Duchenne Muscular Dystrophy

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46 Duchenne Muscular Dystrophy Trials Near You

Power is an online platform that helps thousands of Duchenne Muscular Dystrophy 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
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Sex:Male

9 Participants Needed

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:4 - 17
Sex:Male

12 Participants Needed

This trial is testing a special powered leg brace called the Nomad powered KAFO. It aims to help people who have trouble walking due to injuries. The brace provides extra support and power to make walking easier and safer.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

36 Participants Needed

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

This trial tests the safety and effectiveness of GIVINOSTAT, a liquid medicine taken by mouth, in patients with Duchenne's muscular dystrophy who have used it before. The medicine aims to improve muscle function and slow down muscle damage.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:7+
Sex:Male

206 Participants Needed

This trial tests a robotic exoskeleton that helps children with walking difficulties due to conditions like cerebral palsy or spinal cord injury. The device can either assist or challenge their walking to strengthen muscles and improve movement. Children will use the exoskeleton in different settings to see if it improves their ability to walk. Robotic exoskeletons have been increasingly used in pediatric rehabilitation to assist children with cerebral palsy in improving their walking abilities.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:3 - 17

44 Participants Needed

Background: - Cerebral palsy (CP) is the most common motor disorder in children. CP often causes crouch gait, an abnormal way of walking. Knee crouch has many causes, so no single device or approach works best for everybody. This study s adjustable brace provides many types of walking assistance. Researchers will evaluate brace options to find the best solution for each participant, and whether one solution works best for the group. Objective: - To evaluate a new brace to improve crouch gait in children with CP. Eligibility: * Children 5 17 years old with CP. * Healthy volunteers 5 17 years old. Design: * All participants will be screened with medical history and physical exam. * Healthy volunteers will have 1 visit. They will do motion analysis, EMG, and EEG described below. * Participants with CP will have 6 visits. * Visit 1: \<TAB\>1. Motion analysis: Balls will be taped to participants skin. This helps cameras follow their movement. \<TAB\>2. EMG: Metal discs will be taped to participants skin. They measure electrical muscle activity. \<TAB\>3. Participants knee movement will be tested. \<TAB\>4. Participants will walk 50 meters. \<TAB\>5. Participants legs will be cast to make custom braces. * Visit 2: * Participants will wear their new braces and have them adjusted. * Steps 1 3 will be repeated. * EEG: Small metal discs will be placed on the participants scalp. They record brain waves. * Participants will have electrical stimulation of their knees and practice extending them. * Participants will take several walks with the braces in different settings. * Visits 3 5: participants will repeat the walking and some other steps from visit 2. * Visit 6 will repeat visit 2.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:5 - 100

85 Participants Needed

This trial is testing the safety and tolerability of an IV dose of AB-1003 in adults with a specific type of muscular dystrophy (LGMD2I/R9). The goal is to see if the treatment can help improve muscle function or slow down muscle damage.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 65

10 Participants Needed

This trial is testing a new gene therapy called fordadistrogene movaparvovec. It aims to see if the therapy is safe and effective over a long period. The treatment works by fixing or replacing problematic genes in the body.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:0+
Sex:Male

7 Participants Needed

This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. A total of approximately 22 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:4+
Sex:Male

23 Participants Needed

PIXI for Neurogenetic Disorders

Research Triangle Park, North Carolina
This trial tests a program called PIXI, which helps parents of infants with genetic disorders. The program educates parents about their baby's condition, coaches them on how to interact with their baby, and provides family support. The goal is to improve the baby's development and family well-being.
No Placebo Group

Trial Details

Trial Status:Enrolling By Invitation
Trial Phase:Unphased
Age:< 99

120 Participants Needed

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:2 - 3
Sex:Male

10 Participants Needed

This trial is testing eteplirsen, a medication for Duchenne Muscular Dystrophy (DMD). It targets patients with specific genetic mutations that can be treated by skipping exon 51. Eteplirsen helps the body make a protein needed for muscle function by bypassing a faulty part of the gene. Eteplirsen has been approved for treating DMD by skipping exon 51, allowing for the production of a functional dystrophin protein.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:4 - 13
Sex:Male

160 Participants Needed

This study is evaluating whether people with tetraplegia may be able to control a computer cursor and other assistive devices with their thoughts.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

27 Participants Needed

The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:2 - 4
Sex:Male

12 Participants Needed

VA research has been advancing a high-performance brain-computer interface (BCI) to improve independence for Veterans and others living with tetraplegia or the inability to speak resulting from amyotrophic lateral sclerosis, spinal cord injury or stoke. In this project, the investigators enhance deep learning neural network decoders and multi-state gesture decoding for increased accuracy and reliability and deploy them on a battery-powered mobile BCI device for independent use of computers and touch-enabled mobile devices at home. The accuracy and usability of the mobile iBCI will be evaluated with participants already enrolled separately in the investigational clinical trial of the BrainGate neural interface.
No Placebo Group

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Unphased

2 Participants Needed

Why Other Patients Applied

"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

"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 tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

ZS
Depression PatientAge: 51

"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

"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

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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
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Frequently Asked Questions

How much do Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy clinical trials?

Most recently, we added Brain-Computer Interface for Paralysis, INS1201 for Duchenne Muscular Dystrophy and Satralizumab for Duchenne Muscular Dystrophy to the Power online platform.

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Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
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