Clinical trials: Muscular Dystrophy, Duchenne
Duchenne Muscular Dystrophy
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for…
- Trial name
- NCT01099761
- Testing
- ACE-031 0.5 mg/kg q4wk, ACE-031 1.0 mg/kg q2wk
- Where
- 5 sites in Canada
- Volunteers
- 24 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Duchenne Muscular Dystrophy
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or…
- Trial name
- NCT04179409
- Testing
- Amondys 45, Exondys 51, Vyondys 53
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Kevin Flanigan
Duchenne Muscular Dystrophy
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial.
- Trial name
- NCT02354781
- Testing
- rAAV1.CMV.huFollistin344
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Jerry R. Mendell
Duchenne Muscular Dystrophy
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24…
- Trial name
- NCT03038399
- 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
- 46 people
- Sponsor
- ReveraGen BioPharma, Inc.