods to see w…

This study tests whether psilocybin (the active ingredient in magic mushrooms) is safe and tolerable for veterans with spinal cord injury who also have depression. Thirty participants will receive low, medium, or high doses and be monitored for side effects like pain, muscle spas…

This study tests three different injections—exosomes, platelet-rich plasma (PRP), and steroids—to see which best eases long-term nerve pain after shingles. 45 adults with pain lasting at least 3 months will get four treatments every two weeks. Researchers will measure pain, quali…

This study tests whether a whole-food, plant-based diet (no added oils) can reduce painful diabetic neuropathy better than standard medication. Forty adults with type 2 diabetes and nerve pain will be randomly assigned to either the diet or medication optimization. The main goal …

This pilot study is testing whether CBD oil can help reduce pain and nerve damage in people with diabetic peripheral neuropathy (DPN). About 20 adults with mild to moderate DPN will receive either CBD or a placebo for 6 weeks. The goal is to see if CBD is safe and effective, and …

This study tests whether injecting a long-lasting numbing medication (Exparel) into the side of the nose can reduce severe face and headache pain. About 15 adults with conditions like migraine or cluster headache will receive either the drug or a placebo, then switch groups. The …

This study tests a gentle, hands-on technique called Strain-Counterstrain for people with pain from the piriformis muscle. Thirty adults with a tender spot in that muscle will receive a passive leg maneuver held for 90 seconds. Researchers will measure changes in pain threshold a…

Stanford University is testing a non-invasive device that uses pulsed radiofrequency to treat chronic nerve pain after injury. The device sends magnetic pulses through the skin to target the affected nerve. This Phase 2/3 trial will enroll 95 adults to see if the treatment reduce…

This study tests whether taking a combination of alpha-lipoic acid, vitamin E, and vitamin B after carpal tunnel surgery can reduce pain, numbness, and improve hand function. About 84 adults with carpal tunnel syndrome will receive either the supplement combo or a comparator, and…

This study tests a noninvasive spinal cord stimulation (tSCS) to improve walking and reduce muscle stiffness in 15 adults with hereditary spastic paraplegia (HSP), a rare nerve condition. Participants receive stimulation through skin electrodes on the lower back twice a week for …

This study tests whether taking amino acid supplements, along with a special diet and exercise program, can help people with facioscapulohumeral muscular dystrophy (FSHD) gain muscle, lose fat, and improve physical abilities like walking and strength. About 48 adults with FSHD an…

This study tests if combining two needle-based treatments works better than either alone for carpal tunnel syndrome. The treatments are a needle release of a wrist ligament and a dextrose (sugar water) injection around the nerve. The study will include 102 adults with moderate to…

This early-stage trial tests whether a cream made from a modified form of capsaicin (the stuff that makes chili peppers hot) can reduce foot pain in people with diabetic neuropathy. About 80 participants will apply the cream or a placebo to their feet twice a day for two weeks an…

This pilot study tests whether Tai Chi, alpha-lipoic acid supplements, and acupuncture can help ease chemotherapy-induced peripheral neuropathy (nerve pain, numbness, tingling) in people with gastrointestinal cancer. About 60 participants will try one or more of these approaches …

This study tests whether a physical therapist-guided exercise program can improve strength and walking ability in adults with myotonic dystrophy type 2 (DM2). Twenty-four participants will be split into three groups, each doing the same exercises but with different levels of ther…

This study tests a device that cools and squeezes the arms and legs during chemotherapy to prevent nerve damage, which can cause numbness, tingling, and pain. Fifty adults with breast or gynecologic cancer receiving taxane chemo will wear the device during infusions. Researchers …

This study tests a new drug called HRS-2129 in 168 Chinese adults with diabetic peripheral neuropathic pain (nerve pain from diabetes). Participants will receive either HRS-2129, a placebo, or the standard drug pregabalin to see if HRS-2129 reduces pain better than placebo. The t…

This study tests whether giving Gamma Knife radiation early—within 2 years of diagnosis—can provide better pain relief for trigeminal neuralgia than waiting. About 80 adults with classic or idiopathic TN will be assigned to early treatment or standard care. Researchers will track…

