Clinical trials: Duchenne's Muscular Dystrophy
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study.
- Trial name
- ENVISION (NCT05881408)
- Testing
- delandistrogene moxeparvovec
- Where
- 46 sites in United States, Australia and 12 more
- Volunteers
- 148 people
- Sponsor
- Sarepta Therapeutics, 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
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Wearable Technology to Assess Gait Function in SMA and DMD
The purpose of this project is to devise instrumented insoles capable of accurately measuring gait at each footfall, over multiple hours in any environment.
- Trial name
- NCT04193085
- Testing
- Observational
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Columbia University
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.
- Trial name
- NCT06839469
- Where
- 3 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Columbia University
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon
Duchenne Muscular Dystrophy
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with…
- Trial name
- DYSTANCE 51 (NCT03907072)
- Testing
- WVE-210201 (suvodirsen)
- Where
- 22 sites in United States, Belgium and 6 more
- Volunteers
- 6 people
- Sponsor
- Wave Life Sciences USA, Inc.
Becker Muscular Dystrophy
(-)- Epicatechin Becker Muscular Dystrophy
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial.
- Trial name
- NCT03236662
- Testing
- (-)-Epicatechin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Craig McDonald, MD
Becker Muscular Dystrophy, Muscular Dystrophies
Defining Endpoints in Becker Muscular Dystrophy
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Trial name
- GRASP-01-002 (NCT05257473)
- Where
- 11 sites in United States, New Zealand and 1 more
- Volunteers
- 59 people
- Sponsor
- Virginia Commonwealth University
Muscular Dystrophy, Duchenne
Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
Placebo-controlled, multi-centre, randomized, double-blind dose escalation study. The aim is to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a…
- Trial name
- NCT02383511
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Muscular Dystrophy, Duchenne
A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)
To establish a collaborative group of clinical trial centers, with standardized equipment and protocols, able to conduct both drug and gene therapy trials in DMD.
- Trial name
- NCT00018109
- Testing
- glutamine, creatine monohydrate
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Clinical Trial
The primary objective of this investigation is to assess the effectiveness of transcutaneous electrical nerve stimulation applied using VECTTOR to reduce the symptoms of Duchenne Muscular Dystrophy and reduce the impact of DMD…
- Trial name
- DMD (NCT01874275)
- Testing
- VECTTOR
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alan Neuromedical Technologies, LLC
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystrophies
Taking part in community activities is essential for health and well-being. Yet, it is highly restricted for young people with Duchenne and Becker Muscular Dystrophies (DBMD), especially as they grow into adulthood.
- Trial name
- NCT06540365
- Testing
- Pathways and Resources for Engagement and Participation (PREP)
- Volunteers
- 6 people
- Sponsor
- University of Haifa
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
Duchenne Muscular Dystrophy
Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), the most common muscular dystrophy, leads to skeletal and cardiac muscle damage. Treatment of pulmonary complications has improved survival; however, heart muscle disease or cardiomyopathy has…
- Trial name
- E-SCAR DMD (NCT01521546)
- Testing
- eplerenone
- Where
- 3 sites in United States
- Volunteers
- 42 people
- Sponsor
- Subha Raman
Duchenne Muscular Dystrophy
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo…
- Trial name
- NCT04740554
- Testing
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy…
- Conditions
- Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP), Duchenne Muscular Dystrophy (DMD)
- Trial name
- NCT07254988
- Testing
- Meal test, GIP + GLP-2
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- University of Copenhagen
Duchenne Muscular Dystrophy
PDE Inhibitors in DMD Study (Acute Dosing Study)
PDE5A inhibition, which boosts NO-cGMP signaling, will relieve functional muscle ischemia and restore normal blood flow regulation (i.e., functional sympatholysis) during exercise in boys with DMD.
- Trial name
- NCT01580501
- Testing
- Tadalafil and Sildenafil
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy
Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy
This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY).
- Trial name
- DMD (NCT04529707)
- Testing
- Transdiagnostic Behavioral Sleep Intervention
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- University of Pittsburgh
Muscular Dystrophy, Duchenne
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD.
- Trial name
- PolarisDMD (NCT03703882)
- Testing
- Edasalonexent
- Where
- 40 sites in United States, Australia and 6 more
- Volunteers
- 131 people
- Sponsor
- Catabasis Pharmaceuticals
Duchenne Muscular Dystrophy
Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study
Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding of DMD natural history. This study aim to assess the natural history of DMD through a composite assessment tool capable of capturing…
- Trial name
- NCT02780492
- Testing
- Set of assessment tools
- Where
- 5 sites in France, Netherlands and 1 more
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- University College, London