Popular filter options for duchenne muscular dystrophy trials
DMD Clinical Trials
View 42 DMD medical studies.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
This trial looks at the safety and effects of an experimental gene therapy. Participants who have taken part in a previous Pfizer study are invited to take part and be monitored for 10 years. Participants will have 1 annual onsite visit and some remote visits with their doctor.
Exon Skipping Agent
DYNE-251 for Duchenne Muscular Dystrophy
This trial is testing a new drug to treat Duchenne Muscular Dystrophy. The drug will be given intravenously to participants and the safety, tolerability, and effectiveness of the drug will be monitored over the course of 24 weeks.
Cell Therapy
CAP-1002 for Duchenne Muscular Dystrophy
This trial is testing a cell therapy called CAP-1002 for Duchenne muscular dystrophy. Boys and young men with the disease will be given the therapy or a placebo every 3 months for a year, then will have the option of receiving the therapy for another year.
DMD Positive Clinical Trials
View 18 DMD positive medical studies.
Exon Skipping Agent
High-Dose Eteplirsen for Duchenne Muscular Dystrophy
This trial will test if eteplirsen is safe and effective in treating Duchenne Muscular Dystrophy. There will be two parts to the study. In the first part, around 10 people will test two different doses of the drug to see if it is safe and tolerable. In the second part, around 144 people will test different doses of the drug to see if it is effective.
Prostaglandin Inhibitor
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.
Dystrophin Positive Clinical Trials
View 12 dystrophin positive medical studies.
Exon Skipping Agent
DYNE-251 for Duchenne Muscular Dystrophy
This trial is testing a new drug to treat Duchenne Muscular Dystrophy. The drug will be given intravenously to participants and the safety, tolerability, and effectiveness of the drug will be monitored over the course of 24 weeks.
Gene Therapy
PF-06939926 for Duchenne Muscular Dystrophy
This trial will test whether gene therapy is safe and effective for boys with Duchenne Muscular Dystrophy. Two thirds of participants will get the therapy, while the other third will get a placebo. Those in the placebo group will have the opportunity to get the therapy at the beginning of the second year.
Phase 3 Duchenne Muscular Dystrophy Clinical Trials
View 45 phase 3 duchenne muscular dystrophy medical studies.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
This trial looks at the safety and effects of an experimental gene therapy. Participants who have taken part in a previous Pfizer study are invited to take part and be monitored for 10 years. Participants will have 1 annual onsite visit and some remote visits with their doctor.
Cell Therapy
CAP-1002 for Duchenne Muscular Dystrophy
This trial is testing a cell therapy called CAP-1002 for Duchenne muscular dystrophy. Boys and young men with the disease will be given the therapy or a placebo every 3 months for a year, then will have the option of receiving the therapy for another year.
Exon Skipping Agent
High-Dose Eteplirsen for Duchenne Muscular Dystrophy
This trial will test if eteplirsen is safe and effective in treating Duchenne Muscular Dystrophy. There will be two parts to the study. In the first part, around 10 people will test two different doses of the drug to see if it is safe and tolerable. In the second part, around 144 people will test different doses of the drug to see if it is effective.
Gene Therapy
Gene Transfer Therapy for Duchenne Muscular Dystrophy
This trial will test if gene transfer therapy is safe and effective in boys with Duchenne Muscular Dystrophy. It is a placebo-controlled study, which means that some participants will receive a placebo instead of the treatment. Those in the placebo group will be given the opportunity to receive gene transfer therapy at the beginning of the second year.
Gene Therapy
PF-06939926 for Duchenne Muscular Dystrophy
This trial will test whether gene therapy is safe and effective for boys with Duchenne Muscular Dystrophy. Two thirds of participants will get the therapy, while the other third will get a placebo. Those in the placebo group will have the opportunity to get the therapy at the beginning of the second year.
Gene Transfer Therapy
Gene Transfer Therapy for Duchenne Muscular Dystrophy
This trial studies a gene therapy to treat Duchenne Muscular Dystrophy in boys. It's a 2-part, randomized, double-blind, placebo-controlled study lasting 128 weeks. Participants may get IV SRP-9001.
Duchenne Muscular Dystrophy Clinical Trials With No Placebo
View 45 duchenne muscular dystrophy medical studies that do not have a placebo group.
Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
This trial looks at the safety and effects of an experimental gene therapy. Participants who have taken part in a previous Pfizer study are invited to take part and be monitored for 10 years. Participants will have 1 annual onsite visit and some remote visits with their doctor.
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.
Exon Skipping Agent
SRP-5051 for Duchenne Muscular Dystrophy
This trial will have 2 parts: 1) determining how much of the drug can be given safely, and 2) testing how well the drug works. People who participate in the first part may continue on to the second part.
View More Duchenne Muscular Dystrophy Trials
See another 16 many medical studies focused on duchenne muscular dystrophy.
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, 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 dystrophye 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.