This early study tests a small device that sends gentle electrical pulses to nerves to relieve severe, long-lasting knee, elbow, or shoulder pain. About 30 adults with chronic pain will first try a temporary implant for up to 8 hours. If it works well, they can get a permanent ve…

This study tests whether using a long-acting local anesthetic called Exparel, along with education about opioids, can help people avoid taking opioid painkillers after carpal tunnel release surgery. About 64 adults with carpal tunnel syndrome will be split into two groups: one ge…

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

This study tests whether wrapping a stitched finger nerve with MatriDerm, a collagen-elastin matrix normally used for skin healing, improves sensation recovery. Seventy people with traumatic finger nerve cuts will be randomly assigned to receive either standard nerve stitching al…

This study is testing a self-hypnosis program that runs on a virtual reality headset to help people with HIV manage chronic pain. About 25 adults with HIV and long-term pain will use the software at home for 21 days. Researchers will check if it is safe, easy to use, and if it he…

This study looks at whether a personalized strength exercise program can improve muscle function and reduce weakness attacks in adults with hypokalemic or hyperkalemic periodic paralysis. Over 24 weeks, 36 participants will be tested at the start, at 12 weeks, and after 12 weeks …

This study tests whether adding peripheral magnetic stimulation to balance exercises can lower fall risk in older adults with diabetic polyneuropathy. Forty participants will receive either real or sham magnetic pulses to their feet while doing balance training. The goal is to se…

This phase 3 trial compares a capsaicin patch (applied once) to the daily pill duloxetine for treating painful nerve damage caused by chemotherapy. About 274 people whose nerve pain persists at least a month after chemo will be randomly assigned to one treatment. The goal is to s…

This study tests whether a special belt that vibrates can help people with balance disorders walk better and feel more steady. Researchers will enroll 90 adults aged 18-80 who have balance issues from conditions like inner ear problems or ataxia. Participants will use the belt an…

This study tests whether a new drug called LY4065967 can safely reduce nerve pain in people with diabetes. About 150 adults with diabetic nerve pain in their legs for at least 6 months will receive either the study drug or a placebo. The main goal is to see if the drug lowers ave…

This study tests a gel called VMD-3866 for pain from chemotherapy-induced peripheral neuropathy (CIPN). It is applied to the skin and works by blocking pain signals in nerves. The trial includes 16 adults with stable CIPN pain for at least 3 months. Researchers compare the gel to…

This study tests a non-invasive brain stimulation technique called deep rTMS to see if it can reduce chronic pain from nerve damage or fibromyalgia. About 70 adults with daily pain will receive either real or fake (sham) treatment over 3 months. The goal is to measure pain relief…

This study tests a powered neck brace (exoskeleton) in 10 people with ALS who have neck weakness. Participants will do simple tasks while wearing the device to see if it improves head movement and is comfortable. The goal is to help patients regain the ability to move their heads…

This study tests a smartphone app called Tuntun Rehabilitation Assistant to help people who have been in the intensive care unit (ICU) regain strength and mobility. About 70 adults from two hospitals in South Korea will use the app for daily 30-minute rehab sessions. Researchers …

This study compares two treatments—kinesiotaping (a special tape) and peloidotherapy (mud therapy)—plus home exercises, against exercises alone for people with mild-to-moderate carpal tunnel syndrome. 90 adults will be randomly assigned to one of three groups. The goal is to see …

This study tests whether pairing non-invasive brain and spinal cord stimulation with hand exercises can improve hand strength and dexterity in veterans with ALS. Thirty-two participants will first undergo a personalized optimization period, then receive two weeks of the combined …

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

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

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

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

This study aims to collect movement data from up to 7,500 people, including healthy volunteers and those with movement disorders. Participants perform tasks like walking or reaching while cameras track reflective markers on their body. The goal is to build a reference database th…

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

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

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

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

This study will check if certain blood markers can predict how well a nerve block procedure works for people with nerve pain. Researchers will measure pain levels and blood inflammation markers before and after the procedure in 120 adults. The goal is to see if these blood tests …

