Neuromuscular disease

Researchers are testing whether a new oral polio vaccine called nOPV2, given at birth, helps infants block type 2 poliovirus in their intestines. The trial enrolls 740 healthy newborns who have received standard injected polio vaccines. Scientists will check stool samples for sig…

This study tests whether giving two new oral polio vaccines (types 1 and 2) together is safe and works as well as giving them separately in healthy 16-week-old infants. About 675 babies in Panama will receive either the combo or single vaccines. The goal is to see if the combo ap…

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 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 will follow 36 people with spinal muscular atrophy (SMA) who receive a gene therapy called onasemnogene abeparvovec (ITVISMA) injected into the spinal fluid. The goal is to see how well it works and how safe it is in everyday medical practice. Participants must be at l…

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 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 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…

Researchers are comparing two sequences of treatment for metastatic castration-resistant prostate cancer that carries certain DNA repair gene mutations. Participants receive either carboplatin first and olaparib second, or olaparib first and carboplatin second, switching after th…

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

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

This study tests whether a soft, FDA-approved belly band can help premature babies on breathing support develop stronger abdominal muscles. Researchers will measure muscle thickness with ultrasound and see if babies reach full oral feedings earlier. About 40 premature infants bet…

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 early study tests a device called MyoRegulator® that uses mild electrical currents to calm overactive motor neurons in people with ALS. The goal is to see if it is safe and feasible, and whether it might slow disease progression. Fifteen participants will receive the treatme…

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 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 pilot study tests whether terazosin, a drug used for high blood pressure, can increase energy levels in people with ALS. Twenty participants will take either terazosin or a placebo for 12 weeks. The goal is to see if the drug is safe and if it raises ATP, a key energy molecu…

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

This 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 single injection of TRCN-1023 into the spinal fluid of adults with ALS. The goal is to see if it is safe and shows early signs of slowing the disease over 24 weeks. Participants are randomly assigned to receive the drug or a placebo, and neither they nor their …

This trial is testing a robotic exoskeleton called ABLE Kids to see if it is safe and practical for children who have trouble walking due to conditions like cerebral palsy, spinal cord injury, or muscle weakness. Fifteen children will use the device in up to ten one-hour training…

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

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

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

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

This Phase 3 trial tests whether 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 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 Phase 2 study tests a drug called apitegromab in 60 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. Participants will receive either the drug or a placebo every 4 weeks for a year. The main goal is to see if the dru…

This 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 whether the drug usnoflast can slow the progression of ALS, a severe nerve disease that causes muscle weakness and breathing problems. About 240 adults with ALS will receive either usnoflast or a placebo for 36 weeks. Researchers will measure changes in daily fun…

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 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 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 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 study tests a program called PICU Up! that helps critically ill children start moving earlier while in the pediatric intensive care unit. The program includes daily activity plans, better sedation management, and family involvement. Researchers want to see if this approach r…

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 trial investigates different types of steroid injections for back, neck, and related pain to see which are most effective and safe. It compares various steroids, doses, and injection sites, including epidural and facet joint injections. The study measures pain relief and fun…

This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping a specific part of the gene (exon 44). The trial has two parts: first, to find the safest and most effecti…

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 early-stage trial tests an experimental drug called empasiprubart in 60 adults with multifocal motor neuropathy (MMN), a rare condition that causes muscle weakness. The drug is given as an IV infusion, and researchers will use ultrasound to measure nerve thickness, along wit…

This study tests whether a 12-week program of supervised foot and ankle exercises can help people with diabetes who have nerve damage in their feet. The exercises are done in groups at primary care clinics. Researchers will measure changes in symptoms, foot movement, and quality …

This study tests a drug called salanersen in babies with spinal muscular atrophy (SMA) who have already received gene therapy. The drug aims to help the body make more SMN protein, which is needed for muscle function. About 42 babies with two copies of the SMN2 gene will be rando…

This early study tests whether repeatedly opening the blood-brain barrier with ultrasound is safe and might slow ALS. Twenty-three adults with ALS will receive 9 ultrasound sessions over 24 weeks. The first part checks safety; the second looks for signs of slowing the disease.

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 trial tests whether a robot-assisted surgical procedure can relieve chronic pain caused by trapped nerves in the pelvic area. The surgery aims to free the pudendal and inferior cluneal nerves, which may be compressed and causing pain. Researchers will measure changes in pain…

This study tests whether a device called the LIC Trainer, used twice daily at home, can slow the decline in lung function and delay the need for a breathing machine in people with ALS. Fifteen adults with early-stage ALS will use the device and be monitored every three months. Re…

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

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

About 10-20% of people with ALS have a specific antibody (anti-NRIP) in their blood that is linked to faster disease progression. This trial tests whether removing that antibody through plasmapheresis — a procedure that filters the blood — can slow the decline. Twenty participant…

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

This study follows 250 boys (ages 2 and older) with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety an…

This study tests a wearable device called the Leia sensory prosthesis in 80 adults with peripheral neuropathy, a condition that often causes balance problems and unsteady walking. Participants will either use the active device or a sham version for two months. The main goal is to…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 tests a temporary electrical stimulation system that delivers a single, one-hour therapy to help heal acutely injured peripheral nerves. Researchers will enroll 110 people with recent nerve injuries to see if the treatment is safe and improves sensory recovery. The app…

This study tests several already-approved drugs to see if they can slow down motor neuron disease (MND) and help people live longer. About 1150 adults with MND will be randomly assigned to receive one of the study drugs or a placebo. The trial uses a flexible design, allowing ine…

