Popular Trials
Gene Therapy
PF-06939926 Gene Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1
Los Angeles, California
This trial is a safety and tolerability study of a new drug for Duchenne muscular dystrophy. The drug will be given to 22 people, some of whom will also take sirolimus. The trial will be conducted in two phases, with a review by an external data monitoring committee before progressing to the next phase.
Prostaglandin Inhibitor
Oral Ifetroban for Duchenne Muscular Dystrophy
Recruiting0 awardsPhase 2
Little Rock, Arkansas
This trial is testing a new therapeutic strategy for Duchenne muscular dystrophy (DMD), which is a devastating disease that leads to loss of ambulation, respiratory failure, and cardiomyopathy (CM). There is currently no cure for DMD, and this new therapeutic strategy aims to address this unmet medical need.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 3
London, Ontario
This trial tests a gene therapy treatment for boys aged 4 to 7 with muscle problems. The treatment aims to fix the genetic issues causing their muscle problems. Participants will receive the treatment and be monitored for safety and effectiveness. The treatment involves using a specific gene therapy, which has been researched for its potential to correct muscle issues in children with muscle problems.
Exon Skipping Agent
PGN-EDO51 for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 2
Fredericton, New Brunswick
This trial is being conducted to assess the safety and tolerability of giving multiple increasing doses of PGN-EDO51 through an IV to individuals with Duchenne muscular dystrophy. The trial will have
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Trials for Duchenne Muscular Dystrophy Patients
Cell Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 3
Sacramento, California
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.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1
Palo Alto, California
This 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.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
Recruiting2 awardsPhase 3
Atlanta, Georgia
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.
Monoclonal Antibodies
Canakinumab for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1 & 2
Washington, District of Columbia
This trial is testing canakinumab, a medication that reduces inflammation, in young boys with Duchenne Muscular Dystrophy (DMD) who have not been treated with steroids. The study aims to see if the medication is safe and effective by monitoring changes in blood markers over a short period. Canakinumab has been studied for various inflammatory conditions.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 2 & 3
Sacramento, California
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.
Trials for DMD Patients
Cell Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 3
Sacramento, California
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.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1
Palo Alto, California
This 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.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
Recruiting2 awardsPhase 3
Atlanta, Georgia
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.
Monoclonal Antibodies
Canakinumab for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1 & 2
Washington, District of Columbia
This trial is testing canakinumab, a medication that reduces inflammation, in young boys with Duchenne Muscular Dystrophy (DMD) who have not been treated with steroids. The study aims to see if the medication is safe and effective by monitoring changes in blood markers over a short period. Canakinumab has been studied for various inflammatory conditions.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 2 & 3
Sacramento, California
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.
Trials for DMD Positive Patients
Cell Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 3
Sacramento, California
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.
EDG-5506 for Duchenne Muscular Dystrophy
Recruiting0 awardsPhase 2
Atlanta, Georgia
This 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.
Phase 3 Trials
Cell Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 3
Sacramento, California
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.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
Recruiting2 awardsPhase 3
Atlanta, Georgia
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.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 2 & 3
Sacramento, California
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.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Recruiting2 awardsPhase 3
Durham, North Carolina
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.
Trials With No Placebo
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1
Palo Alto, California
This 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.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
Recruiting2 awardsPhase 3
Atlanta, Georgia
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.
Monoclonal Antibodies
Canakinumab for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1 & 2
Washington, District of Columbia
This trial is testing canakinumab, a medication that reduces inflammation, in young boys with Duchenne Muscular Dystrophy (DMD) who have not been treated with steroids. The study aims to see if the medication is safe and effective by monitoring changes in blood markers over a short period. Canakinumab has been studied for various inflammatory conditions.
Gene Therapy
Gene Therapy for Limb-Girdle Muscular Dystrophy
Recruiting1 awardPhase 1 & 2
Columbus, Ohio
This 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.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 2 & 3
Sacramento, California
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.
View More Related Trials
Frequently Asked Questions
Do I need insurance to participate in a trial?
Almost all clinical trials will cover the cost of the 'trial drug' — so no insurance is required for this. For trials where this trial drug is given alongside an already-approved medication, there may be a cost (which your insurance would normally cover).
Is there any support for travel costs?
Many of the teams running clinical trials will cover the cost of transportation to-and-from their care center.
Will I know what medication I am taking?
This depends on the specific study. If you're worried about receiving a placebo, you can actively filter out these trials using our search.
How long do clinical trials last?
Some trials will only require a single visit, while others will continue until your disease returns. It's fairly common for a trial to last somewhere between 1 and 6 months.
Do you verify all the trials on your website?
All of the trials listed on Power have been formally registered with the US Food and Drug Administration. Beyond this, some trials on Power have been formally 'verified' if the team behind the trial has completed an additional level of verification with our team.
How quickly will I hear back from a clinical trial?
Sadly, this response time can take anywhere from 6 hours to 2 weeks. We're working hard to speed up how quickly you hear back — in general, verified trials respond to patients within a few days.