Clinical trials: Duchenne's Muscular Dystrophy
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.
Duchenne Muscular Dystrophy
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up.
- Trial name
- HOPE-OLE (NCT06304064)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Capricor Inc.
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT03532542
- Testing
- Casimersen, Golodirsen
- Where
- 50 sites in United States, Belgium and 11 more
- Volunteers
- 171 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
- Trial name
- ASCEND (NCT06817382)
- Testing
- INS1201
- Where
- 10 sites in United States
- Volunteers
- 12 people
- Sponsor
- Insmed Gene Therapy LLC
Duchenne Muscular Dystrophy
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
- Trial name
- NCT03167255
- Testing
- NS-065/NCNP-01
- Where
- 6 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
A Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT02246478
- Testing
- TAS-205
- Where
- Japan
- Volunteers
- 23 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Duchenne Muscular Dystrophy
NS-050/NCNP-03 in Boys With DMD (Meteor50)
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD…
- Trial name
- NCT06053814
- Testing
- NS-050/NCNP-03
- Where
- 19 sites in United States, Canada and 3 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
NSAA NON-Interventional Study Protocol
The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT06054971
- Testing
- Remote Administration of NSAA
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
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 Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients.
- Trial name
- NCT03760029
- Testing
- Visit frequency
- Where
- 8 sites in China
- Volunteers
- 312 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study.
- Trial name
- EXPEDITION (NCT05967351)
- Testing
- delandistrogene moxeparvovec
- Where
- 38 sites in United States, Belgium and 7 more
- Volunteers
- 400 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported…
- Trial name
- DMD-001 SUMMIT (NCT06564974)
- Testing
- Vamorolone
- Where
- 33 sites in United States, Puerto Rico
- Volunteers
- 250 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Duchenne Muscular Dystrophy (DMD)
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367. This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.
- Trial name
- NCT04184882
- Testing
- Bocidelpar
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Astellas Pharma Inc
Duchenne Muscular Dystrophy
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data.
- Trial name
- NCT01239758
- Testing
- ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03), ACE-031 (Extension of cohort 3 from core study, A031-03)
- Where
- 4 sites in Canada
- Volunteers
- 11 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Muscular Dystrophies
Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations correctable by drisapersen-induced DMD Exon 51…
- Trial name
- NCT01890798
- Testing
- Drisapersen
- Sponsor
- GlaxoSmithKline
Augmentation, Augmentation Revision
Spectra Breast Implant Study
The purpose of this study is to demonstrate safety and effectiveness of Mentor's Spectra/Becker 80 Adjustable Breast Implants in women who are undergoing primary or revision breast augmentation.
- Conditions
- Augmentation, Augmentation Revision, General Breast Enlargement, Post-lactational Involution
- Trial name
- NCT00981266
- Testing
- Mentor Spectra/Becker 80 Breast Implant
- Where
- United States
- Sponsor
- Mentor Worldwide, LLC
Duchenne Muscular Dystrophy
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies.
- Trial name
- NCT05689164
- Testing
- fordadistrogene movaparvovec
- Where
- 12 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pfizer
Becker Muscular Dystrophy
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
- Trial name
- MESA (NCT06066580)
- Testing
- Sevasemten
- Where
- 48 sites in United States, Australia and 10 more
- Volunteers
- 260 people
- Sponsor
- Edgewise Therapeutics, Inc.