This study looks at whether drinking non-alcoholic red wine, which is rich in natural compounds called polyphenols, can help older adults keep their thinking sharp and muscles strong. Researchers will give 72 people aged 60-74 either non-alcoholic red wine or water, both as part …

This early-stage study is testing whether clenbuterol, a drug used for breathing problems, is safe for people with facioscapulohumeral muscular dystrophy (FSHD). About 30 adults with FSHD will receive one of three doses of the drug. Researchers will monitor side effects, heart ra…

This phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, the…

This study tests whether adding a daily probiotic tablet and a coenzyme Q10 toothpaste to standard gum cleaning can improve oral health in pregnant women. Forty pregnant women will be split into two groups: one gets the probiotic plus Q10 toothpaste, the other gets only the Q10 t…

This study tests whether taking N-acetylcysteine (NAC) and EH-301 together with the standard drug riluzole can slow down amyotrophic lateral sclerosis (ALS). About 90 people with early-stage ALS will receive either the active combination or a placebo for 6 months. After that, eve…

This early-stage study tests a single dose of a gene therapy called CTx1000 in 15 people with ALS. The main goal is to see if it is safe and tolerable. Participants must have had ALS for 2 years or less and be on stable standard medications. This is not a cure; it aims to control…

This study tests a new drug called ATX-01 in 56 adults with myotonic dystrophy type 1, a condition that causes muscle stiffness and weakness. The drug aims to block a molecule that may contribute to the disease. The trial will first give a single dose, then multiple doses, to che…

This study is testing whether a robotic surgical system called Symani can safely and effectively repair damaged nerves in the hand. One hundred adults with a finger nerve injury will be randomly assigned to receive either robotic-assisted or traditional manual surgery. The main g…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This early study tests the safety and function of a brain-computer interface (BCI) implanted by a robot in people with severe paralysis. The device aims to let users control external devices like computers or phones using their thoughts. The trial enrolls 15 adults with quadriple…

This study tests whether giving a 10-minute electrical stimulation to a repaired finger nerve during surgery can speed up healing and improve sensation. Sixty adults aged 18-80 with a cut digital nerve will receive either standard surgical repair alone or with added electrical st…

This study tests whether giving the drug natalizumab (Tyruko) within two weeks of first MS symptoms can help repair nerve damage. Forty adults with a first episode of MS-like inflammation will receive either the drug or a placebo. The main goal is to see if early treatment improv…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This phase 3 trial tests whether the drug rituximab can improve disability in people with anti-MAG neuropathy, a rare nerve condition that causes progressive weakness and numbness. The study will enroll 90 participants who have had the disease for 5 years or less and have shown r…

This study follows 30 adults with chronic inflammatory demyelinating polyneuropathy (CIDP) who are switching to the treatment HyQvia. Researchers will use questionnaires and medical records to understand why patients chose to switch, how satisfied they are, and how the treatment …

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

Motor neuron disease (MND), including ALS, is hard to diagnose and often takes years to confirm. This pilot study uses a powerful 7 tesla MRI scanner to look for detailed brain features, called imaging biomarkers, that are specific to MND and its subtypes. Researchers will scan 3…

Researchers at Gødstrup Hospital are studying whether balance and dizziness problems in young children come from the inner ear's balance system. The study includes 30 children aged 6 months to 10 years who have delayed gross motor development or dizziness and balance issues. Chil…

Researchers are studying how often children with sensorineural hearing loss also have problems with their balance system. The study enrolls 48 children aged 3 to 10 with hearing loss in one or both ears. Children complete questionnaires and a series of balance and inner-ear tests…

This study is testing a smartphone app that records and analyzes speech to help diagnose and monitor neurodegenerative conditions like dementia, Parkinson's disease, multiple sclerosis, and motor neuron disease. Researchers will compare speech patterns from 150 people with these …

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

This study will test whether artificial intelligence (AI) can effectively screen for diabetic retinopathy (eye damage) in 400 children with type 1 diabetes. Each child will have their eyes examined using an AI-powered camera, standard eye exams, and a detailed scan. The researche…

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

This study is 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 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 tests a portable DNA device called MinION that can identify bacteria and fungi in foot wounds of people with diabetes in under 4 hours. Standard lab cultures take longer and may miss some germs. The goal is to see if this faster method gives a more complete picture of …

This study tests whether measuring a specific receptor (P2X4) on blood cells can help diagnose amyotrophic lateral sclerosis (ALS), a severe nerve disease. Researchers will compare P2X4 levels in 50 people — some with ALS and some healthy volunteers. If the test works, it could l…

This Phase 3 study is testing whether markers in pancreatic fluid and blood can detect early pancreas cancer in people with high genetic risk. Participants receive a secretin injection to collect fluid during an endoscopy. The goal is to improve early detection in those with here…

This study is translating and testing a questionnaire called PainPREDICT for Thai-speaking people with diabetes. The goal is to create a mobile app that can screen for painful diabetic neuropathy, a common complication that causes nerve pain. Researchers will check if the Thai ve…

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 phase 2 trial is testing an experimental drug called SIR2501 in adults with solid tumors who are about to receive paclitaxel-based chemotherapy. The goal is to see if SIR2501 is safe, tolerable, and might reduce the development of chemotherapy-induced peripheral neuropathy—a…

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…

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 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 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 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 if wearing surgical gloves during oxaliplatin chemotherapy can prevent nerve damage in the hands. About 84 adults with advanced colon cancer will be randomly assigned to wear gloves or not during treatment. The goal is to see if this simple, low-cost method reduc…

This study tests whether a special exercise program, called PRESIONA, can prevent nerve damage (peripheral neuropathy) caused by chemotherapy in breast cancer patients. The program combines aerobic and strength exercises with blood flow restriction cuffs. Researchers will compare…

