Clinical trials: Muscular Dystrophy
Cerebral Palsy, Spina Bifida
A Peer E-mentoring Intervention to Improve Employment
Despite the strong business case of hiring people with disabilities, a significant proportion of youth with disabilities leave high school and neither work nor continue their education and are unprepared to meet the demands of a…
- Conditions
- Cerebral Palsy, Spina Bifida, Juvenile Arthritis, Spinal Cord Injury
- Trial name
- NCT02522507
- Testing
- Empowering youth towards employment
- Where
- Canada
- Volunteers
- 44 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Type 1 Facioscapulohumeral Muscular Dystrophy
New Clinical Outcome Measures to Remotely Evaluate Patients With FacioScapuloHumeral Muscular Dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common adult muscular dystrophy with an estimated prevalence range of 2-7 per 100,000.
- Trial name
- PROGRESS-FSHD (NCT05812144)
- Testing
- Remote monitoring program
- Where
- 3 sites in France
- Volunteers
- 70 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Limb-Girdle Muscular Dystrophy, Social Competence
Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular Dystrophy
This clinical trial aims to evaluate the efficacy of a telecare-mediated psychosocial and cognitive intervention for children and adolescents aged 7 to 17 with limb-girdle muscular dystrophy.
- Conditions
- Limb-Girdle Muscular Dystrophy, Social Competence, Self Esteem, Health Related Quality of Life
- Trial name
- TPCLGMD (NCT07711730)
- Testing
- Neuro-psychosocial teleassistance program
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- University of Deusto
Myotonic Dystrophy
The United Kingdom National Registry for Myotonic Dystrophy
Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene.
- Trial name
- NCT04003363
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 900 people
- Sponsor
- Newcastle University
LGMD2I
Study of BBP-418 in Patients With LGMD2I
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the…
- Trial name
- NCT04800874
- Testing
- BBP-418
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- ML Bio Solutions, Inc.
Duchenne Muscular Dystrophy
Treatment of a Single Patient With CRD-TMH-001
The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.
- Trial name
- NCT05514249
- Testing
- CRD-TMH-001
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Cure Rare Disease, Inc
Duchenne Muscular Dystrophy
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the…
- Trial name
- NCT01753804
- Testing
- Observational study
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 269 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis
Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
The hypothesis is that a mechanical insufflation-exsufflation (MI-E) is associated with a decrease in the number of intubations and more rapid clinical improvement in children and adults with neuromuscular disease who are…
- Conditions
- Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases
- Trial name
- Nemucough (NCT00839033)
- Testing
- mechanical insufflation - exsufflation, Standard respiratory physiotherapy
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.