Clinical Trials for Neuromuscular Disorders

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Neuromuscular Disorders from the ClinicalTrials.gov database.

Trial Summary

• Total studies: 50
• Recruiting: 25
• Active, not recruiting: 8
• Completed: 10
• Other: 7

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

NCT00004568 — Study of Inherited Neurological Disorders

This study is designed to learn more about the natural history of inherited neurological disorders and the role of heredity in their development. It will examine the genetics, symptoms, disease progression, treatment, and psychological and behavioral impact of diseases in the following categories: hereditary peripheral neuropathies; hereditary myopathies; muscular dystrophies; hereditary motor …

NCT00059748 — Studies of the Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases Including Juvenile Dermatomyositis

Purpose:

The purpose of this protocol is 1. To comprehensively evaluate patients with autoinflammatory diseases clinically, genetically and immunologically at the autoinflammatory disease clinic at the NIH. 2. To follow patients with autoinflammatory Diseases that are genetically defined including Neonatal-Onset Multisystem Inflammatory Disease (NOMID), the most severe clinical phenotype of Cr…

NCT01900132 — Electrical Impedance Myography: Natural History Studies inNeuromuscular Disorders and Healthy Volunteers

Background:

- Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorders. EIM looks at how a mild, painless electrical current travels through muscles. Researchers want to gain experience in using the EIM device. They will collect information on the results of using it on people with and without nerve and mu…

NCT02089789 — Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation

Background:

- Proteins, fats, and other molecules are the body s building blocks. Many of these molecules must have sugars, or chains of sugars, attached to work properly. People with congenital disorders of glycosylation (CDGs) cannot attach these sugars or sugar chains properly. A child or adult with a CDG can have symptoms in different parts of the body, including brain, nerves, muscles, l…

NCT02390752 — Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor …

Background:

- Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing.

Objectives:

- To find the highest safe dose and side effects of TURALIO(R). To see if it helps treat certain types of cancer.

Eligibility:

- People ages 3-35 with a…

NCT05067179 — Analysis of Human ALS Tissues and Registry of ALS Patients

Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig’s Disease, is a progressive, terminal condition of muscle weakness that is associated with degeneration of neurons in the spinal cord and brain. This devastating disorder afflicts people in the prime of their lives. At the present time, there are no cures for this disorder, and current treatments are marginal at best. Despite …

NCT04222413 — Metarrestin (ML-246) in Subjects With Metastatic Solid Tumors

Background:

Metastasis is the spread of cancer from one organ to a nonadjacent organ. It causes 90% of cancer deaths. No treatment specifically prevents or reduces metastasis. Researchers hope a new drug can help. It stops cancer cells from growing and spreading further and possibly shrink cancer lesions in distant organs.

Objective:

To find a safe dose of metarrestin and to see if this dose…

NCT05518188 — Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Sec…

NCT06154252 — RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

NCT07690449 — Three CT Signs of Nerve-root Suffering in Low-back Sciatica: a Study of Their Frequency in Patients Referred for CT-guided Nerve-root Infiltration.

Retrospective, observational, single-centre study conducted at Nice University Hospital (Pasteur 2), Department of Diagnostic and Interventional Radiology. Objective: to assess the prevalence of three CT signs sought next to the clinically suspected nerve root in patients with low-back sciatica referred for CT-guided foraminal/peri-radicular infiltration: (1) nerve-root size asymmetry/hypertrop…

NCT06138639 — A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to < 7 years of age. Cohort 2 will include participants 7 to < 12 years of age. Cohort 3 will include particip…

NCT07689214 — Normast3 for Adults With Diabetic Polyneuropathy

The goal of this clinical trial is to find out if a new supplement called Normast® 3 can help reduce nerve pain and improve nerve function in adults with diabetic polyneuropathy (DPN). DPN is a common complication of diabetes that damages nerves, often causing burning, tingling, or stabbing pain, as well as problems with the autonomic nervous system, which controls things like sweating and hear…

NCT06193889 — KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis

A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis

NCT06743490 — Capturing Key MG-symptoms Using Smartphone Recordings.

This study will make use of a cross-sectional design of MG patients and non-MG participants to quantitatively assess key MG symptoms, and to explore the applicability of machine learning algorithms to their measurement.

