Clinical trials: Duchenne Dystrophy Muscular
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.
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Carrier of Duchenne Muscular Dystrophy
Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study
Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle.
- Trial name
- NCT01712152
- Where
- Austria
- Volunteers
- 20 people
- Sponsor
- Hospital Rudolfstiftung
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.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 2
The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.
- Trial name
- NCT04668716
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 339 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing…
- Trial name
- NCT06241950
- Testing
- delandistrogene moxeparvovec, imlifidase
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Sarepta Therapeutics, Inc.
Spinal Muscular Atrophy, Duchenne Muscular Dystrophy
Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices
The addition of SMA and DMD muscle diseases to newborn screening and premarital carrier screening has been controversial. In this study, researchers aim to measure the awareness level of SMA and DMD muscle diseases of individuals…
- Conditions
- Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Healthy Individuals
- Trial name
- NCT05110885
- Testing
- Questionnaire
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Bahçeşehir University
Duchenne Muscular Dystrophy
Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy
The aim of this population based study is to examine, quantify and describe physical activity level in Norwegian boys with DMD, and to compare the level of physical activity level between boys with DMD and age matched healthy…
- Trial name
- NCT03947112
- Testing
- Physical activity registration
- Where
- Norway
- Volunteers
- 28 people
- Sponsor
- Haukeland University Hospital
Duchenne Muscular Dystrophy
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD.
- Trial name
- NCT00758225
- Testing
- Idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy, Gamification
Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy
This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program.
- Conditions
- Duchenne Muscular Dystrophy, Gamification, Occupational Therapy
- Trial name
- DMD (NCT06402942)
- Testing
- Gamified occupational therapy programme, Classic occupational therapy programme
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Başak Çağla Arslan
Duchenne Muscular Dystrophy
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Primary Objective The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.
- Trial name
- NCT02851797
- Testing
- givinostat
- Where
- 42 sites in United States, Belgium and 9 more
- Volunteers
- 179 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy, Pompe Disease (Late-onset)
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
This is a multicenter prospective non-drug screening study. The working period is 12 months. There is no research product to be followed or used in the study.
- Trial name
- VICTORIA (NCT04120168)
- Testing
- Laboratory Tests
- Where
- 51 sites in Turkey (Türkiye)
- Volunteers
- 590 people
- Sponsor
- Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
Inflammatory Myopathy, Duchenne Muscular Dystrophy
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
The aim of our study is to Assess skeletal muscle structural status in children with inflammatory myositis and Duchenne muscular dystrophy using musculoskeletal ultrasound and to perform a longitudinal follow up of these changes…
- Trial name
- NCT03786913
- Testing
- Quantitative muscle ultrasound measurements
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Benha University
Muscular Dystrophy, Duchenne
KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033813
- Testing
- Oxatomide (tinset)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
- Trial name
- ASCEND (NCT06817382)
- Testing
- INS1201
- Where
- 10 sites in United States
- Volunteers
- 12 people
- Sponsor
- Insmed Gene Therapy LLC
Duchenne Muscular Dystrophy
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01847573
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Processa Pharmaceuticals
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
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.
Duchenne Muscular Dystrophy
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.
- Trial name
- NCT01957059
- Testing
- Regimen Selection Phase Group 2, Regimen Selection Phase Group 3, Treatment Phase Group 4
- Where
- 6 sites in Belgium, France and 3 more
- Volunteers
- 9 people
- Sponsor
- BioMarin Pharmaceutical