Clinical trials: Duchenne's Muscular Dystrophy
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 / Becker Muscular Dystrophy, Dystrophia Myotonica 1
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases.
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Trial name
- Dystro-miR1 (NCT07415837)
- Testing
- dosage of blood biomarker miR1
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
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
Breast Reconstruction
Compassionate Use of the Becker Expander/Breast Implant
To provide access of the Becker Expander/Breast implant to women who do not meet inclusion/exclusion criteria of the Becker Continued Access Study Patients' physician will contact Mentor to request use of the device and each…
- Trial name
- NCT01000012
- Testing
- Becker 50 Expander/Breast implant, Becker 25 Expander/Breast implant
- Where
- United States
- Sponsor
- Mentor Worldwide, LLC
Duchenne Muscular Dystrophy
Trial of Cell Based Therapy for DMD
This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals…
- Trial name
- NCT06692426
- Testing
- MyoPAXon, Tacrolimus
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Other Diseases or Conditions
Mother-caregivers of Children With Duchenne Muscular Dystrophy
The incidence of Duchenne Muscular Dystrophy (DMD) is approximately 1 in 3.500 male newborns. During its progression there is loss of mobility, swallowing difficulties and a significant reduction in respiratory capacity.
- Trial name
- DMDK (NCT01921374)
- Testing
- sleep parameters, Hormonal profile, Inflammatory profile
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Monica Levy Andersen
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55.
- Trial name
- NCT07429240
- Testing
- PBGENE-DMD (IV)
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Precision BioSciences, Inc.
Duchenne Muscular Dystrophy
CoQ10 and Prednisone in Non-Ambulatory DMD
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD.
- Trial name
- NCT00308113
- Testing
- Prednisone, Coenzyme Q10
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Cooperative International Neuromuscular Research Group
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
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, Duchenne Muscular Dystrophy
Follow-up Study on Female Carriers With DMD Gene Variants
Background Duchenne and Becker muscular dystrophies are X-linked recessive allelic disorders caused by mutations of the dystrophin gene on chromosome Xp21.
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Trial name
- NCT05715957
- Testing
- No intervention
- Where
- Denmark
- Volunteers
- 103 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
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
Muscular Dystrophy, Duchenne
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically…
- Trial name
- MoveDMD® (NCT02439216)
- Testing
- Edasalonexent
- Where
- 5 sites in United States
- Volunteers
- 31 people
- Sponsor
- Catabasis Pharmaceuticals
Muscular Dystrophies
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876…
- Trial name
- NCT01803412
- Testing
- Drisapersen
- Where
- 13 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT00428935
- Testing
- rAAV2.5-CMV-minidystrophin (d3990)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
Telerehabilitation in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive inherited disease that affects the muscles and causes functional limitations to varying degrees. It is vital to start physiotherapy follow-ups immediately after diagnosis.
- Trial name
- Duchenne (NCT05305976)
- Testing
- Telerehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Duchenne Muscular Dystrophy
Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is…
- Trial name
- NCT06295718
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Sahra Şirvan
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