Clinical trials: Becker's Muscular Dystrophy (BMD)
Duchenne Muscular Dystrophy
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S.
- Trial name
- NCT02592941
- Testing
- Deflazacort
- Where
- 42 sites in United States
- Sponsor
- PTC Therapeutics
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
Open Label Extension Study of HT-100 in Patients With DMD
This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01).
- Trial name
- NCT01978366
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Processa Pharmaceuticals
Neuromuscular Diseases
Muscle Oxygenation in Effort in Neuromuscular Diseases
Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin.
- Trial name
- OXYNEMU (NCT02789059)
- Testing
- muscle oxygenation
- Where
- 3 sites in France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University Hospital, Lille
Becker Muscular Dystrophy
Sodium Nitrate to Improve Blood Flow
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD).
- Trial name
- NCT02847975
- Testing
- Sodium nitrate
- Where
- 2 sites in United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy
Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
This study, supported by Parent Project Muscular Dystrophy, will determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with Duchenne muscular dystrophy.
- Trial name
- NCT01359670
- Testing
- Tadalafil, Sildenafil
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN…
- Trial name
- NCT02958202
- Testing
- BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 7 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24…
- Trial name
- NCT03038399
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 46 people
- Sponsor
- ReveraGen BioPharma, Inc.
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 Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
- Trial name
- ENDEAVOR (NCT04626674)
- Testing
- delandistrogene moxeparvovec
- Where
- 12 sites in United States
- Volunteers
- 83 people
- Sponsor
- Sarepta Therapeutics, Inc.