Neuromuscular Disease

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25 Neuromuscular Disease Trials Near You

Power is an online platform that helps thousands of Neuromuscular Disease patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication

Taldefgrobep Alfa for Spinal Muscular Atrophy

Columbus, Ohio
This trial will test if taldefgrobep alfa can help people with SMA who are already on other treatments. The drug works by blocking a protein that stops muscle growth, potentially making muscles stronger and improving movement. Taldefgrobep alfa has shown promise in increasing muscle mass and function in SMA patients.
Stay on current meds
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:4 - 21

269 Participants Needed

Long-Term Apitegromab for Spinal Muscular Atrophy

Columbus, Ohio
This trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+

238 Participants Needed

AOC 1001 for Myotonic Dystrophy

Columbus, Ohio
This trial is testing a new medicine called AOC 1001 to see if it is safe and effective for adults with a muscle disease called Myotonic Dystrophy Type 1. The medicine is given through an IV, and researchers want to know if it helps muscles work better.

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2

37 Participants Needed

ABC008 for Inclusion Body Myositis

Columbus, Ohio
This trial is testing a new drug called ABC008 to help people with Inclusion Body Myositis, a muscle disease with few treatment options. The drug may work by affecting certain immune cells. Researchers will check if it improves muscle strength and quality of life.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:40+

231 Participants Needed

Anitocabtagene Autoleucel for Myasthenia Gravis

Columbus, Ohio
A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1

30 Participants Needed

Cell Therapy for Duchenne Muscular Dystrophy

Cincinnati, Ohio
This trial tests a cell therapy called CAP-1002 in boys and young men with Duchenne muscular dystrophy. The therapy involves giving special cells through an IV to help improve muscle function. The goal is to see if this treatment can repair or regenerate damaged muscles.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:10+
Sex:Male

104 Participants Needed

ssNPA Device for Upper Airway Obstruction

Ann Arbor, Michigan
The researchers are investigating if the Self-Supporting Nasopharyngeal Airway (ssNPA) device can be used in the treatment of obstructive sleep apnea in children with Hypotonic Upper Airway Obstruction (HUAO).
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:3 - 21

40 Participants Needed

CABA-201 for Myasthenia Gravis

Chicago, Illinois
RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

12 Participants Needed

AZD5492 for Lupus and Myositis

Hamilton, Ontario
The purpose of this study is to measure the safety, tolerability, PK, and PD of AZD5492 administered subcutaneously in adult participants with SLE or IIM. Study details include: • The study duration will be a minimum of 180 days in addition to the screening period. Additional follow-up visits may be required up to 12 months from study start. * Depending on the study part they are assigned to, participants will be administered AZD5492 once (Part 1) or twice (Part 2). * Study visits will occur at: Screening, Days 1-4, 8, 15, 22, 30, 60, 90, 120, 150, and 180 in Part 1, Screening, Days 1-4, 8-11, 15, 22, 29, 43, 60, 90, 120, 150, and 180 in Part 2.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65

50 Participants Needed

VX-670 for Myotonic Dystrophy

Winston-Salem, North Carolina
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 64

36 Participants Needed

PGN-EDO51 for Duchenne Muscular Dystrophy

Toronto, Ontario
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks). The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne Muscular Dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:6 - 16
Sex:Male

7 Participants Needed

EIM Testing for Neuromuscular Disorders

Bethesda, Maryland
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 muscle diseases, and compare that with information from other standard tests. First, they will test the device on healthy people. Then they will test people with a variety of neuromuscular diseases. Because the test is noninvasive and not painful, researchers will test both children and adults. Objectives: - To gain experience using the EIM muscle testing device. Eligibility: * Healthy volunteers at least 2 years old. * Individuals at least 2 years old who have neuromuscular disease. Design: * Participants will be screened with a medical history and physical exam. * Participants will have one 2-3 hour clinic visit. Researchers may request follow-up visits. * Participants will be tested with the EIM device. The device and small electrodes will be placed on their skin. An electric current will pass through the device, but the participants will not feel this. * Participants may have an ultrasound test. A gel will be put on their skin, and a device will be moved over the skin. * Participants may have a nerve test. Electrodes will be placed on their skin, and they will feel a small shock. * Participants may have a test where a thin needle is inserted in their muscle.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:2+

