Clinical trials: Duchenne's Muscular Dystrophy
Duchenne Muscular Dystrophy
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory…
- Trial name
- HORIZON (NCT06597656)
- Testing
- delandistrogene moxeparvovec, Plasmapheresis
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an…
- Trial name
- NCT00016653
- Testing
- Creatine Monohydrate, Glutamine
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Stem Cell Therapy in Duchenne Muscular Dystrophy
The purpose of this study was to study the effect of stem cell therapy in patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT02241434
- Testing
- Stem Cell
- Where
- India
- Sponsor
- Neurogen Brain and Spine Institute
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock
Duchenne Muscular Dystrophy
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in…
- Trial name
- NCT03863119
- Testing
- Vamorolone
- Where
- 12 sites in United States, Canada and 1 more
- Sponsor
- Santhera Pharmaceuticals
Muscular Dystrophy, Duchenne
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in…
- Trial name
- CPI (NCT02195999)
- Testing
- Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophin (DMD)
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys.
- Trial name
- MUSCLE (NCT06594094)
- Testing
- HG302
- Where
- China
- Volunteers
- 4 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Becker Muscular Dystrophy
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG).
- Trial name
- NCT01539772
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 85 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Duchenne Muscular Dystrophy (DMD)
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and…
- Trial name
- NCT02036463
- Testing
- Prednisone
- Where
- United States
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Scoliosis Neuromuscular, Duchenne Muscular Dystrophy
Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device
This study will be conducted without blind method. The portable seat device devised to maintain lumbar lordosis will be made within 1 year after the loss of ambulation in the participants with Duchenne muscular dystrophy with…
- Conditions
- Scoliosis Neuromuscular, Duchenne Muscular Dystrophy, Lordosis Lumbar
- Trial name
- NCT03611244
- Testing
- Portable seat device devised to maintain lumbar lordosis
- Where
- 2 sites in South Korea
- Volunteers
- 98 people
- Sponsor
- Seoul National University Hospital
Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy
Muscle MRI Outlining of Neuromuscular Diseases Using Artificial Intelligence
Background and aim: Neuromuscular diseases encompass a range of conditions affecting muscle cells, nerves, or the interaction between the two.
- Conditions
- Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, Hypokalemic Periodic Paralysis
- Trial name
- NCT06917430
- Testing
- No intervention
- Volunteers
- 120 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Inherited Non-Duchenne Myopathies
Clinical and Functional Assessment of Patients With Inherited Non-Duchenne Myopathies in Sohag University Hospital
Muscular Myopathies are a heterogenous group of inherited muscular disorders characterized by progressive muscle weakness. Historically, these disorders are difficult to treat.
- Trial name
- NCT06574919
- Where
- 3 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Sohag University
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
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies.
The purpose of this study is to analyze the effectiveness of a 5-weeks respiratory digital intervention program in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Muscular Dystrophy
- Trial name
- DMDrespy2024 (NCT06363526)
- Testing
- Digital respiratory intervention program
- Where
- Spain
- Volunteers
- 12 people
- Sponsor
- University of Malaga
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Muscular Dystrophy, Duchenne and Becker Types
Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker.
Duchenne's Muscular Dystrophy and Becker Dystrophy, hereafter DMD and BMD, is a serious and progressive disease that affects 1 in 3,500-6,000 males born alive.
- Trial name
- RVDuchenne (NCT03879304)
- Testing
- multimodal physiotherapy program with RV, traditional physiotherapy program
- Where
- Spain
- Volunteers
- 12 people
- Sponsor
- University of Malaga
Duchenne Muscular Dystrophy (DMD)
Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
Objective: The objective of this observational study is to evaluate and quantify trunk muscle compensatory movement patterns in patients with Duchenne Muscular Dystrophy (DMD) using computer vision technology.
- Trial name
- NCT06755138
- Testing
- Observational Assessment
- Where
- South Korea
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Seoul National University Hospital
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege