Clinical trials: Duchenne Muscular Dystrophin (DMD)
Duchenne Muscular Dystrophy
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT00428935
- Testing
- rAAV2.5-CMV-minidystrophin (d3990)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
This is a Phase 1/2a, monocentric, open label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of SQY51 in patients with Duchenne muscular dystrophy
- Trial name
- AVANCE1 (NCT05753462)
- Testing
- Phase 1, SQY51, Phase 2a, SQY51 (cohort 1), Phase 2a, SQY51 (cohort 2)
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Sqy Therapeutics
Dilated Cardiomyopathy, Lef Ventricular Dysfunction
Prospective Becker-Heart-Study
The purpose of this study is to determine whether electrocardiogram, echocardiography, cardiac MRI, sera biomarkers can improve early detection of myocardial involvement and clinical outcome.
- Conditions
- Dilated Cardiomyopathy, Lef Ventricular Dysfunction, Heart Failure
- Trial name
- Becker-HS (NCT02020954)
- Testing
- ECG, echocardiography, cardiac MRI, sera biomarkers
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Karim WAHBI
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
The goal of this observational study is to investigate whether the type, location, and extent of pathogenic variants in the DMD gene are associated with cardiac dysfunction in male children, adolescents, and young adults with…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Cardiomyopathy
- Trial name
- NCT07515235
- Where
- Greece
- Volunteers
- 65 people
- Sponsor
- Aristotle University Of Thessaloniki
Duchenne Muscular Dystrophy
Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
The proposed phase I clinical trial is a pilot study to evaluate safety and biological activity of the rAAVrh74.MCK.micro-Dystrophin vector administered by an intramuscular route.
- Trial name
- NCT02376816
- Testing
- rAAVrh74.MCK.micro-Dystrophin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Jerry R. Mendell
Muscular Dystrophy, Duchenne Muscular Dystrophy
Follow-up Study on Female Carriers With DMD Gene Variants
Background Duchenne and Becker muscular dystrophies are X-linked recessive allelic disorders caused by mutations of the dystrophin gene on chromosome Xp21.
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Trial name
- NCT05715957
- Testing
- No intervention
- Where
- Denmark
- Volunteers
- 103 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy (DMD)
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02420379
- Testing
- eteplirsen
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)
The purpose of this study is to describe the progression of tissular and functional myocardial abnormalities in patients with Duchenne muscular dystrophy using cardiac magnetic resonance imaging and blood biomarkers assays.
- Trial name
- DMD-CMP (NCT05558813)
- Testing
- Cardiac MRI, Blood assays
- Where
- France
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Duchenne Muscular Dystrophy
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
- Trial name
- ENVOL (NCT06128564)
- Testing
- delandistrogene moxeparvovec
- Where
- 7 sites in Belgium, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
Duchenne Muscular Dystrophy
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
- Trial name
- NCT00844597
- Testing
- AVI-4658 for Injection
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
- Trial name
- ENDEAVOR (NCT04626674)
- Testing
- delandistrogene moxeparvovec
- Where
- 12 sites in United States
- Volunteers
- 83 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), a fatal muscle degenerative disorder, arises from mutations in the dystrophin gene. Antisense therapy with the use of antisense oligonucleotides (AON) has the potential to restore effectively…
- Trial name
- NCT00159250
- Testing
- AVI-4658 (PMO)
- Where
- United Kingdom
- Volunteers
- 7 people
- Sponsor
- Imperial College London
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
Gentamicin Treatment of Muscular Dystrophy
This study will evaluate the antibiotic gentamicin for treating patients with muscular dystrophy caused by a specific genetic abnormality known as a nonsense mutation.
- Trial name
- NCT00005574
- Testing
- Gentamicin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy, Duchenne
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD.
- Trial name
- PolarisDMD (NCT03703882)
- Testing
- Edasalonexent
- Where
- 40 sites in United States, Australia and 6 more
- Volunteers
- 131 people
- Sponsor
- Catabasis Pharmaceuticals
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.