Clinical trials: Duchenne's Muscular Dystrophy
Muscular Dystrophy, Duchenne
KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033813
- Testing
- Oxatomide (tinset)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy
The goal of this clinical trial is to learn if providing a multicomponent intervention improves the confidence of caregivers of children with DMD or SMA.
- Trial name
- SEE-NMD (NCT07356063)
- Testing
- Multicomponent Intervention
- Where
- Pakistan
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
Duchenne Muscular Dystrophy (DMD)
Extension Study of NS-089/NCNP-02 in DMD
This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05135663
- Testing
- NS-089/NCNP-02
- Where
- 2 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Nippon Shinyaku Co., Ltd.
Duchenne Muscular Dystrophy
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
The study is to demonstrate non-inferiority of spironolactone vs. eplerenone in preserving cardiac and pulmonary function in patients with preserved LV ejection fraction.
- Trial name
- NCT02354352
- Testing
- Eplerenone, Spironolactone
- Where
- 6 sites in United States
- Volunteers
- 52 people
- Sponsor
- Ohio State University
Duchenne Muscular Dystrophy
Once Weekly Infant Corticosteroid Trial for DMD
The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
- Trial name
- NCT05412394
- Testing
- Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 26 people
- Sponsor
- Anne M. Connolly
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]
Until twenty years ago physical exercise in muscular dystrophies was considered harmful to the muscle cells, inducing an acceleration of cell necrosis.
- Conditions
- Muscular Dystrophies, Limb Girdle Muscular Dystrophy, Facio-Scapulo-Humeral Dystrophy, Becker Muscular Dystrophy
- Trial name
- RIMUDI (NCT06378203)
- Testing
- tele-rehabilitation system with virtual reality Home Kit - Virtual Reality Rehabilitation System (VRRS KHYMEIA)
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Eugenio Medea
Duchenne Muscular Dystrophy
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen…
- Trial name
- EVOLVE (NCT06606340)
- Testing
- Eteplirsen, Golodirsen, Casimersen
- Where
- 23 sites in United States
- Volunteers
- 300 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration.
- Trial name
- Acti-nRoll (NCT07664124)
- Testing
- Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
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)
Duchenne Muscular Dystrophy
Comprehensive Study of Duchenne Muscular Dystrophy at Sohag University Hospital
Muscular dystrophies are a heterogenous group of inherited muscular disorders characterized by progressive muscle weakness. Historically, these disorders are difficult to treat.
- Trial name
- NCT05029232
- Testing
- MLPA for duchenne
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Sohag University
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
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
Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy
Whole-body vibration therapy (WBVT) is a novel, non-pharmacological intervention aimed at improving muscle strength and endurance as well as bone density.
- Trial name
- NCT01954940
- Testing
- Whole Body Vibration Therapy
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Children's Hospital of Eastern Ontario
Muscular Dystrophy, Duchenne
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Improved standards of care and the regular early use of glucocorticoid treatment have changed the natural history of Duchenne muscular dystrophy (DMD), affecting both survival and time of loss of functional milestones.
- Trial name
- NCT05833633
- Testing
- MUSCLE MAGNETIC RESONANCE IMAGING (MRI)
- Where
- Italy
- Volunteers
- 17 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
CureDuchenne Link®: A Resource for Research
CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants.
- Trial name
- CDLink (NCT04972604)
- Where
- 10 sites in United States
- Volunteers
- 240 people
- Sponsor
- CureDuchenne
Carrier of Duchenne Muscular Dystrophy
Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study
Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle.
- Trial name
- NCT01712152
- Where
- Austria
- Volunteers
- 20 people
- Sponsor
- Hospital Rudolfstiftung
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
The study will include 120 participants aged 8 and up with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies that have no clinical cardiac symptoms.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01126697
- Testing
- Coenzyme Q10 and Lisinopril
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 63 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
- Trial name
- NCT04956289
- Testing
- Viltolarsen
- Where
- 8 sites in United States, China and 4 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.