This study compares a high-tech rehabilitation program—using robotic systems, virtual reality, and balance platforms—to standard physical therapy for people with acute neuromuscular diseases like Guillain-Barré syndrome. About 124 adults aged 18 to 80 will participate. The goal i…

Small fiber neuropathy damages tiny nerve fibers, causing severe pain that often resists standard painkillers. This trial tests whether repetitive transcranial magnetic stimulation (rTMS), a non-invasive technique that sends magnetic pulses to specific brain regions, can reduce t…

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…

Researchers are testing whether rehabilitation that uses virtual reality, robotic devices, and balance platforms works as well as traditional therapy for adults with chronic neuromuscular diseases such as ALS, CIDP, and Charcot-Marie-Tooth disease. The trial enrolls about 60 peop…

Researchers test whether adding rectal ozone therapy to usual care can reduce numbness and tingling in people with chemotherapy-induced peripheral neuropathy. The trial enrolls 42 adults whose cancer is stable or in remission and who have had persistent numbness or tingling for a…

Researchers compare two exercise programs to see which better improves posture and stability in adults with diabetic peripheral neuropathy, a nerve condition that raises fall risk. Thirty-six participants aged 40 and older with type 2 diabetes and diagnosed neuropathy join the si…

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…

Carpal tunnel syndrome happens when the median nerve gets squeezed at the wrist, causing numbness, tingling, and pain in the hand. This study compares three ultrasound-guided injections for adults with mild to moderate cases: hyaluronic acid, corticosteroid, and 5% glucose. Sixty…

Researchers are testing whether a home-based program combining adapted exercise and mindfulness training can improve walking ability and sleep quality in people with rare neurodegenerative diseases such as leukodystrophy, ataxia, and adrenomyeloneuropathy. The study enrolls about…

Researchers are testing whether EEG neurofeedback can reduce chronic nerve pain in adults with type 1 or type 2 diabetes. The technique uses real-time brainwave imaging to help people learn to adjust their own brain activity. In this blinded, randomized trial, 60 participants rec…

Trigeminal neuralgia causes sudden, severe facial pain that can send people to the emergency department. This observational study at a Spanish hospital follows 20 adults who receive intravenous lacosamide, an anti-seizure drug, as a rescue treatment for a pain flare. Researchers …

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 an immersive virtual reality headset during physical therapy can improve satisfaction and recovery for patients in the intensive care unit. Ninety adult ICU patients will be randomly assigned to either traditional rehab or rehab with VR. The goal is…

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 …

Carpal tunnel syndrome happens when a nerve in the wrist gets squeezed, causing pain, numbness, and weakness in the hand. Open surgery has long been the standard fix, but a newer minimally invasive method uses ultrasound to guide the release through a much smaller opening. This s…

This study tests whether adding acupuncture to virtual exercise can help manage nerve pain caused by a specific bladder cancer treatment (EV-Pembro). About 50 adults with advanced bladder cancer will be split into two groups: one gets acupuncture plus virtual exercise, the other …

This study tests whether auriculotherapy (a type of ear acupuncture) can help manage pain after carpal tunnel surgery. Fifty adults will receive either real or sham auriculotherapy before surgery, and their pain levels will be tracked for three days afterward. The goal is to see …

This trial tests whether duloxetine, a drug commonly used for depression, can reduce the painful nerve damage that some cancer patients experience after receiving taxane chemotherapy. The study enrolls adults with solid tumors who have at least moderate pain from this side effect…

This study tests a device called SWAN that guides people through head movements to reduce motion sickness and improve balance. It is for adults with inner ear disorders, motion sickness, or those recovering from vestibular schwannoma surgery. Participants use the device at home f…

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

This study tests whether a type of physical therapy called explicit sensory retraining can reduce nerve pain and improve balance in people who have chemotherapy-induced peripheral neuropathy (CIPN). Participants receive either three weekly sessions of sensory retraining for the l…

This trial tests whether a steroid injection near the spine can reduce nerve pain in people with lumbosacral radiculopathy, a condition where a herniated disc presses on a nerve root. Researchers will measure pain using a standard scale before the injection and again at 3 weeks a…

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 trial tests whether trimetazidine, a drug already used for heart conditions, can reduce nerve pain and improve daily function in adults with diabetic peripheral neuropathy. Researchers will give 60 participants trimetazidine tablets twice daily for three months and track cha…

This study tests whether combining electro-acupuncture with a structured exercise program helps cancer survivors manage chemotherapy-induced peripheral neuropathy (CIPN) — nerve damage causing numbness, pain, and balance problems in hands and feet. Participants receive either ele…

This study tests a remote program called Move-MORE for people with sciatica (nerve pain from the lower back into the leg). The program combines mindfulness, gentle movement, and motivational coaching to help manage pain and improve daily function. Researchers will enroll 32 adult…

This trial tests whether a topical blend of essential oils can reduce the painful, tingling, or numb sensations caused by chemotherapy-induced neuropathy in breast cancer patients treated with paclitaxel. The study enrolls women with bothersome nerve symptoms that standard medica…

This study tests a minimally invasive procedure to release a trapped median nerve at the elbow (lacertus syndrome) using ultrasound guidance. Instead of a large cut, doctors make a tiny incision and cut the tight band pressing on the nerve. The goal is to reduce pain, improve han…

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 …

Diabetes patients often suffer from dry mouth, which affects eating, speaking, and quality of life. This study tests whether endocrinologists can deliver simple oral care—like screening, counseling, and saliva substitutes—during routine diabetes visits. Researchers will follow 80…

