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Duchenne Muscular Dystrophy Clinical Trials
A listing of 29 Duchenne Muscular Dystrophy clinical trials actively recruiting volunteers for paid trials and research studies in various therapeutic areas.
1 - 12 of 29
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There are currently 29 active clinical trials seeking participants for Duchenne Muscular Dystrophy research studies. The states with the highest number of trials for Duchenne Muscular Dystrophy participants are California, Ohio, Pennsylvania and Ontario.
Featured Trial
Paid Clinical Studies Nationwide
Recruiting
Nationwide clinical trials offered in your area. Some trials offering up to several thousand dollars in compensation for participation.
Featured Trial
Chronic Cough Clinical Study
Recruiting
We are evaluating an investigational treatment to see if it may help people dealing with chronic cough.
Eligible participants will receive study-related medical care at no cost. You may be compensated for study-related travel and time. Health insurance is not required. If you qualify, you may receive:
Payment up to $1500, which varies by study.
Eligible participants will receive study-related medical care at no cost. You may be compensated for study-related travel and time. Health insurance is not required. If you qualify, you may receive:
Payment up to $1500, which varies by study.
Conditions:
Cough
Chronic Cough
Asthma
Allergic Asthma
Sinusitis
Featured Trial
Transcranial Magnetic Stimulation (TMS) for Obsessive Compulsive Disorder (OCD)
Recruiting
Transcranial magnetic stimulation (TMS) is an FDA-approved, non-invasive form of brain stimulation for OCD. TMS has been shown to be an effective form of treatment in individuals with OCD. By using a form of TMS termed theta-burst stimulation (TBS), we hope that this will result in a more effective treatment by producing faster symptom reduction. We are trialing a novel form of accelerated TMS, where we will deliver ten 10-minute sessions per day, for 5 days. This study will compare the efficacy of TMS for OCD at two distinct brain regions. Participants will be randomized into one of two study groups, and receive stimulation at either the DMPFC or R-OFC. Both regions have been linked to OCD by research. Patients that don't respond to their study region will have the option to receive stimulation at the other region.
Conditions:
Obsessive-Compulsive Disorder
Obsessive Compulsive Disorder
Obsessive Compulsive Disorder (OCD)
Obsessive-Compulsive Disorder (OCD)
Obsessive-compulsive Disorders and Symptoms
Featured Trial
High Triglyceride Clinical Research Study
Recruiting
Living with high triglycerides? See if our clinical study is right for you.
Don’t let financial stress hold you back. All clinical study-related assessments, care, and the investigational study drug are available at no cost. You could also be eligible for compensation for travel.
Don’t let financial stress hold you back. All clinical study-related assessments, care, and the investigational study drug are available at no cost. You could also be eligible for compensation for travel.
Conditions:
High Triglycerides
High Triglyceride Level
Hypertriglyceridemia
Elevated Triglycerides
Triglycerides High
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Recruiting
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Gender:
MALE
Ages:
All
Trial Updated:
01/17/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +38 locations
Conditions: Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Recruiting
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 2 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. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
Gender:
MALE
Ages:
Between 4 years and 11 years
Trial Updated:
01/16/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +5 locations
Conditions: Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants with Duchenne Muscular Dystrophy (DMD)
Recruiting
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.
Gender:
MALE
Ages:
1 year and above
Trial Updated:
01/15/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +4 locations
Conditions: Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
Recruiting
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory participants with DMD age ≥ 8 to \< 18 years old receiving corticosteroid therapy.
Gender:
MALE
Ages:
Between 8 years and 17 years
Trial Updated:
01/02/2025
Locations: Corewell Health, Grand Rapids, Michigan +9 locations
Conditions: Duchenne Muscular Dystrophy
NS-050/NCNP-03 in Boys With DMD (Meteor50)
Recruiting
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
Gender:
MALE
Ages:
Between 4 years and 14 years
Trial Updated:
12/12/2024
Locations: Children's Hospital Los Angeles, Los Angeles, California +10 locations
Conditions: Duchenne Muscular Dystrophy
UTSW HP [13-C] Pyruvate Injection in HCM
Recruiting
The study objective is to identify the earliest changes in energy substrate metabolism in patients with cardiomyopathies (CMP). To achieve this objective, we plan first to test the hypothesis that patients with CMP present focal alterations in myocardial hyperpolarized \[1-13C\]pyruvate flux.
Gender:
ALL
Ages:
Between 18 years and 60 years
Trial Updated:
12/11/2024
Locations: UT Southwestern Medical Center - Advanced Imaging Research Center, Dallas, Texas
Conditions: Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis, Becker Muscular Dystrophy, Heart Failure With Preserved Ejection Fraction, Heart Failure With Reduced Ejection Fraction
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy.
Recruiting
The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term safety and quality of life.
Gender:
MALE
Ages:
2 years and above
Trial Updated:
12/10/2024
Locations: Arkansas Childrens Hospital, Little Rock, Arkansas +5 locations
Conditions: Duchenne Muscular Dystrophy
An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
Recruiting
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.
Gender:
MALE
Ages:
4 years and above
Trial Updated:
11/27/2024
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +12 locations
Conditions: Duchenne Muscular Dystrophy
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Recruiting
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via elect... Read More
Gender:
ALL
Ages:
All
Trial Updated:
11/12/2024
Locations: xCures, Los Altos, California
Conditions: Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Recruiting
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.
The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
Gender:
MALE
Ages:
Between 4 years and 14 years
Trial Updated:
10/30/2024
Locations: Children's Hospital Colorado, Aurora, Colorado +8 locations
Conditions: Duchenne Muscular Dystrophy
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
Recruiting
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 58 weeks.
Gender:
MALE
Ages:
Between 4 years and 8 years
Trial Updated:
10/15/2024
Locations: Washington University School of Medicine in St. Louis, Saint Louis, Missouri
Conditions: Duchenne Muscular Dystrophy
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants with Mutations Amenable to Exon44 Skipping
Recruiting
AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
Gender:
MALE
Ages:
Between 7 years and 27 years
Trial Updated:
09/20/2024
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +9 locations
Conditions: DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
1 - 12 of 29