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

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

This study looks at how the location of brain-stimulating electrodes affects pain relief in adults with long-term nerve pain that hasn't improved with other treatments. Researchers will use brain scans to see which areas are activated and how that relates to pain reduction. The g…

This study follows 209 adults who survived a stay in the intensive care unit (ICU) for sepsis or respiratory failure. Researchers will track their muscle strength, physical function, and cellular markers over a year to understand why some patients recover while others do not. The…

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

This study follows 250 people who had ankle fractures to see how many develop nerve-related chronic pain after the bone heals. Participants answer pain questionnaires and undergo simple sensory tests. The goal is to better understand this type of pain, not to test any new treatme…

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

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

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

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

This study is looking for biological clues in the blood that explain why many people with ALS lose weight. Researchers will follow 1,000 patients in France and Germany, collecting blood samples and questionnaires alongside standard care. The goal is to find markers that could lea…

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

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

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

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

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

This study follows 50 children who had a nerve-freezing procedure (cryoneurolysis) before surgery to correct a sunken chest (pectus excavatum). Researchers check for long-term nerve pain, quality of life, and sensory recovery using questionnaires. The goal is to see if the pain r…

This study will give 100 adults with diabetes a thermal camera that attaches to their smartphone to take daily pictures of their feet. The goal is to collect temperature data and see if hot spots can predict where foot ulcers might form. Participants will use the device at home f…

This study is translating a questionnaire about brachial plexus birth palsy into Turkish and checking if it works well. 50 adults with this condition will fill out the new Turkish version along with other standard surveys. The goal is to provide a reliable tool for Turkish-speaki…

This study follows 600 adults who are genetically at risk for ALS due to family history. Over three years, researchers collect blood samples, speech recordings, and health data to track early signs of the disease. The goal is to build a rich database that scientists can use to be…

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

This study is gathering medical information, blood samples, and speech recordings from 2,000 people with ALS and healthy volunteers over two years. The goal is to create a shared resource that scientists can use to better understand ALS and develop new treatments. Participants ca…

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

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

This study follows adults who had shoulder tendon transfer surgery as children for obstetric brachial plexus palsy, a nerve injury during birth. Researchers will measure pain, arm movement, strength, and social appearance anxiety in 80 participants and compare them to healthy pee…

This study follows 60 people with ALS to see how swallowing problems change over time and how they affect quality of life. Researchers also want to understand patients' feelings about getting a feeding tube (PEG). The goal is to learn more, not to test a new treatment.

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

This study looks at why some people who survive a serious illness in the ICU develop long-lasting muscle weakness. Researchers will test 50 former ICU patients with exercise on a bike to measure heart and muscle function. The goal is to better understand this condition, not to te…

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

This study checks if a French version of the MEPS/MEPI elbow score works well for children aged 4 to 17. It includes kids with and without elbow injuries to see if the score accurately measures elbow pain and function. The goal is to make sure doctors can use this tool reliably i…

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

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

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

This study looks at whether smartwatches can help monitor chronic inflammatory demyelinating polyneuropathy (CIDP), a rare nerve disease that causes weakness and numbness. 35 adults with CIDP who use subcutaneous immunoglobulin therapy at home will wear a smartwatch for 12 months…

This study looks at how cancer treatments can cause nerve pain. Researchers will use simple tests, like checking how you feel heat, cold, and touch, along with small skin samples, to understand why this pain happens. The goal is to find better ways to measure and eventually treat…

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

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

This study tracks quality of life in 200 adults with chronic respiratory failure who use breathing support like non-invasive ventilation. Researchers will use questionnaires to see how treatments impact daily living over time. The goal is to better understand patient experiences,…

This study looks at how to best use a machine that helps people with neuromuscular disease cough and clear mucus from their lungs. Researchers will test different pressure settings to see which works best without causing side effects. About 30 adults with stable or slowly progres…