275 Participants Needed

Ischemic Conditioning for Stroke

Milwaukee, Wisconsin
Stroke survivors have compromised vascular function which may contribute to secondary stroke risk, cardiovascular disease, and may limit their exercise tolerance. No studies have examined how femoral blood flow responds to both passive leg movement, a measure of microvascular function, as well as active leg contractions, a measure of the hyperemic response to exercise. Leg muscles with a reduced blood flow response to movement could be associated with decreased neuromuscular function, such as leg strength and fatigue. Preliminary data showing a single bout of ischemic conditioning may improve vascular function and muscle activation in healthy adults and individuals post-stroke. Therefore, the investigators want to examine if ischemic conditioning will also improve the blood flow response to passive leg movements as well as during single leg active contractions.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

55 Participants Needed

Implantation of brain-computer interface for communication in ALS, quadriplegia, and Locked In Syndrome

Baltimore, Maryland
The CortiCom system consists of 510(k)-cleared components: platinum PMT subdural cortical electrode grids, a Blackrock Microsystems patient pedestal, and an external NeuroPort Neural Signal Processor. Up to two grids will be implanted in the brain, for a total channel count of up to 128 channels, for six months. In each participant, the grid(s) will be implanted over areas of cortex that encode speech and upper extremity movement.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:22 - 70

3 Participants Needed

PGN-EDODM1 for Myotonic Dystrophy

Richmond, Virginia
This trial is testing the safety and tolerability of a drug called PGN-EDODM1, given through an IV, in people with Myotonic Dystrophy Type 1 (DM1). The drug aims to address the underlying issues causing muscle problems in these patients. The study includes initial assessments followed by a period of receiving the drug and monitoring.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 60

32 Participants Needed

MuSK-CAART for Myasthenia Gravis

Chapel Hill, North Carolina
This trial is testing a new cell therapy called MuSK-CAART for patients with a severe muscle disease known as MuSK myasthenia gravis. These patients have harmful antibodies that attack their muscles. The therapy uses special cells to find and stop these bad antibodies, potentially leading to long-term relief from the disease. Rituximab has been used to treat MuSK myasthenia gravis, showing some success in patients who are refractory to standard treatments.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1

24 Participants Needed

Respiratory Monitoring Techniques for Neuromuscular Disease

Wilmington, Delaware
This study will clinically evaluate a newly developed respiratory monitor - pneuRIP. The pneuRIP uses Respiratory Inductance Plethysmography (RIP) bands to measure key breathing indices non-invasively. This study compares the pneuRIP to an existing Respitrace system (Carefusion, Yorba Linda CA). 10 normal children and 10 children with breathing difficulties will be monitored with both systems.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:11 - 17

20 Participants Needed

Mechanical Insufflation for ALS

Philadelphia, Pennsylvania
The goal of this clinical trial is to learn how doing mechanical insufflation (MI) using a mechanical insufflator-exsufflator (MI-E) device affects breathing in early amyotrophic lateral sclerosis (ALS). This will be a single-center, single-arm study of MI in 20 patients with ALS at Penn. Based on prior research, we believe that 6-months of MI may slow decline in cough strength, measured as peak cough flow (PCF). Participants will perform MI using a device designed for mechanical insufflation-exsufflation (MI-E) known as the BiWaze Cough system. The BiWaze Cough is used for mucus clearance . It is connected to tubing and mouthpiece (or mask). The device will use programmed pressure and timing settings. An insufflation includes inflating the lungs for a maximal size inhalation before exhaling. The daily routine for the device includes 5 sets of 5 insufflations twice daily. Researchers will compare how use of MI in early ALS affects peak cough flow compared to 20 subjects who did not use MI in early ALS.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

20 Participants Needed

Auto-EPAP for Airway Blockage

Columbia, South Carolina
To evaluate the safety and effectiveness of the Automatic Expiratory Positive Airway Pressure (Auto-EPAP) feature versus manual expiratory positive airway pressure (EPAP) in the Vivo 45 LS Ventilator.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

20 Participants Needed

Virtual Cycling for Parkinson's Disease

Newark, New Jersey
This study asks three questions about Persons with Parkinson Disease that use a bicycle for exercise. 1. Does the use of virtual reality increase the intensity and and enjoyment of the experience compared to bicycling without virtual reality? 2. Does the way in which the bicycling (interval compared to continous) is performed affect the experience? 3. How does the way the virtual reality is delivered (with goggles or projected on a screen) affect the experience?
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:45 - 75

