Clinical trials: Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy (FSHD)
Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
This is a study to evaluate the safety and efficacy of Losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.
- Trial name
- FSHD (NCT04003974)
- Testing
- Losmapimod oral tablet
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 80 people
- Sponsor
- Fulcrum Therapeutics
Facioscapulohumeral Muscular Dystrophy 1
Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
This clinical trial is a study to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments of Losmapimod for patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with an open-label…
- Trial name
- FSHD (NCT04004000)
- Testing
- Losmapimod
- Where
- Netherlands
- Volunteers
- 14 people
- Sponsor
- Fulcrum Therapeutics
Facioscapulohumeral Muscular Dystrophy (FSHD)
A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD
The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of SFL-0821 in adult patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
- Trial name
- NCT07798609
- Testing
- SFL-0821 for injection
- Where
- 6 sites in Australia, New Zealand
- Volunteers
- 90 people
- Sponsor
- Soufflé Therapeutics, Inc.
Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)
A Registered Cohort Study on FSHD1
The data to be collected is intended to help healthcare providers make important medical and financial decisions concerning FSHD1, through an enhanced understanding of the prevalence, progression and natural history of FSHD1.
- Trial name
- NCT04369209
- Where
- China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Ning Wang, MD., PhD.
Facioscapulohumeral Muscular Dystrophy
Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy.
- Trial name
- ReSolve (NCT03458832)
- Testing
- FSHD-specific functional rating scale, Electrical Impedance Myography
- Where
- 14 sites in United States, France and 4 more
- Volunteers
- 324 people
- Sponsor
- University of Kansas Medical Center
Facioscapulohumeral Muscular Dystrophy
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies.
- Trial name
- NCT02531217
- Testing
- ATYR1940
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 9 people
- Sponsor
- aTyr Pharma, Inc.
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Facioscapulohumeral Muscular Dystrophy (FSHD)
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.
- Trial name
- FSHD (NCT02603562)
- Testing
- ATYR1940
- Where
- 7 sites in United States, France and 1 more
- Volunteers
- 8 people
- Sponsor
- aTyr Pharma, Inc.
Myotonic Muscular Dystrophy
Study of Muscle Wasting and Altered Metabolism in Patients With Myotonic Dystrophy
OBJECTIVES: I. Examine the interrelationships between muscle wasting (phenotype), the degree of myotonic dystrophy (DM) gene expression (genotype) in patients with DM. II. Characterize the insulin resistance in these patients.
- Trial name
- NCT00004769
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Facio-Scapulo-Humeral Dystrophy, FSHD2
Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
This multi-centre, randomised, double-blind, placebo-controlled crossover trial will compare changes in strength-related motor function following treatment with creatine monohydrate to treatment with placebo, as measured by the…
- Conditions
- Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
- Trial name
- NCT02948244
- Testing
- Creatine Monohydrate
- Where
- Australia
- Volunteers
- 11 people
- Sponsor
- Murdoch Childrens Research Institute
Facioscapulohumeral Muscular Dystrophy
ADVANCED FSHD-COM: New Clinical Outcome Measures to Evaluate Non-ambulant FSHD Patients, a Pilot Study
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
- ADVANCED (NCT05453461)
- Testing
- Validation of new COMs for non ambulant FSHD patients
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Facioscapulohumeral Muscular Dystrophy (FSHD)
A Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
This is a two-part, Phase 2, randomized, placebo-controlled, double-blind study, followed by a randomized active treatment period, to evaluate the safety and efficacy of orally administered forazapadin (SAT-3247) in adult…
- Trial name
- NCT07872306
- Testing
- forazapadin
- Volunteers
- 51 people
- Sponsor
- Satellos Bioscience, Inc.
Muscular Dystrophy, Facioscapulohumeral
Study of Morphology and Functional Magnetic Resonance Imaging (MRI) Muscle Patients With Muscular Dystrophy Type FSHD Benefiting a Physical Training Introduced.
The safety of guided practice of physical activity in myopathies is increasingly accepted, including muscular dystrophies. In facioscapulohumeral dystrophy (FSHD), one of the most common muscular dystrophy, the aerobic training…
- Trial name
- FSHD3 (NCT01990976)
- Testing
- MRI, Biopsy
- Where
- 2 sites in France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Muscular Dystrophy, Facioscapulohumeral
Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study
The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a…
- Trial name
- ReSOLVE_France (NCT04038138)
- Testing
- Validation of new COA for FSHD patients
- Where
- 3 sites in France
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Nice