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

This study looks at how the FDA-approved drug edaravone (Radicava) affects people with different forms of ALS. Researchers will collect blood, urine, and spinal fluid samples from 160 participants to measure markers of oxidative stress. The goal is to identify which ALS patients …

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

This study looks at whether a vestibular implant (a device that helps with balance) can improve how people with severe inner ear damage perform tasks like staying upright, navigating, and thinking. About 20 people who already have the implant will be tested with it on and off, an…

This study looks at how spinal cord stimulation (SCS) affects brain activity in veterans with long-term nerve pain. Researchers will use non-invasive brain scans (fNIRS and EEG) to compare brain connectivity in SCS users and those being evaluated for SCS. The goal is to understan…

This study looks at how inhaled cannabis affects the brain in people with chronic low back pain. Researchers will use MRI scans to measure changes in brain blood flow and oxygen levels, and ask participants to rate their pain. The goal is to understand the brain mechanisms behind…

This study aims to learn more about Chediak-Higashi syndrome, a rare genetic disorder that causes light skin and hair, easy bruising, and frequent infections. Researchers will observe up to 60 patients over time, collecting clinical and genetic data to better understand the disea…

This study tests whether a playful coaching feature on a cough-assist device helps children learn to use it more effectively. Researchers will compare learning speed and cough quality in healthy children aged 6-12, and later in children with neuromuscular diseases. The goal is to…

This study looks at whether a longer fasting period before an upper endoscopy can lower the risk of stomach contents entering the lungs (aspiration) in people taking GLP-1 receptor agonists (like Ozempic or Wegovy). Researchers at Mayo Clinic will check the stomachs of 150 adults…

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

This early-stage study tests a brain implant called BrainGate in just 3 people with severe paralysis or speech loss from conditions like ALS or spinal cord injury. The goal is to see if the device is safe and can let users control a computer cursor or other tools by thought alone…

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

This study looks at how the drug enfortumab vedotin affects nerves in people with advanced or muscle-invasive bladder cancer. Researchers will use nerve tests and patient surveys to track changes over 9-15 weeks. The goal is to better understand and potentially reduce nerve damag…

This study will collect electronic health records from up to 2500 people with Duchenne or Becker muscular dystrophy, including female carriers, across U.S. clinics. The data will be combined with patient-reported information to give researchers a fuller picture of the diseases. T…

This study collects blood, skin, and spinal fluid samples along with health information from 2,500 adults with neuromuscular diseases like ALS, neuropathy, and muscular dystrophy. The goal is to store these samples and share them with researchers to speed up discoveries about how…

This study uses three wearable devices—a continuous glucose monitor, a heart monitor, and an activity tracker—to see if high blood sugar is linked to heart problems in people with Duchenne muscular dystrophy. Researchers will compare 80 participants with and without DMD. The goal…

This study looks at people with long-term nerve pain caused by taxane chemotherapy. It compares two groups: one using capsaicin 8% patches (made from chili peppers) and another receiving standard pain treatments. The goal is to see if the patches help small nerve fibers in the fe…

This study is following 600 people in Germany with a type of advanced lung cancer (non-squamous non-small cell) who are starting a three-drug treatment: tremelimumab, durvalumab, and platinum chemotherapy. Researchers want to see how well the treatment works in real life, especia…

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

This study is building a large database of health information from 5,000 people with ALS and other motor neuron diseases. Participants share details like symptoms, test results, and disease progression during regular clinic visits. The de-identified data is then shared with resea…

This study aims to find genetic causes and risk factors for ALS by analyzing DNA from 1,200 people in Norway with probable or definite ALS. Researchers will look for known disease-causing mutations and search for new genes linked to the disease. The goal is to better understand w…

This study is testing whether a smartphone app can accurately measure how people walk, without needing special markers or equipment. Researchers will compare the app's measurements to a standard motion-capture system in 30 volunteers—some with neuromuscular diseases like SMA or m…

This study looks at whether changes in smell and taste contribute to poor nutrition in people with ALS. Researchers will measure food preferences in 60 adults with ALS using a computer test. The goal is to find new ways to improve diet and quality of life by personalizing food ad…

This study tests whether non-invasive sensors can detect reduced blood flow in the legs during long pelvic surgeries. Thirty adults will be monitored during their procedure. The goal is to see if this approach can help prevent complications like limb ischemia.

