Clinical trials: Duchenne's Muscular Dystrophy
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Cardiac Magnetic Resonance in Children With Muscular Dystrophy
This protocol will exploit novel state of the art cardiovascular magnetic resonance techniques to examine important changes in the heart in children with muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01081080
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid…
- Trial name
- NCT01125709
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 30 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle.
- Trial name
- NCT01610440
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
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
Dystrophinopathy, Duchenne Muscular Dystrophy
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids.
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Trial name
- NCT01388764
- Testing
- L-arginine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts General Hospital
Duchenne Muscular Dystrophy
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
The purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.
- Trial name
- NCT00451074
- Testing
- Gentamicin infusions twice a week for six months
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Nationwide Children's Hospital
Muscular Dystrophy, Duchenne
Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an…
- Trial name
- NCT00016653
- Testing
- Creatine Monohydrate, Glutamine
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
Pentoxifylline in Duchenne Muscular Dystrophy
In this study, the primary aim will be to estimate the magnitude and variability of strength change over time that may be expected for subjects on the study treatment.
- Trial name
- NCT00102453
- Testing
- Pentoxifylline
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
High-dose Prednisone in Duchenne Muscular Dystrophy
This study will help to determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00110669
- Testing
- Prednisone
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD) have changes in strength when given the drug Pentoxifylline as a rescue treatment.
- Trial name
- NCT00243789
- Testing
- Pentoxifylline
- Where
- 11 sites in United States, Argentina and 4 more
- Volunteers
- 64 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy (DMD)
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level.
- Trial name
- DELPHI (NCT00654784)
- Testing
- idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD.
- Trial name
- NCT00758225
- Testing
- Idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Muscular Dystrophy, Duchenne
An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the nutritional supplement Coenzyme Q10 when added to steroids as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033189
- Testing
- Coenzyme Q10
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophies
The Efficacy of Using Far Infrared Radiation to Manage Muscular Dystrophies
The muscular dystrophies (MD) are a group of more than 30 neuromuscular disorders that are characterized by progressive skeletal muscle weakness, defects in muscle proteins and the death of muscle cells and tissue.
- Trial name
- NCT00674843
- Testing
- Far Infrared Radiation
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- GAAD Medical Research Institute Inc.
Duchenne Muscular Dystrophy
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein.
- Trial name
- NCT00264888
- Testing
- PTC124
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- PTC Therapeutics
Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
Gentamicin Treatment of Muscular Dystrophy
This study will evaluate the antibiotic gentamicin for treating patients with muscular dystrophy caused by a specific genetic abnormality known as a nonsense mutation.
- Trial name
- NCT00005574
- Testing
- Gentamicin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Duchenne Muscular Dystrophy, Cardiomyopathies
The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
Purpose This cardiac dysfunction in patients with Duchenne muscular dystrophy is associated with minor cardiac damage as indicated by elevation of plasma cardiac troponin I (cTnI).
- Trial name
- NCT00606775
- Testing
- Carvedilol
- Where
- Japan
- Volunteers
- 60 people
- Sponsor
- Suzuka Hospital
Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Study Evaluating MYO-029 in Adult Muscular Dystrophy
The purpose of this phase I/II, multicenter, safety trial is to study MYO-029 in adult patients with muscular dystrophy.
- Conditions
- Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
- Trial name
- NCT00104078
- Testing
- MYO-029
- Where
- 9 sites in United States
- Volunteers
- 108 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Muscular Dystrophy, Duchenne
Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether long-term oral glutamine supplementation is effective in improving muscle mass and function in children with Duchenne muscular dystrophy (DMD).
- Trial name
- MDB-GLN (NCT00296621)
- Testing
- L-Glutamine
- Where
- 5 sites in France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Duchenne Muscular Dystrophy
Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy. II.
- Trial name
- NCT00004646
- Testing
- prednisone
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)