Popular Trials
Prostaglandin Inhibitor
Oral Ifetroban for Duchenne Muscular Dystrophy
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.
Behavioral Intervention
Sleep Intervention for Duchenne Muscular Dystrophy
This trial will study whether a sleep and circadian intervention can be consistently taught and implemented by parents into the home setting, with the goal of improving youth sleep.
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Trials for DMD Patients
Cell Therapy
Cell Therapy for Duchenne Muscular Dystrophy
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
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
Vesleteplirsen for Duchenne Muscular Dystrophy
This trial tests vesleteplirsen, a drug, for safety and tolerance. It involves participants from previous studies and new ones. The goal is to find the highest safe dose and monitor side effects.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
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
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.
Trials for DMD Positive Patients
Cell Therapy
Cell Therapy for Duchenne Muscular Dystrophy
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
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
Cell Therapy for Duchenne Muscular Dystrophy
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
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.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
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.
Gene Therapy
Gene Transfer Therapy for Duchenne Muscular Dystrophy
This trial tests a gene therapy that adds healthy genes to help boys with Duchenne Muscular Dystrophy (DMD) improve their muscle function. Gene therapy has been explored as a potential treatment for Duchenne Muscular Dystrophy (DMD) through various studies and trials.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
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.
Trials With No Placebo
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
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
Vesleteplirsen for Duchenne Muscular Dystrophy
This trial tests vesleteplirsen, a drug, for safety and tolerance. It involves participants from previous studies and new ones. The goal is to find the highest safe dose and monitor side effects.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
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
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
PF-06939926 Gene Therapy for Duchenne Muscular Dystrophy
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.
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Frequently Asked Questions
Introduction to duchenne muscular dystrophy
What are the top hospitals conducting duchenne muscular dystrophy research?
When it comes to advancing research and treatment options for Duchenne muscular dystrophy, several hospitals have emerged as leaders in this field. Nationwide Children's Hospital in Columbus is at the forefront with nine ongoing clinical trials focused on this devastating condition. Their dedication is evident through their impressive history of 38 completed trials since embarking on their first Duchenne muscular dystrophy study in 2009. Similarly, Children's Hospital Colorado in Aurora has made significant strides with six active trials and a track record of 17 previous studies, dating back to their initial trial in 2009.
In Little Rock, Arkansas Children's Hospital joins the ranks with six ongoing clinical trials for Duchenne muscular dystrophy. While they may have a relatively smaller number compared to others, it is worth noting that they only initiated their inaugural trial focusing on this condition recently,in2018.Nevertheless,it demonstrates profound commitment from both medical professionals and patients alike towards exploring new treatment avenues.
Another notable institution making remarkable contributions is Rare Disease Research, LLC located in Atlanta.They are currently conducting five active clinical trials dedicated to Duchenne muscular dystrophy while having previously accomplished fourteen investigations since commencing their pioneering workin2010.Finally,the University of Utah Hospital situatedin Salt Lake City also strives towards enhancing understandingofthis debilitating condition;withfiveongoingclinicaltestsinprogressandhaving conducted just six priorinvestigationsincefirstengaginginaDuchennemusculardystrophystudyin2016.
These hospitals exemplify the collective effort being made across various locations to combat Duchenne muscular dystrophy.Medical pioneers are diligently working toward finding innovative solutions and therapiesfor those affected by this rare disease.With each trial undertaken,solidifying knowledge abouttheconditionbrings us closer to unraveling its mysteriesand offering hope for improved quality of lifeamongstpatientsworldwide
Which are the best cities for duchenne muscular dystrophy clinical trials?
When it comes to duchenne muscular dystrophy clinical trials, several cities are leading the way in research and development. Los Angeles, California boasts 27 active trials focusing on treatments like Cohort 1, PF-06939926, and Pamrevlumab. Salt Lake City, Utah follows closely with 24 ongoing studies investigating interventions such as PF-06939926 and DYNE-251. Additionally, Durham, North carolina is conducting 12 active trials studying treatments like PF-06939926 and Viltolarsen for all participants. These cities offer individuals affected by duchenne muscular dystrophy access to cutting-edge clinical trials that hold promise for improved outcomes in their battle against this debilitating condition.
Which are the top treatments for duchenne muscular dystrophy being explored in clinical trials?
Clinical trials are shedding light on potential breakthroughs in the treatment of Duchenne muscular dystrophy. Among the top contenders, PF-06939926 has emerged with two active trials and a total of three all-time studies dedicated to this condition since its listing in 2018. Another promising option is pamrevlumab, currently being explored in two ongoing clinical trials, which brings the total number of duchenne muscular dystrophy trials to three since its introduction in 2016. Eteplirsen also continues to show promise with two active trials and a total of five all-time duchenne muscular dystrophy studies since it was first listed back in 2014. Similarly, delandistrogene moxeparvovec exhibits potential with two active and five all-time duchenne muscular dystrophy trials registered since its debut last year. As researchers strive for progress through these clinical endeavors, hope continues to flourish for individuals affected by this debilitating disorder.
What are the most recent clinical trials for duchenne muscular dystrophy?
Exciting progress is being made in the field of Duchenne muscular dystrophy with recent clinical trials offering new hope and potential treatments. One such trial, NS-089/NCNP-02, has entered Phase 2, showcasing promising results for individuals with this condition. Another notable study involves delandistrogene moxeparvovec in Phase 3, demonstrating its potential to be a game-changer for those affected by Duchenne muscular dystrophy. Additionally, ongoing Phase 1 and 2 trials are exploring the effectiveness of RGX-202 and AOC 1044-CS1 as potential treatment options. With these advancements on the horizon, individuals living with Duchenne muscular dystrophy can anticipate brighter prospects ahead.
What duchenne muscular dystrophy clinical trials were recently completed?
Recently, several clinical trials exploring potential treatments for Duchenne muscular dystrophy have reached significant milestones. In December 2021, PTC Therapeutics successfully completed a trial investigating the efficacy of Ataluren in addressing this debilitating condition. Similarly, NS Pharma concluded their study on Viltolarsen in July 2021. Although not as recent, Northwestern University completed a trial evaluating Prednisone's impact on Duchenne muscular dystrophy back in July 2019. These advancements signify important progress in the search for effective therapies against this devastating disease and offer hope to individuals affected by Duchenne muscular dystrophy.