Clinical trials: Muscular Dystrophy, Becker
Duchenne Muscular Dystrophy
Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)
- Trial name
- NCT05338099
- Testing
- EN001
- Where
- South Korea
- Volunteers
- 7 people
- Sponsor
- ENCell
Muscular Dystrophy, Duchenne
Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is inherited neuromuscular disorders due to mutation in the gene that encodes critical muscle protein called dystrophin. Currently, there is no effective treatment option for the disease.
- Trial name
- NORTH POLE DMD (NCT01918384)
- Testing
- NPC-14
- Where
- 4 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Kobe University
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.
Muscular Dystrophy, Duchenne
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in…
- Trial name
- NCT03675126
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
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
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
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.
- Trial name
- NCT04433234
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
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 of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy (DMD)
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
- Trial name
- NCT06867107
- Testing
- SAT-3247
- Where
- 6 sites in United States, Australia
- Volunteers
- 30 people
- Sponsor
- Satellos Bioscience, Inc.
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 (DMD)
A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants
The purpose of this trial is to evaluate the safety, tolerability, and Pharmacokinetics (PK) of a single oral dose of GRT6019 in healthy male participants, including food effect.
- Trial name
- NCT07317063
- Testing
- GRT6019
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Healthy Volunteer, Becker Muscular Dystrophy
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult…
- Trial name
- NCT04585464
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
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
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
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02056808
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
This is an investigation of the efficacy and safety of CRD007 in Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers.
- Trial name
- NCT01540604
- Testing
- CRD007
- Where
- Sweden
- Sponsor
- RSPR Pharma AB
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
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