Clinical trials: Muscular Dystrophy
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Limb Girdle Muscular Dystrophy
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Neurologic Disorder, Paralysis
SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis
The Synchron Motor Neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex.
- Conditions
- Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis
- Trial name
- NCT05041114
- Testing
- Motor Neuroprosthesis
- Where
- 3 sites in Australia
- Sponsor
- Synchron Medical, Inc.
Duchenne Muscular Dystrophy
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed…
- Trial name
- GalaxyDMD (NCT03917719)
- Testing
- Edasalonexent
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 130 people
- Sponsor
- Catabasis Pharmaceuticals
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
- Trial name
- LELANTOS-2 (NCT04632940)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 52 sites in United States, Australia and 9 more
- Volunteers
- 73 people
- Sponsor
- Kyntra Bio
Primary Familial Dilated Cardiomyopathy
Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities
The purpose of this Phase 2a study is to establish safety and preliminary efficacy of treatment with danicamtiv in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants or other causalities.
- Trial name
- NCT04572893
- Testing
- danicamtiv
- Where
- 19 sites in United States, Germany and 2 more
- Volunteers
- 41 people
- Sponsor
- Bristol-Myers Squibb
Duchenne Muscular Dystrophy
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage…
- Trial name
- NCT02667483
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD).
- Trial name
- NCT05154851
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Clinical Trial
The primary objective of this investigation is to assess the effectiveness of transcutaneous electrical nerve stimulation applied using VECTTOR to reduce the symptoms of Duchenne Muscular Dystrophy and reduce the impact of DMD…
- Trial name
- DMD (NCT01874275)
- Testing
- VECTTOR
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alan Neuromedical Technologies, LLC
Facioscapulohumeral Muscular Dystrophy
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy…
- Trial name
- NCT02927080
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 95 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.
- Trial name
- NCT01957059
- Testing
- Regimen Selection Phase Group 2, Regimen Selection Phase Group 3, Treatment Phase Group 4
- Where
- 6 sites in Belgium, France and 3 more
- Volunteers
- 9 people
- Sponsor
- BioMarin Pharmaceutical
Facioscapulohumeral Muscular Dystrophy (FSHD)
Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
This is a study to evaluate the safety and efficacy of losmapimod in treating participants with Facioscapulohumeral Muscular Dystrophy (FSHD).
- Trial name
- NCT05397470
- Testing
- Losmapimod
- Where
- 33 sites in United States, Canada and 7 more
- Volunteers
- 260 people
- Sponsor
- Fulcrum Therapeutics
Duchenne Muscular Dystrophy
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
- Trial name
- NCT04687020
- Testing
- Viltolarsen
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- NS Pharma, Inc.