This study looks at why some people develop nerve pain after a spinal cord injury. Researchers will use pain tests, nerve measurements, and heart rate checks in 300 participants to understand the underlying causes. The goal is to better classify pain types, not to test a new trea…

This study follows 100 adults who had surgery for traumatic nerve injuries in the hand or arm. Researchers will measure hand function, strength, and quality of life over time to better understand long-term recovery. The goal is to gather information, not to test a new treatment.

This study follows 100 adults who had a chest tube placed after breaking their ribs. The goal is to learn how many develop long-term nerve pain (intercostal neuralgia) and how it affects their quality of life. Researchers will track pain levels and daily function over time to bet…

This study looks at whether a new eye-tracking device can help doctors see how well multiple sclerosis (MS) treatment is working. About 224 Canadian adults with relapsing-remitting MS will use the device during routine care. The goal is to find out if eye movement changes can rep…

This study looks at how tired people with spinal muscular atrophy (SMA) feel during daily activities while taking the drug nusinersen. Researchers will use a new questionnaire called the SMA EFFORT to measure this fatigue. The goal is to better understand fatigue in SMA and see i…

This study follows up to 60 babies with spinal muscular atrophy (SMA) found at birth, plus 30 healthy babies, for up to 30 months. Researchers use wearable devices worn at home to track muscle development and find when movements start to differ from normal. The goal is to identif…

This study is for people with ALS and healthy volunteers. It uses brain and spine MRI scans and blood tests to find markers that show how the disease progresses. The goal is to develop better tools to measure ALS over time. About 90 participants will be followed for up to one yea…

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

This study is building a worldwide online registry for people with Duchenne or Becker muscular dystrophy, including female carriers. Participants share their health information to help researchers learn more about the disease and to match patients with clinical trials. The goal i…

This study looks at a specific gene (IGLV1-44) to understand why it causes two different diseases: AL amyloidosis and POEMS syndrome. Researchers will analyze blood and bone marrow samples from 100 adults with these conditions or multiple myeloma. The goal is to find genetic patt…

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

This study looks at how certain substances applied to the skin affect blood flow in people with nerve pain caused by chemotherapy. Researchers will compare healthy volunteers and patients to better understand the pain. The goal is to learn more about the condition, not to test a …

This study is developing a standard test to measure how well assistive technologies (like eye trackers) and brain-computer interfaces help people with spinal cord injury or ALS use computers and phones. Researchers will test 60 participants, including healthy volunteers and peopl…

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

This study gathers medical information from 200 people with glycogen branching enzyme deficiency, known as GSD IV or APBD. The goal is to learn how these rare diseases progress over time. No new treatments are tested; instead, researchers review clinic records to better understan…

This study is testing a new, non-invasive brain monitor (Neurosteer) in 100 ICU patients who are on breathing machines and sedatives. The monitor is a small device placed on the forehead that measures brain activity. Researchers want to see if it can accurately track levels of se…

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

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

This study follows 105 boys from birth to age 3 who have Duchenne muscular dystrophy (DMD), identified through newborn screening. Researchers will measure motor and cognitive skills over time to understand how the disease develops in early childhood. The goal is to gather informa…

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

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

This study is testing whether tiny particles in the blood, called extracellular vesicles, can predict nerve damage caused by chemotherapy. Researchers will follow 120 adults with various cancers who are receiving specific chemo drugs. Participants give blood samples and answer qu…

This study is building a large registry of people with ALS in the United States. The goal is to count how many people have the disease and learn about possible causes, such as environmental or genetic factors. Anyone 18 or older with ALS can join. The registry does not test a tre…

This study looks at how nerves are affected in people who develop nerve damage from the chemotherapy drug cisplatin. Researchers will use a special test to measure nerve activity and compare it to normal nerves. The goal is to better understand this side effect and find early war…

This study looks at whether a common heart procedure (using the wrist artery) can damage nerves in the hand. Researchers will check 400 patients for signs of carpal tunnel syndrome or other nerve problems. The goal is to understand the risks better, not to test a new treatment.

