Clinical trials: Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon…
- Trial name
- NCT02530905
- Testing
- SRP-4045
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance…
- Trial name
- NCT01523964
- Testing
- Testing with EIM
- Where
- 6 sites in United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Dart Therapeutics. LLC
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
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.
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
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Dystrophy (DMD)
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
- Trial name
- NCT02515669
- Testing
- RO7239361
- Where
- 12 sites in United States, Canada
- Volunteers
- 43 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD.
- Trial name
- NCT03648827
- Testing
- Ataluren
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03039686
- Testing
- RO7239361
- Where
- 44 sites in United States, Argentina and 11 more
- Volunteers
- 166 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
Duchenne/Becker muscular dystrophy (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
- NCT01009294
- Testing
- Ataluren, Chronic Corticosteroid Therapy
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Duchenne Muscular Dystrophy
Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
This single center open-label pilot study will enroll 15 non-ambulatory children with Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical cardiomyopathy (defined as a cardiac ejection fraction…
- Trial name
- NCT02964377
- Testing
- (+)- Epicatechin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Craig McDonald, MD
Duchenne Muscular Dystrophy
Tadalafil as Adjuvant Therapy for DMD
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler…
- Trial name
- NCT05195775
- Testing
- Tadalafil
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Florida
Nonsene Mutation Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20…
- Trial name
- NCT04336826
- Testing
- Ataluren
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01207908
- Testing
- IGF-1
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Duchenne Muscular Dystrophy
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
- Trial name
- NCT03167255
- Testing
- NS-065/NCNP-01
- Where
- 6 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy.
- Trial name
- NCT04281485
- Testing
- PF-06939926
- Where
- 53 sites in United States, Australia and 13 more
- Volunteers
- 114 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne…
- Trial name
- NCT01396239
- Testing
- AVI-4658 (Eteplirsen)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have…
- Trial name
- NCT03985878
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.