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

This trial tests whether taking Ceylon cinnamon supplements for six months can reduce pain and improve blood sugar, cholesterol, and body weight in adults with painful diabetic peripheral neuropathy. Participants are randomly assigned to receive either cinnamon capsules or a plac…

This study tests a new drug called (2R,6R)-hydroxynorketamine (HNK) for chronic nerve pain in the arms or legs. 25 adults will receive a single infusion of HNK, ketamine, or a placebo in random order, with 5 weeks between each. Researchers will measure pain levels, sleep, and dai…

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 trial tests a wearable-sensor system called MINDGAPS that tracks eye and body movements during balance exercises. Researchers want to see if it can improve dizziness and stability in older adults with vestibular hypofunction — a common cause of falls. The study also checks w…

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 phase 3 trial tests whether an extended-release (once-daily) form of pregabalin works as well as the standard immediate-release (twice-daily) form for relieving nerve pain caused by diabetic peripheral neuropathy or postherpetic neuralgia. About 348 adults aged 18 to 75 with…

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

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

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

This study tests whether a 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 wearing Boarding Ring glasses can help people with vestibular neuritis (a sudden, severe vertigo attack) recover their balance more quickly. Researchers will measure changes in balance and eye movements over one week in 51 adult patients. The glasses are …

This study tests whether daily symptom reporting via an app, website, or phone, plus follow-up calls from a nurse practitioner, can better manage chemotherapy-induced peripheral neuropathy (nerve damage). 422 adults who have completed certain chemotherapies will track symptoms fo…

This study tests whether a combination of sensory-motor exercises (like pelvic rocking and strength training) can reduce pain and improve balance in postmenopausal women with polyneuropathy. One hundred women with type 2 diabetes and numbness in their arms and legs will be random…

This phase 2 trial is testing whether cannabis-based medicines—THC, CBD, or a combination—can safely reduce chronic nerve pain in Veterans. 320 participants will receive one of these treatments or a placebo for six weeks. The goal is to see if these options offer better pain reli…

This study explores whether acupuncture can reduce nerve pain and numbness caused by chemotherapy in people with gynecologic, urologic, or rectal cancers. About 75 participants will be randomly assigned to receive real acupuncture, sham acupuncture, or usual care for 6 weeks. The…

This study tests a new brace that applies small, repeated forces to the wrist during sleep, aiming to relieve carpal tunnel syndrome symptoms. Researchers will compare it to a standard brace in 116 adults. The main goal is to see if the device reduces pain and improves hand funct…

This study tests whether a high-dose capsaicin patch (made from chili pepper extract) can reduce chronic tailbone pain (coccygodynia) better than a low-dose patch. About 188 adults with pain lasting over 3 months will receive either the 8% patch or a control patch applied once fo…

This study compares two treatments for mild to moderate carpal tunnel syndrome: osteopathic manipulation (hands-on therapy) and wearing a wrist brace at night. Ten adults diagnosed with carpal tunnel will be randomly assigned to one of the two groups. Researchers will use MRI sca…

This study tests whether immersive virtual reality (IVR) can reduce persistent shortness of breath in people with ALS who already use a breathing machine (non-invasive ventilation). 35 participants will try both a VR session based on medical hypnosis and a music therapy session. …

This study in Ukraine compares two minimally invasive procedures—cryoablation (freezing) and radiofrequency ablation (heating)—to treat nerve pain caused by neuromas from combat injuries. Fifty adults with moderate to severe pain will be randomly assigned to one treatment. The go…

This study looks at whether a surgery called RPNI can reduce nerve pain in people who have had a leg amputated. The surgery takes the cut nerve end and wraps it in a small piece of the patient's own muscle to help it heal. Researchers will follow 20 adults for 24 months to see if…

This study tests whether a simple breathing exercise device can help people with ALS maintain the strength of their inspiratory muscles—the muscles that pull air into the lungs. Over 44 participants will do resisted breathing exercises twice daily for 12 weeks, then twice weekly …

This study looks at whether repairing blocked leg blood vessels within 48 hours helps heal foot ulcers in people with type 2 diabetes faster than waiting for a scheduled procedure. About 200 adults with diabetic foot sores will be randomly assigned to early or standard treatment.…

This study tests two minimally invasive procedures—cryoablation (freezing) and radiofrequency ablation (heating)—to control severe chest pain after combat injuries. About 50 adults in Ukraine will be randomly assigned to one treatment, and researchers will measure pain levels, op…

This study tests whether a fun, game-based occupational therapy program can improve quality of life, daily skills, and emotional health in 20 teens with Duchenne muscular dystrophy. Participants will use a computer or tablet at home. Researchers will compare results to standard t…

This study tests a new, faster type of brain stimulation (called AiTBS) to see if it relieves nerve pain better than the standard method. 120 adults with long-term nerve pain will receive either AiTBS or standard stimulation over 10 days. Researchers will measure pain levels and …

This study tests whether early, intensive respiratory physiotherapy can improve cough strength in ICU patients with muscle weakness who are struggling to come off a ventilator. Fifty adults on breathing machines will receive either three daily sessions of specialized breathing ex…

This study looks at whether adding a specific pressure setting (PEP) to a standard cough-assist machine can help people with ALS cough more effectively. ALS weakens the muscles needed to cough, raising the risk of lung infections. The trial will compare cough strength with and wi…

This study tests whether a 12-week progressive resistance exercise program can reduce chronic pain in people with multiple sclerosis. 116 participants will be randomly assigned to either the exercise group or a waitlist control group. The goal is to see if exercise provides a mea…

This study tests whether a personalized physical therapy program, designed based on each patient's specific muscle strengths and weaknesses using a robotic leg press, can help people with ALS maintain physical function better than standard physical therapy. Thirty-eight participa…

