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.
MAP Kinase Inhibitor
Losmapimod for Facioscapulohumeral Muscular Dystrophy
This trial is testing if the medication Losmapimod is safe for long-term use in patients with FSHD1. Patients will take the medication twice daily and visit the clinic regularly to check for any side effects. Losmapimod has been previously tested in other conditions but did not show significant efficacy.
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.
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Trials for Duchenne Muscular Dystrophy 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
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.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
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
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.
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
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.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
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
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.
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.
Histone Deacetylase Inhibitor
Givinostat for Duchenne Muscular Dystrophy
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 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
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 Limb-Girdle Muscular Dystrophy
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
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
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 Related Trials
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.