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

This study aims to see if a quick bedside sensory test can predict which painkiller—pregabalin, duloxetine, or a placebo—works best for people with nerve pain in their feet. About 190 adults with diabetic, chemotherapy-related, or idiopathic nerve pain will try each treatment for…

This study is testing a device called the mScan to see if it can track how ALS progresses over time. Researchers will measure electrical signals in muscles and compare them to standard tests. The goal is to find a simple, objective way to monitor the disease that could be used at…

This study looks back at the last 10 years in Denmark to understand how many people use breathing machines at home through a tube in their windpipe. Researchers will track which diseases lead to this need and how many patients survive one year after starting. The goal is to spot …

This study follows 100 people with ALS to see how their autonomic and sensory nerves change over time. Researchers will use skin biopsies and other tests to track nerve damage and look for biomarkers that could help predict disease progression. The goal is to better understand AL…

This study looks at whether a balance exercise called IVA is safe and helpful for adults who have vision problems, with or without inner-ear balance issues. Researchers will compare two groups: people with vision problems only and those with both vision and inner-ear problems. Pa…

This study is looking at whether people with benign paroxysmal positional vertigo (BPPV) have differences in their autonomic nervous system compared to healthy people. Researchers will measure heart rate variability (HRV) in 104 participants (52 with BPPV and 52 matched controls)…

This study looks at people with shoulder pain lasting at least 3 months to understand if nerve-related pain and a condition called central sensitization (where the nervous system becomes overly sensitive) play a role. Researchers will use questionnaires and simple movement tests—…

This study aims to find early biological markers (biomarkers) for Charcot-Marie-Tooth disease type 1A (CMT1A), a common inherited nerve disorder. Researchers will analyze blood and nerve samples from 70 people aged 10-30 with CMT1A, along with animal models, to identify signs tha…

This study takes skin biopsies and blood samples from people with ALS, healthy carriers of ALS-related gene mutations, and control subjects. Researchers will grow skin cells in the lab to study how the disease works at a cellular level. The goal is to better understand ALS, not t…

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

This study watches how children under 5 with LAMA2-related muscular dystrophy move and develop over two years. Researchers will test muscle function, swallowing, breathing, and collect blood samples. The goal is to learn more about the disease so future treatments can be tested e…

This study aims to uncover genetic and biological differences between people with Menière's disease, those with vestibular migraine, and healthy individuals. Researchers will analyze blood samples from 250 participants to look for specific gene variations and biohumoral factors. …

This study is testing whether special insoles worn inside shoes can measure how well children with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk. About 106 participants, including healthy children, will wear the insoles during walking tests and in daily …

This study looks at the long-term safety of the drug tofersen (Qalsody®) in people with a rare, inherited form of ALS called SOD1-ALS. Researchers will collect health information from about 125 participants in Europe and the US over at least 7 years. The main goal is to see what …

This study is testing whether a protein called titin, found in urine, can serve as a reliable marker of muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after activities like walking down stairs, and track daily …

This observational study follows 75 people with shoulder impingement syndrome who are already receiving standard physical therapy. Researchers want to see if nerve-related pain and negative thinking about pain (called pain catastrophizing) affect how well patients stick to their …

This study aims to create a simple, objective test to detect nerve damage caused by the chemotherapy drug vincristine in children and teens. Researchers will use a new device to measure nerve function and compare it to standard assessments. The goal is to help doctors adjust chem…

This study aims to understand how different types of changes in the DMD gene are linked to heart problems in boys and young men with Duchenne or Becker muscular dystrophy. Researchers will use heart tests and blood samples to look for early signs of heart issues. The study involv…

This study follows nearly 3,000 people, including healthy adults and those with memory or movement problems, to learn how brain diseases like Alzheimer's and Parkinson's develop. Researchers use advanced brain scans and tests to track changes over time. The goal is to improve ear…