This pilot study tests whether scrambler therapy, a device that uses mild electrical signals on the skin, can reduce nerve pain in people with corticobasal syndrome (CBS). Twenty-five adults with CBS and moderate-to-severe pain will be randomly assigned to receive either scramble…

This study tests whether taking ginger root extract daily for 8 weeks can reduce pain and improve gut health in people with chronic sciatica. Researchers will measure pain levels, gut bacteria, and brain changes in 80 adults. The goal is to see if ginger affects the gut-brain con…

This study tests a new home-based program that uses a special treadmill and virtual reality to help children with conditions like cerebral palsy, muscle diseases, or obesity improve their walking. About 30 children aged 6 to 17 will try the program at home for at least 3 sessions…

This study is testing whether a closed-loop spinal cord stimulator can reduce pain and improve quality of life in people with nerve damage from chemotherapy. The device automatically adjusts stimulation based on nerve responses. Twenty adults with persistent pain will be followed…

This study tests whether a non-invasive magnetic stimulation device (AtMS) can reduce nerve pain caused by injury or surgery in veterans and active-duty military. 180 participants will receive either active, fixed, or sham stimulation over 16 weeks. Researchers will measure pain,…

This study tests whether using a frozen glove plus a tight surgical glove on one hand works better than a frozen glove alone to prevent nerve damage caused by the chemotherapy drug paclitaxel. About 60 women with early-stage breast cancer will be enrolled, each acting as their ow…

This study looks at whether moving patients on breathing machines out of bed and into a chair helps them regain strength and function better than staying in bed. About 150 adults in the ICU will be randomly assigned to either chair sitting or bed rest. The goal is to see if chair…

This study tests whether a video-based home exercise program can improve trunk and limb control and daily activities in 20 people with ALS. Participants will follow the program 3 days a week for 8 weeks. The goal is to see if this approach can ease symptoms and improve quality of…

This study tests whether adding gentle electrical stimulation to physical therapy can help people who have had a severe stroke and developed muscle weakness in the ICU. About 24 participants will receive either standard therapy or therapy combined with functional electrical stimu…

This study tests whether injecting a botulinum toxin (similar to Botox) around nerves in the lower legs can safely reduce pain from diabetic neuropathy. About 80 adults with diabetes and chronic foot pain will receive either the active drug or a placebo injection every 12 weeks f…

This phase 3 trial is testing whether metformin, a common diabetes drug, can improve muscle function in adults with myotonic dystrophy type 1 (Steinert's disease). The study will enroll 142 participants who will receive either metformin or a placebo for 6 months. Researchers will…

This study tests whether deep brain stimulation (DBS) can safely reduce severe, long-lasting pain after a stroke. Thirty adults with pain that hasn't improved with other treatments will have a DBS device surgically implanted. Over 10 months, researchers will compare different sti…

This study tests whether adapted Argentine Tango dancing can reduce nerve damage symptoms caused by breast cancer chemotherapy. About 140 survivors who completed taxane-based chemo at least 3 months ago will take 16 dance sessions over 8 weeks. Researchers will measure changes in…

This study tests whether fenofibrate, a drug typically used for high cholesterol, can prevent or reduce nerve damage caused by a common chemotherapy regimen for multiple myeloma. The trial will enroll 44 newly diagnosed patients, half receiving fenofibrate alongside their standar…

This study tests a safe, home-use device that sends mild electrical current to the brain (tDCS) to help people with long-term pain who only got short-term relief from a similar hospital treatment. About 70 adults with chronic nerve or pain-sensitization pain will use the device 2…

This study tests whether a non-invasive brain stimulation technique called rTMS can reduce chronic nerve pain in people with cancer. Sixty participants will receive both real and sham (placebo) stimulation in a crossover design, with an 8-week break in between. The goal is to see…

This study tests whether adding a nerve block to local anesthesia reduces pain after carpal tunnel release surgery. 118 adults with carpal tunnel syndrome will be randomly assigned to receive either local anesthesia alone or local anesthesia plus a nerve block. Pain levels will b…

This study compares two hands-on techniques—SNAG (a gentle spinal glide) and sciatic nerve mobilization exercises—for easing non-specific sciatica pain. Thirty adults with pain lasting over 12 weeks will try both methods in a crossover design, with each session separated by 10 da…

This study follows 105 adults with chronic pain, nerve damage, or slow wound healing from cancer treatments. They receive ozone therapy as an add-on to their usual care. Researchers track changes in quality of life, anxiety, and depression to see if ozone therapy helps.

This study uses a special PET scan to measure tau protein buildup in the brains of people with frontotemporal lobar degeneration (FTLD), atypical Alzheimer's disease, and healthy volunteers. Researchers want to see if this scan can help tell these conditions apart. About 72 adult…

ALS is a rapidly progressive nerve disease with no cure, and extending breathing support into later stages raises hard questions about quality of life. Researchers in Norway are following 200 ALS patients, their partners and their children with questionnaires before and after lon…

Laminopathies and emerinopathies are rare disorders caused by mutations in the LMNA or EMD genes, often affecting skeletal muscles and the heart. Researchers at French hospitals are building a registry of 800 patients with confirmed mutations to record detailed genetic, neurologi…

Researchers are building a large database of cognitive, behavioral, lifestyle, and genetic information from people with neurodegenerative diseases such as Parkinson's disease, multiple system atrophy, and progressive supranuclear palsy. The goal is to find biomarkers that help do…

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 will compare two methods for locating the ulnar nerve in the wrist: using ultrasound imaging versus measuring physical landmarks. Researchers will enroll 40 healthy adults to see which technique is more reliable for placing stimulating electrodes. The goal is to improv…

