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59 Muscular Dystrophy Trials
Power is an online platform that helps thousands of 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.
Gene Therapy for Limb-Girdle Muscular Dystrophy
Columbus, OhioThis is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:4+
Sex:All
17 Participants Needed
AOC 1020 for Facioscapulohumeral Muscular Dystrophy
Columbus, OhioA Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Age:16 - 70
Sex:All
84 Participants Needed
This trial is testing the safety of a medicine called SRP-6004, given through a vein, in people who can walk but have a specific type of muscular dystrophy (LGMD2B/R2).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:18 - 50
Sex:All
2 Participants Needed
Gene Therapy for Limb-Girdle Muscular Dystrophy
Columbus, OhioThe primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:4 - 50
Sex:All
6 Participants Needed
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:All
Sex:Male
400 Participants Needed
Casimersen + Golodirsen for Duchenne Muscular Dystrophy
Columbus, OhioThe main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:6 - 13
Sex:Male
228 Participants Needed
Ataluren for Duchenne Muscular Dystrophy
Columbus, OhioThis trial tests the safety and tolerance of ataluren in patients with a specific type of Duchenne Muscular Dystrophy who have been part of previous studies. Ataluren helps the body make important proteins that are usually missing due to genetic errors. The study includes these patients and their siblings who have completed earlier trial phases. Ataluren is the first approved drug for Duchenne muscular dystrophy (DMD) patients with premature stop codon mutations and has been conditionally approved in Europe.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:All
Sex:Male
270 Participants Needed
Gene Therapy for Duchenne Muscular Dystrophy
Columbus, OhioThe 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.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:All
Sex:Male
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Investigational Medication, Others
Must Be Taking:Corticosteroids
148 Participants Needed
Weekly Prednisolone for Duchenne Muscular Dystrophy
Columbus, OhioThis trial is testing if a lower dose of steroids given once a week is as effective as a higher dose for infants and young boys with Duchenne muscular dystrophy. The goal is to see if the lower dose can reduce muscle damage while causing fewer side effects, like weight gain. The study will measure improvements in motor skills over time. Prednisolone has been shown to improve strength in Duchenne dystrophy, but high doses are associated with significant side effects.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:1 - 30
Sex:Male
Key Eligibility Criteria
Disqualifiers:None
Must Not Be Taking:Glucocorticosteroids
26 Participants Needed
Gene Therapy for Duchenne Muscular Dystrophy
Columbus, OhioThis 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:4 - 11
Sex:Male
Key Eligibility Criteria
Disqualifiers:Gene Transfer Drugs, Exon 1-11, Others
Must Be Taking:Prednisone, Deflazacort
43 Participants Needed
DYNE-251 for Duchenne Muscular Dystrophy
Columbus, OhioThis trial tests DYNE-251, an IV drug, in people with Duchenne muscular dystrophy who can benefit from exon 51 skipping. The drug aims to help their muscles by increasing the dystrophin protein. It works by skipping a faulty part of their gene to produce more of this important protein. Eteplirsen (Exondys 51) was the first drug approved for exon 51 skipping in Duchenne muscular dystrophy.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:4 - 16
Sex:Male
Key Eligibility Criteria
Disqualifiers:Congestive Heart Failure, Major Surgery, Others
Must Be Taking:Glucocorticoids
88 Participants Needed
AOC 1044 for Duchenne Muscular Dystrophy
Columbus, OhioAOC 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.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:7 - 27
Sex:Male
35 Participants Needed
EDG-5506 for Duchenne Muscular Dystrophy
Columbus, OhioThe FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:6 - 17
Sex:Male
Key Eligibility Criteria
Disqualifiers:Low LVEF, Others
Must Be Taking:Corticosteroids
48 Participants Needed
EDG-5506 for Becker Muscular Dystrophy
Columbus, OhioEDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Age:All
Sex:Male
260 Participants Needed
AOC 1020 for Facioscapulohumeral Muscular Dystrophy
Columbus, OhioA Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:16 - 70
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, BMI >35, Bleeding Disorders, Others
Must Not Be Taking:Investigative Medications
90 Participants Needed
EDG-5506 for Duchenne Muscular Dystrophy
Columbus, OhioThis trial tests EDG-5506, a pill taken regularly, in children aged 4 to 9 with Duchenne muscular dystrophy. It aims to see if the medication is safe and can reduce muscle damage. The study includes both children who are and are not currently on corticosteroids.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:4 - 9
Sex:Male
76 Participants Needed
EDG-5506 for Becker Muscular Dystrophy
Columbus, OhioThis trial is testing a new drug called sevasemten to see if it can help people with Becker muscular dystrophy, a condition that weakens muscles. The study aims to find out if the drug is safe and effective in improving muscle function and reducing symptoms.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:12 - 50
Sex:Male
175 Participants Needed
Gene Therapy for Duchenne Muscular Dystrophy
Columbus, OhioThis trial uses a special virus to deliver genetic material to boys with Duchenne muscular dystrophy who have a specific gene issue. The treatment helps their bodies make a better version of an important muscle protein.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:6 - 13
Sex:Male
3 Participants Needed
SRD-001 for Duchenne Muscular Dystrophy
Columbus, OhioThis research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Abnormal Blood Pressure, Liver Issues, Others
Must Be Taking:Cardiac Medications, Glucocorticoids
12 Participants Needed
Gene Therapy for Duchenne Muscular Dystrophy
Columbus, OhioThis 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:4 - 8
Sex:Male
Key Eligibility Criteria
Disqualifiers:Cardiomyopathy, Significant Illness, Others
Must Be Taking:Corticosteroids
16 Participants Needed
Gene Therapy for Duchenne Muscular Dystrophy
Columbus, OhioThis trial is testing a new gene therapy called delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy (DMD). The therapy aims to insert a healthy gene into the body to help improve muscle function. The study will last several years to evaluate safety and effectiveness.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:2+
Sex:Male
55 Participants Needed
AOC 1044 for Duchenne Muscular Dystrophy
Columbus, OhioAOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.
Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.
Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:7 - 55
Sex:Male
64 Participants Needed
SRP-9004 for Limb-Girdle Muscular Dystrophy
Columbus, OhioThe primary objective of this study is to evaluate the safety of SRP-9004.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 1
Age:4+
Sex:All
4 Participants Needed
AOC 1001 for Myotonic Dystrophy
Columbus, OhioThis 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
Age:18 - 66
Sex:All
37 Participants Needed
Del-desiran for Myotonic Dystrophy
Columbus, OhioA Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:16 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Diabetes, Heart Failure, Others
Must Not Be Taking:Anti-myotonic Medications
150 Participants Needed
Screening Tool for Myeloid Cancer
Columbus, OhioThis MyeloMATCH Master Screening and Reassessment Protocol (MSRP) evaluates the use of a screening tool and specific laboratory tests to help improve participants' ability to register to clinical trials throughout the course of their myeloid cancer (acute myeloid leukemia or myelodysplastic syndrome) treatment. This study involves testing patients' bone marrow and blood for certain biomarkers. A biomarker (sometimes called a marker) is any molecule in the body that can be measured. Doctors look at markers to learn what is happening in the body. Knowing about certain markers can give doctors more information about what is driving the cancer and how to treat it. Testing patients' bone marrow and blood will show doctors if patients have markers that specific drugs can target. The marker testing in this study will let doctors know if they can match patients with a treatment study (myeloMATCH clinical trial) that tests treatment for the type of cancer they have or continue standard of care treatment with their doctor on the Tier Advancement Pathway (TAP).
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Prior Anti-cancer Therapy, Others
Must Not Be Taking:Cytarabine
2000 Participants Needed
Gene Therapy for Limb-Girdle Muscular Dystrophy
Columbus, OhioThis trial tests SRP-9003, a gene therapy, in patients with LGMD2E, a type of muscular dystrophy. The therapy uses a harmless virus to deliver a healthy gene into muscle cells to fix the genetic issue. SRP-9003 is a gene therapy that uses a harmless virus to deliver a healthy gene into muscle cells, a method previously demonstrated to be effective in treating muscular dystrophies.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:4 - 15
Sex:All
6 Participants Needed
This trial is testing a new drug, SEA-CD70, alone and with azacitidine, to see if it is safe and works for adults with certain blood cancers that haven't responded to other treatments. The study will determine the best dose and check for side effects. Azacitidine is a treatment that improves survival, reduces the need for transfusions, and lowers the risk of progression to acute myeloid leukemia in patients with higher risk myelodysplastic syndromes.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Stem Cell Transplant, CNS Leukemia, Others
Must Not Be Taking:CYP3A Inducers
178 Participants Needed
Treosulfan for Acute Myeloid Leukemia
Columbus, OhioThis study aim is to assess, if treosulfan pharmacokinetics are influenced by declined renal function and by race/ethnicity of patients. The study also aims to determine an appropriate safe dose of treosulfan, when patient's renal function is impaired. The participants of this study are undergoing allogenic hematopoietic stem cell transplantation for treatment of acute myeloid leukemia or myelodysplastic syndrome.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 80
Sex:All
Key Eligibility Criteria
Disqualifiers:Severe Renal, Pulmonary, Hepatic Impairment, Others
36 Participants Needed
T Cell Therapy for AML or MDS
Columbus, OhioThis Research study is being done to characterize the safety, tolerability, and preliminary antitumor activity of the NEXI-001 T cell product (a new experimental therapy), which contains populations of CD8+ T cells targeting multiple leukemia associated antigen peptides in patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) who have relapsed disease after an allogeneic hematopoietic cell transplant (HCT).
The study will enroll AML or MDS patients who have either Minimal Residual Disease (MRD) or relapsed disease after a human leukocyte antigen (HLA)-matched allogeneic HCT. Patients who have had an HLA-mismatched or haploidentical allogeneic HCT will not be eligible to participate in this study. Eligible patients for this study must also have ≥ 50% T-cell chimerism from the original donor at the time study entry.
The enrolled patients will undergo bridging therapy for the purposes of disease control while the NEXI-001 T cell product is being manufactured. Choice of bridging therapy administered will be per the Investigator's discretion, but is limited to acceptable agents as specified in the protocol. Bridging therapy will be administered prior to lymphodepleting (LD) therapy, with the last dose of the bridging therapy administered ≥ 14 days prior to initiation of LD therapy. Within 72 hours after completing LD therapy, patients will receive a single IV infusion of the NEXI-001 T cell product.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
22 Participants Needed
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Frequently Asked Questions
How much do 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 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 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 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 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 Muscular Dystrophy clinical trials ?
Most recently, we added Olutasidenib for Leukemia, SRP-9004 for Limb-Girdle Muscular Dystrophy and SRD-001 for Duchenne Muscular Dystrophy to the Power online platform.