Muscular Dystrophy Clinical Trials
Here are the 6 most popular medical studies for muscular dystrophy
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.
Exon Skipping Agent
Vesleteplirsen 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.
Popular filter options for muscular dystrophy trials
Duchenne Muscular Dystrophy Clinical Trials
View 49 Duchenne Muscular Dystrophy medical studies.
Cell Therapy
Cell Therapy 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 DMD
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
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.
DMD Clinical Trials
View 49 DMD medical studies.
Cell Therapy
Cell Therapy 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 DMD
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
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.
DMD Positive Clinical Trials
View 7 DMD positive medical studies.
Cell Therapy
Cell Therapy 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.
Phase 3 Muscular Dystrophy Clinical Trials
View 82 phase 3 muscular dystrophy medical studies.
Cell Therapy
Cell Therapy 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 DMD
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
Gene Therapy 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.
Muscular Dystrophy Clinical Trials With No Placebo
View 82 muscular dystrophy medical studies that do not have a placebo group.
Exon Skipping Agent
High-Dose Eteplirsen for DMD
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
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.
View More Muscular Dystrophy Trials
See another 54 medical studies focused on muscular dystrophy.
Frequently Asked Questions
Introduction to muscular dystrophy
What are the top hospitals conducting muscular dystrophy research?
In the quest to find effective treatments for muscular dystrophy, several leading hospitals are paving the way through their cutting-edge clinical trials. Nationwide Children's Hospital in Columbus takes the forefront with an impressive 13 ongoing trials addressing this debilitating condition, building upon their extensive history of 38 previously conducted studies. Their commitment to research began in 2009 when they recorded their first muscular dystrophy trial. Meanwhile, the University of Florida in Gainesville is making significant strides as well, currently conducting eight active clinical trials and having completed a total of 19 studies dedicated to muscular dystrophy since their inaugural trial also in 2009.
Moving on to Baltimore's Kennedy Krieger Institute, researchers there are actively involved in eight clinical trials focused on discovering breakthroughs for muscular dystrophy patients. With a history encompassing 19 previous investigations since initiating their first trial in 2014, this institute is dedicated to improving outcomes and quality of life for those affected by this genetic disorder.
Additionally contributing to these efforts is Arkansas Children's Hospital located in Little Rock where seven ongoing muscular dystrophy trials highlight their dedication towards finding potential solutions. Although it may seem relatively recent compared to other institutions' involvement, Arkansas Children's Hospital has made notable progress since recording its first trial merely three years ago -in2018-while completing eleven prior studies thus far.
Lastly,the University of Kansas Medical Center situated within Kansas City rounds off our list with seven active clinical trials targeting muscular dystrophy while boasting an admirable count of twenty-three completed investigations overall; such contributions initiated almost twelve years back,since recording its pioneering examination took place-way back-in2009.
These hospitals represent beacons of hope for individuals living with muscular dystrophy and demonstrate the tireless efforts being made globally towards combatting this complex disease. Each study undertaken brings us closer to understanding more about this condition while offering promising prospects for improved treatments that could transform countless lives worldwide
Which are the best cities for muscular dystrophy clinical trials?
When it comes to muscular dystrophy clinical trials, several cities emerge as leading locations for research and advancement. Los Angeles, California tops the list with 30 ongoing trials exploring treatments like Cohort 1, PF-06939926, and Pamrevlumab. Salt Lake City, Utah closely follows with 27 active studies focused on interventions such as PF-06939926, Cohort 1, and Losmapimod. Columbus, Ohio is also a prominent hub for muscular dystrophy research with 16 ongoing trials investigating therapies like Losmapimod and SRP-9003. These cities offer individuals affected by muscular dystrophy access to cutting-edge clinical trials that pave the way for improved care and potential breakthroughs in treatment options.
Which are the top treatments for muscular dystrophy being explored in clinical trials?
Muscular dystrophy continues to be an area of active exploration in clinical trials, with several promising treatments emerging. Leading the charge is PF-06939926, currently being tested in two ongoing trials for muscular dystrophy. Another contender is pamrevlumab, also involved in two active studies and showing promise since its listing in 2016. Last but not least is eteplirsen, which has gained attention with its involvement in two current trials and a total of five all-time muscular dystrophy trials since 2014. These treatments offer hope for patients as researchers strive towards finding effective solutions for this debilitating condition.
What are the most recent clinical trials for muscular dystrophy?
Promising advancements are being made in the realm of muscular dystrophy research, with recent clinical trials offering hope and potential breakthroughs. Notably, NS-089/NCNP-02 has entered Phase 2 trials as a potential treatment option for individuals with muscular dystrophy. Similarly, BBP-418 and delandistrogene moxeparvovec have both advanced to Phase 3 trials—a significant milestone that brings us closer to effective therapies for this condition. Additionally, SRP-6004 is currently undergoing Phase 1 testing, while AOC 1020 Regimen 1 shows promise through its combined Phase 1 and Phase 2 study design. These ongoing clinical trials hold immense promise for patients grappling with muscular dystrophy and offer renewed optimism in their pursuit of improved quality of life.
What muscular dystrophy clinical trials were recently completed?
Several significant clinical trials for muscular dystrophy have recently reached completion, providing important insights into potential treatments. In December 2021, PTC Therapeutics concluded its trial investigating the efficacy of Ataluren in addressing this debilitating condition. Similarly, NS Pharma successfully completed a trial for Viltolarsen in July 2021. While not as recent, Northwestern University's Prednisone study wrapped up in July 2019 and also contributed valuable information to the field of muscular dystrophy research. These milestones demonstrate ongoing efforts to find effective therapies for individuals living with this challenging disorder.