Researchers are building a global registry to collect patient-reported surveys, genetic test results, and medical records from people with Charcot-Marie-Tooth disease and related inherited neuropathies. The study is open to children and adults with a confirmed or suspected diagno…

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

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

Researchers at Peking University First Hospital are building a prospective cohort of 2,000 people with plasma cell disorders, including multiple myeloma, amyloidosis, and related conditions. Participants give blood, bone marrow, and urine samples before and after treatment, and t…

This study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are already receiving olipudase alfa (Xenpozyme®) as part of their routine care. Researchers will monitor side effects and immune responses over 1 to 3 years. No new treatment …

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

This observational study monitors the long-term safety of eplontersen, a treatment for transthyretin amyloidosis, in patients who have had a liver transplant or have severe liver impairment. Researchers will track side effects, lab changes, and serious health events in about 320 …

This study gathers medical information from 200 adults with possible nerve, muscle, or autonomic nervous system disorders who are not already in other NIH studies. Participants undergo standard tests like nerve conduction studies, EMG, and physical exams. The data is stored anony…

This study follows 150 people aged 7 and older with RYR1-related muscle disorders for 3 to 5 years. Researchers will collect medical history, perform physical exams, and use questionnaires, activity trackers, and tests of heart, lung, and muscle function. The goal is to better un…

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

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

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

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

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

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

This study 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 follows 100 intensive care patients on ventilators to see how their thigh muscles recover. Researchers use a handheld strength tester and 3D ultrasound to measure muscle size and strength over time. The goal is to find better ways to predict and track recovery from ICU…

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

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

This study 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 trial tests a brain-computer interface called CortiCom, which is surgically placed on the brain's surface to record signals related to speech and arm movement. The goal is to see if these signals can be decoded to control software and devices, potentially helping people with…

This trial explores whether physical activity, specifically walking in different visual environments, can improve the sense of self in people with schizophrenia, borderline personality disorder, or vestibular syndromes. Participants will complete walking sessions in three environ…

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 will assess physical function in 80 people with chemotherapy-induced peripheral neuropathy (CIPN) using tests like balance, walking, and grip strength. Researchers will compare these objective measures with patients' own symptom reports to develop better ways to track …

This registry study follows adults with myasthenia gravis (MG) over time to understand how symptoms, daily functioning, and quality of life change. Participants complete surveys about their symptoms, treatments, and challenges, and may share medical records. The goal is to gather…

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

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

This study is building a large online registry of US adults who experience sinus, ear, dizziness, and headache symptoms, with or without a migraine diagnosis. Participants answer questions about their symptoms, how these affect daily life, and which treatments they have tried. Th…

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

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

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

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

This study tests whether a non-invasive electrical test on the quadriceps (thigh muscle) can help diagnose and monitor amyotrophic lateral sclerosis (ALS). Researchers will measure nerve signals traveling from the brain to the muscle in 30 people with suspected or confirmed ALS. …

This observational study follows about 200 people in the United States who have chronic inflammatory demyelinating polyneuropathy (CIDP) and still experience disability despite at least three months of standard treatment. Researchers will track changes in disability scores and ex…

This study is testing whether a non-invasive nerve monitoring device called TetraGraph can accurately detect early signs of ICU-acquired weakness. Researchers will compare readings from the device with standard needle-based electromyography (EMG) tests in healthy volunteers and I…

This study looks at 70 adults with sensory neuronopathy, a rare nerve condition that causes balance and coordination problems. Researchers will test several clinical scales over 12 months to see which one best tracks how the disease changes over time. The goal is to find a reliab…

This study creates a biobank to collect and store tissue samples—such as blood, muscle, saliva, urine, and stool—from people with neuromuscular diseases, their family members, and healthy volunteers. The samples will be shared with researchers to help them better understand these…

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

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

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

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

This study 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 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 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 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 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 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 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 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 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 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 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 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, 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 …

This study is collecting health data and blood samples from 100 people with specific types of hereditary spastic paraplegia (SPG4 and SPG5A). The goal is to create a shared database and biobank to better understand how the disease progresses. This information will help prepare fo…

This study collects health information and biological samples from up to 700 people under 30 with early-onset hereditary spastic paraplegia (HSP). Researchers aim to better understand how the disease progresses over time and create a registry for future studies. Participants prov…

This study looks at how changes in muscles and the brain are linked in aging and diseases like ALS, Alzheimer's, and Parkinson's. Researchers will compare healthy older adults with those who have these conditions to find early signs of disease. The goal is to better understand ri…

This study investigates whether serious muscle weakness, known to affect ICU patients, also occurs in people hospitalized outside the ICU. Researchers will follow about 600 adults in Italian hospitals, half with organ failure and half without, to see how often weakness develops a…

This study examines the role of the plantaris, a small calf muscle, in children having surgery for tight ankles (equinus). Researchers will randomly change the order of tendon cuts during surgery to measure how much each contributes to ankle movement. The goal is to understand if…

This study follows 75 adults with CMT1A and 25 healthy volunteers over five years to track how the disease progresses. Researchers will analyze blood and skin samples to find biomarkers that could make future clinical trials faster and more reliable. The goal is to better underst…

This study looks at how nerves work in people with Charcot-Marie-Tooth disease and other nerve disorders like CIDP. Researchers will use special electrical tests and ultrasound to measure nerve and muscle changes. The goal is to better understand these conditions, not to test a n…

This study looks at immune cells called macrophages in people with ALS, a disease that affects movement. Researchers will compare these cells between ALS patients, healthy people, and those with other motor problems. The goal is to find new targets for future treatments and bette…