60 Participants Needed

Why Other Patients Applied

"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

AG
Paralysis PatientAge: 50

"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

ID
Pancreatic Cancer PatientAge: 40

"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

HZ
Arthritis PatientAge: 78

"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

FF
ADHD PatientAge: 31

"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

WR
Obesity PatientAge: 58

VX-670 for Myotonic Dystrophy

Ottawa
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
No Placebo Group

Trial Details

Trial Status:Enrolling By Invitation
Trial Phase:Phase 2

36 Participants Needed

Darifenacin for ALS

Montréal, Quebec
Amyotrophic lateral sclerosis (ALS) is a progressive neurological disorder characterized by selective death of upper and lower motor neurons, which leads to severe disability and fatal outcomes. One of the major hallmarks of ALS is the denervation of neuromuscular junctions (NMJs), which is one of the earliest events seen in ALS patients and mouse models of ALS. Under healthy conditions, glial cells called Perisynaptic Schwann Cells (PSCs) have a key role in regulating the stability and maintenance of NMJs, but they only participate in NMJ repair once denervation occurs. Denervation and the subsequent decline in synaptic activity triggers a loss of muscarinic acetylcholine receptors (mAChRs) in the PSC, and the resulting decrease in mAChR-mediated gene expression drives the "repair mode" of the PSC. In assessing the NMJ under conditions of ALS, a scarcity of process extensions in PSCs was observed for months prior to disease onset in the superoxide dismutase 1 (SOD1) mouse model of ALS, indicating inadequate glial repair. Collectively, these preclinical findings support the hypothesis that dampening glial mAChRs will restore the anticipated "repair" response of PSCs in the NMJ. Hence, the use of a selective M3 muscarinic receptor antagonist, Darifenacin, as a disease-modifying therapeutic in familial and sporadic ALS could improve NMJ function, resulting in a beneficial impact on the autonomy and quality of life of ALS patients. The purpose of the current Phase 2 trial is therefore to test the safety, tolerability, and pharmacology of Darifenacin in patients with ALS. Specifically, 30 eligible subjects between 18 and 85 years of age will take 7.5 mg of darifenacin or placebo daily (by mouth) for two weeks followed by an increased dose of 15 mg for the next 22 weeks. The trial will evaluate the effects of this medication on several outcome measures including patient safety, physical and neurological function, muscle strength, depression levels, and NMJ innervation of patients with ALS. Detailed clinical assessments will be conducted at regular intervals throughout the study in order to achieve these objectives.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

30 Participants Needed

Pelvic Floor Muscle Training for Myotonic Dystrophy

Jonquière, Quebec
Myotonic dystrophy type 1 (DM1) is a neuromuscular disease characterized by multisystem manifestations. DM1 can affect the urinary system through the impact of the pelvic floor muscles (PFM). Urinary incontinence can occur in this situation and is often offset with compensatory measures without restoring the PFM function (e.g. sanitary pads). PFM training have already been shown to be effective in reducing or even eliminating urinary incontinence in the general population. However, no study has been the subject of this modality in people with DM1. Having recently shown that it is possible to gain strength with DM1, a strengthening protocol targeting PFM could prove effective in treating urinary incontinence. The objectives of this study are i) to assess the feasibility and acceptability of PFM training and ii) to investigate the effects of PFM training in women with DM1 with adult phenotype. A quasi-experimental study will be conducted with 12 women having a confirmed diagnosis of DM1 with urinary incontinence. Participants will follow a 12-week PFM training program, comprising weekly sessions with an experienced physiotherapist as well as a home exercise program. Outcomes measures will be assessed at baseline and at post-treatment and will include: feasibility and acceptability variables, frequency of urinary incontinence, urogynecological symptoms and their impact on quality of life, morphometry and function of PFM, and the perceived improvement following the treatments. This study has the potential to improve the management of urinary incontinence and support the implementation of pelvic floor rehabilitation services in this population.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Sex:Female

12 Participants Needed

Virtual Walking Therapy for Neuropathic Pain

College Station, Texas
The purpose of this study is to determine if playing a virtual reality walking game can help improve neuropathic pain in adults with incomplete spinal cord injury.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

48 Participants Needed

Stem Cell Therapy for Chronic and Acute Conditions

Culver City, California
This trial is testing stem cell therapy, which uses special cells to repair or replace damaged tissues. It targets patients with a range of acute and chronic conditions that may not respond well to traditional treatments. The therapy works by transforming stem cells into the needed cell types to improve organ and tissue function.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

5000 Participants Needed

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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

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Bask GillCEO at Power
Learn More About Trials
How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

Frequently Asked Questions

How much do Neuromuscular Disease clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Neuromuscular Disease clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Neuromuscular Disease trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Neuromuscular Disease is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Neuromuscular Disease medical study?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Neuromuscular Disease clinical trials?

Most recently, we added AZD5492 for Lupus and Myositis, Anitocabtagene Autoleucel for Myasthenia Gravis and VX-670 for Myotonic Dystrophy to the Power online platform.

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