NCT07017946 — Intestinal Microbiome Transplant in ALS

This is a 24-week study of intestinal microbiome transplant in people with ALS. All participants will be evaluated clinically in person for 4 visits. Blood and mailed/ fresh stool samples will also be collected. Blood samples will be used to determine changes in neurofilament light chain over time. Stool samples will be processed for microbiome analysis. Participants will have phone visits to f…

NCT07322003 — Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is:

Does pridopidine slow disease progression of ALS?

Researchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine wo…

NCT07689331 — Hungarian National Systemic Amyloidosis Registry

This registry is an observational, multicenter, retrospective and prospective, non-pharmacological study designed to collect and analyze data from patients with systemic amyloidosis treated in inpatient and outpatient cardiology, hematology, nephrology, and neurology departments across Hungary. The registry was established in 2025.

NCT07540221 — A Study to Evaluate the Pharmacokinetics and Safety of XEMBIFY Versus Gamunex-C in Participants With Chronic Inflammatory Demyelinating Polyradiculoneuropathy

The main goal of the study is to find out whether XEMBIFY, given once a week under the skin, provides similar levels of immunoglobulin G in the blood over time as Gamunex-C, which is given into a vein once every 3 weeks in people with CIDP.

Participants with CIDP will first have up to 28 days of screening to make sure they can join the study. Those who qualify will then start a 19-week treatme…

NCT07486934 — Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

NCT07451028 — Effect of an Oral Neuromuscular Training Device on Dysphagia

Dysphagia is a frequent complication following stroke, with an estimated prevalence of 42%. One training modality which have been implemented in both hospitals and municipalities for alleviating dysphagia are oral neuromuscular training devices. These devices are hand-held acrylic devices which are inserted between the lips and teeth by the patient or an assistant and pulled forward against lip…

NCT07687316 — Mental Effort During Low Load Resistance Training in Older Adults

The goal of this clinical trial is to determine the effect of maximal mental effort combined with low-intensity resistance training on strength and neuromuscular function in older adults. The main questions it aims to answer are:

• Does low-intensity resistance training in combination with maximal mental effort increase strength more than low intensity resistance training alone?
• Does low-int…

NCT07647510 — A Phase 3 Study to Evaluate Claseprubart in Adults With Generalized Myasthenia Gravis (EMERGE)

The purpose of this Phase 3 study is to demonstrate the efficacy, safety, and tolerability of claseprubart in participants with generalized myasthenia gravis (gMG).

NCT07660913 — Effectiveness of Matrix Rhythm Therapy on Carpal Tunnel Syndrome

The purpose of this study is to investigate the effects of matrix rhythm therapy, applied in addition to conventional treatment, on symptom severity, function, grip and pinch strength, and quality of life in patients with carpal tunnel syndrome.

NCT07111065 — FAST for DM - Fatty Acid Supplementation Trial (FAST) for Dermatomyositis (DM)

Dermatomyositis (DM) is a rare autoimmune disease that causes muscle weakness, skin rashes, and other symptoms. Researchers think both genetic and environmental factors play a role in this disease. They want to find out more about how diet and lifestyle choices affect people with DM/JDM.

Objective:

To see if omega-3 fatty acid supplements from fish oil, combined with a healthy diet, can help …

NCT07521930 — Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility for more details) to control and receive feedback from assistive devices.

Active, Not Recruiting

NCT00924196 — Natural History Study of Patients With Neurofibromatosis Type I

Background:

Neurofibromatosis Type 1 (NF1) is a genetic disorder in which patients are at increased risk of developing tumors (usually non-cancerous) of the central and peripheral nervous system. The disease affects essentially every organ system.

The natural course of NFI over time is poorly understood. For most patients the only treatment option is surgery. A better understanding of NF1 may…

NCT04535479 — Dry Needling for Spasticity in Stroke

The study team is recruiting 20 adults with spasticity due to chronic stroke and 20 adults with no neurological injuries for a 2 day study. In people with chronic stroke, one of the most common and disabling problems is spasticity (increased muscle tone or muscle stiffness). The purpose of this research study is to examine effects of dry needling on the nervous system (pathways between the musc…

NCT05070858 — A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

This study is researching the experimental drugs called pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized Myasthenia Gravis (gMG). Myasthenia gravis is a disease that causes weakness and fatigue in muscles in the body because the nerves and muscles are not communicating properly.