This study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…

This study looks at how type 1 diabetes affects the nerves that control the heart in children. Researchers will measure heart rate variability in 100 children with diabetes and compare them to healthy kids of the same age and sex. The goal is to find early signs of cardiac dysaut…

This study tests a new training method for caregivers of infants with brachial plexus birth injury (a nerve injury that can cause arm weakness). The goal is to see if the training helps caregivers feel more confident and do daily stretching exercises more consistently, which may …

This study looks at whether quick, easy walking tests can replace the current complex and time-consuming exam used to track walking problems in people with facioscapulohumeral muscular dystrophy (FSH). Researchers will compare results from simple tests like the 6-minute walk with…

This study is collecting information from 4,000 adults with peripheral neuropathy (nerve damage) to better understand the condition. Researchers will combine standard tests with nerve ultrasound to see if it helps diagnose and track the disease. No new treatments are being tested…

This study looks at children and teens who had a limb removed before age 15-16 to see if they experience nerve pain (phantom limb or stump pain). Researchers will review medical records and use questionnaires during regular check-ups. The goal is to better understand how common t…

This study looks at the gut microbiome (the bacteria living in the digestive system) of people with POEMS syndrome, multiple myeloma, MGUS, AL amyloidosis, and healthy household members. Participants provide stool samples at home using a collection kit. The goal is to understand …

This study is testing different ways to measure muscle strength and movement in children with a rare muscle disease called FSHD. The goal is to find the best tests to use in future studies of new treatments. About 80 children aged 5 to 17 with confirmed FSHD will take part. The r…

This study is looking at whether a molecule in the blood called miR-1 can help doctors understand how muscle diseases like Duchenne muscular dystrophy and myotonic dystrophy are progressing. Researchers will compare miR-1 levels in 104 people, including patients with different mu…

This study aims to understand how upper-limb assistive devices—like grasping gloves, mealtime aids, and robotic arms—impact the daily lives of people with motor impairments. Researchers will interview and survey 150 regular users (including those with stroke, spinal cord injury, …

This study looks at whether tiny molecules in the blood, called microRNAs, can help predict how often and how severely cancer patients develop nerve damage from chemotherapy. Researchers will study 90 people receiving specific chemo drugs for lung or colon cancer. The goal is to …

This observational study is gathering data from 150 adults with muscle or joint pain to see how their answers on a pain questionnaire (PainDETECT) relate to where it hurts and their official diagnosis. Researchers hope to better understand when nerve pain is involved in common mu…

This study is gathering information from 1,500 people with cluster headache or trigeminal neuralgia to learn more about these painful conditions. Researchers will collect surveys, genetic samples, and other health data. The goal is to better understand what causes these headaches…

This study looks at whether giving ICU teams real-time feedback and extra nurse support helps them consistently use a set of proven care steps for patients on breathing machines. About 8,100 critically ill adults will take part across multiple ICUs. The goal is to find practical …

This study looks at how often people with cystic fibrosis and diabetes develop complications like eye, kidney, and nerve problems. Researchers will also check blood pressure and cholesterol. The goal is to better understand these risks in a diverse group of 200 adolescents and ad…

This study aims to create a national registry for people with Myotonic Dystrophy Type 1 (DM1) in Spain. Researchers will collect clinical data, genetic information, and patient reports from up to 3,000 participants. The goal is to better understand the disease and identify people…

This study is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…

This study uses surveys to learn about the care, needs, and quality of life of people with ALS who have the C9orf72 mutation, as well as their caregivers. Researchers want to find out what support is missing and how to improve daily life for both groups. About 208 participants wi…

This study follows 60 children with spinal muscular atrophy (SMA) who are already receiving SMN-restoring therapies like nusinersen or gene therapy. Over two years, researchers will track their muscle function, fatigue, breathing, and thinking skills to describe new disease patte…

This study is looking for 75 adults with myotonic dystrophy (a muscle disease) and healthy volunteers to test new muscle imaging techniques. The goal is to find better ways to measure muscle changes over time, which could help future treatment studies. Participants will undergo M…

This study uses a small button-like device worn on the thigh to measure how active people with neuromuscular diseases are over a week, every 6 months for 3 years. Participants also do a walking test and answer questionnaires. The goal is to better understand activity patterns, no…

This study collects information and samples from 1,000 people with or at risk for brain diseases like Alzheimer's, ALS, and frontotemporal degeneration. Researchers will track changes in thinking, language, and brain scans over time. The goal is to build a resource for future stu…

This study follows 30 people with Zellweger Spectrum Disorder over 5 years to understand how their vision changes over time. Participants will have yearly vision tests, physical exams, and blood work. The goal is to define the course of retinal degeneration and find the best ways…

This study looks at two types of spinal cord stimulation for people with long-term nerve pain after back surgery. One type causes a tingling sensation, while newer types do not. Researchers will use brain scans and pain questionnaires to figure out how each type works and which p…

This study looks at whether environmental factors like heavy metals from gold mining, past viral infections, or traditional plant remedies are linked to peripheral neuropathy (nerve damage) in French Guiana. Researchers will compare 78 people with and without nerve damage to see …

This study collects blood and optional spinal fluid samples from 350 people with Parkinson's, ALS, glioblastoma, certain cancers, or rheumatoid arthritis. The goal is to find new genetic markers that could lead to better diagnostic tests for autoimmune encephalitis and paraneopla…

This study is a registry that collects health information from people in Switzerland who have neuromuscular disorders like SMA, DMD, BMD, and others. It aims to track symptoms, treatments, and outcomes over time to help researchers and doctors improve care. No new treatments are …

This study looks at the experiences of 200 adults with spinal muscular atrophy (SMA) to understand how they were diagnosed and treated. Researchers want to find out why some patients are not receiving approved therapies, such as financial or access barriers. The goal is to improv…

