Clinical trials: DMD
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old.
- Trial name
- NCT02760264
- Testing
- Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young…
- Trial name
- DMD (NCT03340675)
- Testing
- Ifetroban
- Where
- 10 sites in United States
- Volunteers
- 46 people
- Sponsor
- Cumberland Pharmaceuticals
Muscular Dystrophy, Duchenne
Spironolactone Versus Prednisolone in DMD
This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy.
- Trial name
- NCT03777319
- Testing
- Spironolactone, Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Duchenne Muscular Dystrophy
Tadalafil as Adjuvant Therapy for DMD
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler…
- Trial name
- NCT05195775
- Testing
- Tadalafil
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy (DMD)
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully…
- Trial name
- NCT01540409
- Testing
- AVI-4658 (Eteplirsen)
- Where
- 11 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a…
- Trial name
- NCT02819557
- Testing
- Ataluren
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded…
- Trial name
- NCT03769116
- Testing
- delandistrogene moxeparvovec
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne…
- Trial name
- NCT02907619
- Testing
- PF-06252616
- Where
- 47 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
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
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon…
- Trial name
- NCT02530905
- Testing
- SRP-4045
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
- Trial name
- NCT04060199
- Testing
- Viltolarsen
- Where
- 40 sites in United States, Australia and 17 more
- Volunteers
- 77 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
- Trial name
- NCT02752048
- Testing
- TAS-205
- Where
- 11 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Nonsene Mutation Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20…
- Trial name
- NCT04336826
- Testing
- Ataluren
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to sequentially deliver vector to…
- Trial name
- NCT03333590
- Testing
- rAAVrh74.MCK.GALGT2
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Becker Muscular Dystrophy
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
Objectives: Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months. Secondary Objectives: To establish the macroscopic muscle effects of Givinostat versus placebo…
- Trial name
- NCT03238235
- Testing
- Givinostat
- Where
- 2 sites in Italy, Netherlands
- Volunteers
- 51 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD…
- Trial name
- NCT02760277
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.