Clinical trials: Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
The Burden of Access in Duchenne Muscular Dystrophy in the US
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these…
- Trial name
- NCT03951675
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
DMD/BMD 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.
- Trial name
- NCT00592553
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 174 people
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne
Characterization of DupEx2 Duchenne Muscular Dystrophy
To characterize the clinical phenotype and possible predictive/prognostic factors of patients with Duchenne muscular dystrophy (DMD) due to duplication of exon 2 (Dup2).
- Trial name
- DMDDup2 (NCT06337669)
- Where
- Italy
- Volunteers
- 26 people
- Sponsor
- IRCCS San Raffaele
Duchenne's Muscular Dystrophy (DMD)
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.
- Trial name
- NCT01995032
- Testing
- 750 mg metformin and 7.5 g L-citrulline daily p.o.
- Where
- Switzerland
- Volunteers
- 47 people
- Sponsor
- University Hospital, Basel, Switzerland
Muscular Dystrophy
The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise.
- Trial name
- NCT02653833
- Testing
- Tadalafil 20 MG, beetroot juice extract
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
Validity and Reliability of the 6 Minute Pegboard Ring Test
This study aimed to investigate the validity and reliability of 6PBRT in individuals with DMD and its applicability on these patients.
- Trial name
- NCT06174025
- Where
- Turkey (Türkiye)
- Volunteers
- 22 people
- Sponsor
- Çağtay Maden
Duchenne Muscular Dystrophy
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with…
- Trial name
- NCT03508947
- Testing
- WVE-210201
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Duchenne Muscular Dystrophy, Ambulation Difficulty
Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment
The aim of this study is to translate the "North Star Ambulatory Assessment (NSAA)" scale into Turkish and make its cultural adaptation and to demonstrate the reliability and validity of the Turkish version in patients with…
- Conditions
- Duchenne Muscular Dystrophy, Ambulation Difficulty, Ambulation Disorder, Neurologic
- Trial name
- NCT05549999
- Testing
- North Star Ambulatory Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Hacettepe University
Muscular Dystrophy, Duchenne
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping…
- Trial name
- NCT03375255
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophies
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- NCT01910649
- Testing
- Drisapersen
- Volunteers
- 12 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
The investigators are performing a gene therapy clinical trial in Becker muscular dystrophy (BMD) and sporadic inclusion body myositis (sIBM) patients.
- Trial name
- NCT01519349
- Testing
- rAAV1.CMV.huFollistatin344
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
Telerehabilitation in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive inherited disease that affects the muscles and causes functional limitations to varying degrees. It is vital to start physiotherapy follow-ups immediately after diagnosis.
- Trial name
- Duchenne (NCT05305976)
- Testing
- Telerehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Duchenne Muscular Dystrophy
A Registered Cohort Study on Duchenne Muscular Dystrophy
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy.
- Trial name
- NCT04012671
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Ning Wang, MD., PhD.
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Stress
Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy
Background: Children with Duchenne/Becker Muscular Dystrophy (DBMD) slowly lose muscle function. They usually die at a young age. Some mothers adapt to the demands of caring for a child with this disease better than others.
- Trial name
- NCT02194725
- Where
- United States
- Volunteers
- 228 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Performance, Energy
Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption
Investigators investigated that the effects of kinesilogy taping on performance, energy consumption and gait characteristics in patients with Duchenne Muscular Dystrophy
- Conditions
- Performance, Energy, Gait, Duchenne Muscular Dystrophy
- Trial name
- NCT03529240
- Testing
- Kinesiology Taping
- Where
- Turkey (Türkiye)
- Volunteers
- 45 people
- Sponsor
- Hacettepe University
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