This study tests a new way to read DNA that may find hidden carriers of spinal muscular atrophy (SMA). Some people carry two copies of the SMN1 gene side by side, making them carriers that standard tests miss. Researchers will take blood samples from 27 adults and use long-read D…

This study aims to find biological markers in the blood that can help measure whether a potential ALS therapy is working. Researchers will collect tiny particles shed by brain cells from 30 ALS patients and 30 healthy volunteers. By comparing these particles, they hope to identif…

This study is testing whether a special type of MRI, called resting-state fMRI, can predict how well people with optic neuritis (inflammation of the optic nerve) will recover their vision. Researchers will scan 200 patients and track their vision over 6 months. The goal is to see…

This study follows 100 adults who needed ECMO support for severe heart or lung failure. Researchers will track muscle size, strength, and quality of life over time to understand why some patients develop lasting weakness. No new treatments are being tested; the goal is to gather …

This study looks at whether tiny particles released by cells into the blood, called extracellular vesicles, can help detect a type of heart disease called ATTR amyloidosis earlier. The disease often goes unnoticed until serious damage occurs. Researchers will compare these partic…

This study checks if special sensors can reliably measure forces inside carbon fiber ankle-foot braces (orthoses). Researchers will test healthy volunteers and people with or without neuropathy who already use such braces. Participants will walk, sit, and stand while sensors reco…

This study follows 140 adults with ALS for up to 3 years, collecting spinal fluid and blood samples along with clinical and cognitive tests. The goal is to understand how certain biological markers relate to different forms of the disease and its progression. This is an observati…

This study looks at why a numbing injection in the thigh (adductor canal block) works so well for knee surgery pain. Researchers think the medicine may spread to the sciatic nerve, but they want to measure how often this happens. About 70 adults having knee surgery will be tested…

This study looks at whether sleep disorders make vertigo worse for people with Ménière's disease, a condition that causes hearing loss, ringing in the ears, and dizziness. Researchers will track 100 adults to see if those with more severe vertigo also have more sleep problems. Th…

This study uses special wearable devices to monitor how babies and children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) move at home. About 100 children will wear a sensor-filled jumpsuit or an ankle monitor to track their motor skills over up to 30 mo…

This study looks at the best way to treat nerve injuries in the fingers. It compares surgery with wearing a cast. About 166 adults with a cut nerve in one finger will be randomly assigned to one of the two treatments. The main goal is to see how well feeling returns after one yea…

This study is testing a new tool called CANoPy that predicts whether a cancer patient will develop long-term nerve pain. Researchers will follow 625 adults with breast, gynecological, colorectal, or lung cancer as they undergo treatments like chemotherapy or surgery. By collectin…

This study is collecting nerve samples and biofluids from 400 people with ALS or peripheral neuropathies to find biological markers for early diagnosis and disease tracking. By analyzing individual cells, researchers hope to identify molecular changes that happen before symptoms …

This study tests a brain implant that reads signals from the brain to help people who have lost the ability to speak fluently communicate through a computer just by trying to talk. It involves up to 2 participants with conditions like spinal cord injury or ALS. The main goal is t…

This study looks at whether using a swallow gel or a semi-solid vehicle can help people with neurological conditions swallow pills more easily and safely. Researchers will observe 500 participants, including patients with swallowing disorders and healthy volunteers, as they swall…

This study looks at whether a sleep breathing test done at home can help doctors check how well a nighttime breathing machine is working for people with neuromuscular diseases. The goal is to see if the test is practical and gives useful information to adjust the machine settings…

This study aims to find new biological markers for neurodegenerative diseases like Alzheimer's, Parkinson's, and ALS. Researchers will analyze genetic, protein, and metabolic data from 1,200 patients to improve diagnosis, predict disease course, and monitor treatment response. Th…

This study looks at how spinocerebellar ataxia (SCA) affects the eyes and vision. Researchers will examine 60 adults with SCA types 1, 2, 3, or 27B, including those with early symptoms and those who are not yet symptomatic. The goal is to measure vision changes and eye damage usi…

This study aims to improve how doctors assess movement in infants with spinal muscular atrophy (SMA), a severe muscle-weakening disease. Researchers will use sensors to measure babies' movements in a special bathtub, where water helps reduce gravity's effects. The goal is to crea…

This study is creating a large collection of blood, spinal fluid, and urine samples from 800 ALS patients and 200 healthy volunteers. Researchers will also track clinical changes and at-home measures over time. The goal is to provide a valuable resource for scientists worldwide t…

This observational study will follow 1,000 people with spinal muscular atrophy (SMA) in Spain over many years. Researchers will collect information on motor skills, breathing support, and feeding needs during routine clinic visits. The goal is to standardize how SMA data is gathe…

This 24-month observational study will follow up to 1000 people with certain types of muscular dystrophy (LGMD, DM2, and late-onset Pompe disease) aged 6-50. Researchers want to see if specific physical tests, like the North Star Assessment and a 100-meter walk, are good ways to …

This study will collect pupil measurements from 1,000 patients in the neuro-intensive care unit using a device called a pupillometer. The goal is to establish what normal pupil size, reactivity, and neurological pupil index values are for critically ill patients. By gathering thi…

This study is creating a national registry of 100 people with ALS in Thailand. Researchers will collect data on symptoms, genetics, and survival to better understand the disease. The goal is to build a foundation for future research and improve care for ALS patients.

This study looks at nerve-related problems like numbness, tingling, and pain in people who have been treated for oral cavity or throat cancer. Researchers will use simple tools like brushes and temperature tests to map these sensations. The goal is to better understand how common…