The aim of the study is to see how effective pozelimab and ce…

NCT04906746 — Ruxolitinib for Cancer Cachexia

To assess toxicity with use of Ruxolitinib in NSCLC cachexia patients; to associate levels of JAK/STAT signaling in blood, adipose, and muscle pre- and post-ruxolitinib treatment with changes in cachexia and anorexia.

NCT05357469 — Innovative Treatment of Chemotherapy-Induced Painful Peripheral Neuropathy in Adolescents and Young Adults With Cancer: A Two Arm Pilot Study

To evaluate the efficacy and safety of Scrambler therapy (ST) for chemotherapy-induced painful peripheral neuropathy (CIPN), and the impact of ST on physical functioning and quality of life (QoL) in adolescents and young adults (AYA) cancer patients.

In this proposed study, we will conduct a two-arm prospective, randomized wait-list controlled clinical trial to investigate the effectiveness of…

NCT07689903 — The Study, Conducted With 54 Individuals Over the Age of 65, Aimed to Reveal the Advantages and Disadvantages of a Combined Exercise Program Compared to Otago Exercise Alone.

Falls are a leading cause of morbidity and mortality in older adults.

Approximately one-third of individuals over 65 years of age fall each year, and 10-15% of these falls result in serious injury.

Balance disorders, decreased muscle strength, prolonged reaction time, and decreased proprioception are key factors that increase the risk of falls.

One evidence-based intervention to reduce the r…

NCT06151600 — A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J

This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J. The study will enroll 20 subjects of any age into a uniform protocol for follow-up and evaluations. Subject visits will occur every 12 months + 4 weeks for up to 2 years.

NCT06659627 — Interventions Against Fatigue in Patients With Myasthenia Gravis

A prospective assessor-blinded randomized clinical trial investigating the effect of aerobic exercise therapy or cognitive behavioural therapy on fatigue in patients with myasthenia gravis.

Not Yet Recruiting

NCT07691359 — Osteoporotic Fracture and Lidocaine Plaster 5% for Neuropathic Pain preventioN

Osteoporotic vertebral fractures may lead to chronic pain with a neuropathic component, which is often underdiagnosed and undertreated in older adults. This proof-of-concept randomized controlled trial aims to evaluate whether early application of a 5% lidocaine plaster can prevent the development of neuropathic pain following an osteoporotic vertebral fracture. Participants will be randomized …

NCT07688239 — The BANYAN Trial, an ALS MyMatch Trial Evaluating Safety, Biomarker Activity, and Microglial Activation of Nasal Foralumab

The goal of this clinical trial is to learn whether a nasal spray medicine called foralumab is safe and easy to tolerate in adults with amyotrophic lateral sclerosis (ALS). ALS is a disease that slowly damages the nerve cells that control movement, causing muscles to grow weaker over time. Foralumab is being studied as a possible way to calm the inflammation and immune system activity that are …

NCT07690150 — Effect of Knee Position on Muscle Loss During Leg Immobilization

This study looks at what happens to leg muscles when the knee is kept straight or bent during five days of wearing a brace. We want to find out if keeping the knee bent (so the thigh muscle is stretched) helps prevent muscle loss compared to keeping the knee straight. Thirty healthy adults will take part. They will wear a knee brace for five days and have several tests before and after, includi…

NCT07689994 — Evaluation of the Effect of a Seaweed Flour- and Leucine-Enriched Food on Metabolic and Inflammatory Status in Older Adults With Probable Sarcopenia or Sarcopenia

Sarcopenia and probable sarcopenia are associated with loss of muscle strength, functional impairment, frailty, and an increased risk of adverse outcomes in older adults. In short-term interventions, structural or functional changes may be modest, whereas certain serum biomarkers of metabolic-nutritional response may be more sensitive. In this protocol version, serum transthyretin (TTR) has bee…

NCT02478450 — Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects with ALS. Following an initial cohort receiving cell transplants unilaterally in the lumbar spinal cord, subsequent cohorts will receive escalating doses transplanted un…

Enrolling by Invitation

NCT07688863 — Comparative Effects of Different Inspiratory Muscle Training Modalities in Patients With Heart Failure.

