Clinical trials: DMD
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
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
Breast Reconstruction
Becker Continued Access Study
While Core Becker patient follow-up is being completed this Continued Access Study is designed to enroll patients at a limited rate per month to allow for continued physician and patient experience with the device.
- Trial name
- NCT01286870
- Testing
- Mentor Becker 25 Expander/Breast Implant, Mentor Becker 50 Expander/Breast Implant
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Mentor Worldwide, LLC
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
Muscular Dystrophy, Duchenne
A Study of Tadalafil for Duchenne Muscular Dystrophy
The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01865084
- Testing
- Tadalafil
- Where
- 62 sites in United States, Argentina and 14 more
- Volunteers
- 331 people
- Sponsor
- Eli Lilly and Company
Duchenne Muscular Dystrophy
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases…
- Trial name
- FOR-DMD (NCT01603407)
- Testing
- Prednisone, Deflazacort
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 196 people
- Sponsor
- University of Rochester
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
Muscular Dystrophy, Duchenne
Rimeporide in Patients With Duchenne Muscular Dystrophy
In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime.
- Trial name
- RIM4DMD (NCT02710591)
- Testing
- Rimeporide
- Where
- 4 sites in France, Italy and 2 more
- Volunteers
- 20 people
- Sponsor
- EspeRare Foundation
Duchenne Muscular Dystrophy
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
- Trial name
- NCT00844597
- Testing
- AVI-4658 for Injection
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy
This is an interventional study on Duchenne muscular dystrophy patients who will be receiving sedation for a muscle biopsy as part of another study.
- Trial name
- NCT01645098
- Testing
- Ketamine, Dexmedetomidine
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy (DMD)
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully…
- Trial name
- NCT01540409
- Testing
- AVI-4658 (Eteplirsen)
- Where
- 11 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy
Weekly Steroids in Muscular Dystrophy
The purpose of this study is to evaluate the safety and efficacy of oral weekly glucocorticoid steroids in patients with Becker Muscular Dystrophy (BMD), an inherited disorder in which patients experience weakness of the legs and…
- Trial name
- WSiMD (NCT04054375)
- Testing
- Prednisone
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Northwestern University
Muscular Dystrophies
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- DMD114044 (NCT01254019)
- Testing
- GSK2402968 6mg/kg/week
- Where
- 47 sites in Argentina, Belgium and 18 more
- Volunteers
- 186 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Cardiomyopathy in DMD: Lisinopril vs. Losartan
This trial is a double-blind randomized clinical trial of lisinopril versus losartan for the treatment of cardiomyopathy in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01982695
- Testing
- Losartan, Lisinopril
- Where
- 6 sites in United States
- Volunteers
- 23 people
- Sponsor
- Nationwide Children's Hospital
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
Duchenne Muscular Dystrophy
Phase I/II Study of SRP-4053 in DMD Patients
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
- Trial name
- NCT02310906
- Testing
- SRP-4053
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne…
- Trial name
- NCT02907619
- Testing
- PF-06252616
- Where
- 47 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Pfizer
Osteoporosis
An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids
- Trial name
- NCT00799266
- Testing
- Zoledronic acid
- Where
- 12 sites in Australia, Canada and 4 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) 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
- NCT00759876
- Testing
- Ataluren
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- PTC Therapeutics