This study looks at how people with ALS and their caregivers communicate verbally. Researchers will record conversations and measure speech clarity, sentence complexity, and pauses. The goal is to understand how communication changes over time. 152 participants will take part in …

This study tests a virtual reality (VR) game to measure hand-eye coordination in children aged 7 to 17 with and without chronic balance (vestibular) disorders. Researchers want to see if the VR tasks are reliable and fun for kids, and how balance problems affect coordination. The…

This study uses a new ultra-high-resolution PET scanner to take detailed pictures of the brain and spinal cord in 300 healthy volunteers and people with conditions like Alzheimer's, Parkinson's, ALS, and psychotic disorders. The goal is to understand how small brain regions chang…

This study measures how hard children with conditions like neuromuscular disease, scoliosis, or heart problems work to breathe. Researchers use a thin tube placed through the nose into the esophagus to measure breathing muscle strength and effort. The goal is to better understand…

This study looks at 57 people aged 20 to 70 who carry the Val50Met gene variant linked to hereditary ATTR amyloidosis, a condition that can damage nerves and the heart. Researchers want to describe the different symptoms people experience, such as heart problems, nerve issues, or…

This study is gathering information from 4500 people who have or will have brain surgery for conditions like aneurysms, tumors, epilepsy, and Parkinson's disease. The goal is to find patterns that help doctors predict how well someone will recover. By standardizing how outcomes a…

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

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

This study aims to understand why myotonic dystrophy type 1 affects people so differently. Researchers will remotely assess muscle strength, memory, and activity in 1,000 participants, and analyze their DNA from a blood sample. All activities are done from home using a mailed too…

This study looks at how the constant vibration from tattoo machines may damage nerves in the hands of tattoo artists. Researchers will test touch, strength, and sensation in 20 tattoo artists and compare them to 20 people who don't use vibrating tools. The goal is to understand i…

This study is creating a large database of 1,000 people with ATTR amyloidosis, a rare disease that causes abnormal protein buildup in organs. Researchers will collect health information over time to better understand how the disease progresses and to develop tools that help docto…

This study is creating a detailed registry of 500 people with hereditary spastic paraplegia (HSP), a rare disorder that causes progressive leg stiffness and weakness. Researchers will collect medical history, genetic data, and patient feedback over time to better understand how t…

This study examines DNA from 575 breast cancer patients who received the chemotherapy drug paclitaxel. Researchers want to find genetic differences that explain why some patients develop nerve damage (peripheral neuropathy) while others do not. The goal is to identify biomarkers …

This observational study will follow 20 adults with a confirmed genetic mutation for hereditary transthyretin amyloidosis (ATTR), a rare and serious condition that causes abnormal protein buildup in organs. Researchers will conduct thorough medical exams, heart tests, and neurolo…

This study looks at how physical abilities impact the quality of life of people with ALS, a disease that weakens muscles over time. About 30 adults with ALS will fill out questionnaires about their daily function and well-being during regular clinic visits for up to two years. Th…

This study explores whether small, skin-friendly sensors can detect early signs of muscle tone problems in newborns up to 8 weeks old. Researchers will compare sensor data with standard medical exams to see if the technology can identify atypical muscle development sooner. The go…

This research study at Boston Children's Hospital is looking at the genes and proteins involved in congenital myopathies—rare muscle diseases that are present from birth. Researchers will analyze DNA from up to 4,000 participants, including patients and their family members, to f…

This study looks at how common nerve pain is in people aged 80 and older. Researchers will check pain levels and overall health in 450 volunteers. The goal is to better understand the link between nerve pain and aging, not to test a new treatment.

This study, called CRiALS, is a large umbrella protocol that aims to recruit up to 10,000 people. It includes individuals with ALS or related neurodegenerative diseases, their family members, and healthy volunteers. The main goal is to build a pool of participants for a variety o…

This study follows healthy people who have a family history and genetic risk for ALS. Researchers want to find early warning signs and understand what triggers the disease. Participants do not receive any treatment, but their health is tracked over time. The goal is to learn how …