The purpose of this study is to compare the effects of three different modalities of inspiratory muscle training (IMT) in patients diagnosed with chronic heart failure who exhibit reduced or mid-range left ventricular ejection fraction (LVEF < 50%). Patients will be recruited from cardiac rehabilitation programs and must be clinically stable before entering the protocol.

The study has a total…

Completed Trials

NCT00111384 — Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)

This study may identify genes that predict the seriousness of neurofibromatosis type 1 (NF1). Finding these genes may explain why some people with NF1 have more medical problems than others. The study will also examine medical problems in NF1 that are rarely seen and are not well understood.

Male and female patients with NF1 who have gone through puberty may be eligible for this study, as well…

NCT00314119 — Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1

This study will explore the growth of dermal neurofibromas (skin tumors) in patients with neurofibromatosis type 1 (NF1). Investigators will try to learn: 1) how fast (or slow) these benign tumors grow in NF1, 2) how often new tumors appear and 3) what genes are involved in the growth of the tumors.

Men and women between 20 and 50 years of age diagnosed with NF1 and their biological parents ar…

NCT01925196 — Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation

Background:

- Some people have a mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). The mutation causes a small piece of DNA to repeat itself thousands of times. The C9ORF gene mutation mostly occurs in families. In those families, some persons have ALS and others have FTD. Occasionally the C9ORF gene mutation occurs in persons witho…

NCT01734369 — Environmental Risk Factors for Myositis in Military Personnel

Background:

• Myositis is a rare disease in which the body s immune cells attack the muscle tissue. It can cause muscle weakness, swelling, and pain. It can develop in people with no history of muscle problems. Environmental exposures may determine who develops myositis. Genes may also affect development of the disease.
• Some people who serve in the military develop myositis. However, other m…

NCT04868123 — Effects of TENS and Mindfulness Meditation in Persons With HIV-related Neuropathy

This study is to explore the effects of transcutaneous nerve stimulation (TENS) and mindfulness meditation in persons living with HIV (PLHIV) and painful neuropathy in the feet.

NCT05237050 — Evaluation of Sound Therapy in a Population of Women With Fibromyalgia Aged Between 30 and 60 Years

This study focusing on sound therapy in patients with fibromyalgia is a single-centre, prospective, randomized study which evaluates the improvement or not of painful symptoms following relaxation sessions with sound therapy.

NCT07690722 — Effect of Whirlpool Therapy Combined With Botulinum Toxin in Children With Cerebral Palsy

This study aims to investigate the effects of adding whirlpool therapy to standard rehabilitation in children with cerebral palsy who recently received botulinum toxin-A injections. Participants will be divided into two groups: one group will receive standard physiotherapy, while the other group will receive physiotherapy combined with whirlpool therapy. The effects of these interventions on sp…

NCT07689916 — Machine Learning-Based Risk Identification and Precision Exercise for Common Running Injuries

This study aims to identify biomechanical risk factors associated with common running-related injuries in recreational runners using machine learning analysis. It also aims to evaluate whether a precision exercise intervention based on these risk factors can improve injury-related biomechanical and kinematic outcomes.

The main questions it aims to answer are:

Which biomechanical features iden…

NCT07689487 — Effects of Cyclical Blood Flow Restriction Exercise on Muscle Hypertrophy and Capillaries in Older Adults

The purpose of this study was to learn if low-load resistance exercise combined with cyclical blood flow restriction (BFR) could improve small blood vessel structure and muscle size in older adults.

Skeletal muscle thinning and the loss of tiny blood vessels (capillaries) commonly happen as people age. While lifting heavy weights can help stop this, heavy loads are often not suitable for older…

NCT07335822 — The Effectiveness of Low-Level Laser Therapy in the Treatment of Meralgia Paresthetica

Patients diagnosed with meralgia paresthetica via electromyography (EMG), who present with neuropathic complaints on the lateral thigh to the Physical Medicine and Rehabilitation outpatient clinics of our hospital, will be included in the study after reviewing exclusion criteria. Patients will be randomized into two equal groups using a sealed-envelope system. The treatment group will receive l…

Other (Terminated)

NCT05891847 — Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia

The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient’s parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia