Clinical trials: Fascioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy…
- Trial name
- FSHD (NCT06907875)
- Testing
- EPI-321
- Where
- 7 sites in United States, Australia and 1 more
- Volunteers
- 12 people
- Sponsor
- Epicrispr Biotechnologies, Inc.
Muscular Dystrophy, Duchenne, Orthotic Devices
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.
- Conditions
- Muscular Dystrophy, Duchenne, Orthotic Devices, Upper Extremity, Neuromuscular Diseases (NMD)
- Trial name
- NCT06363357
- Testing
- Shoulder orthosis
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Seoul National 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.
Muscular Dystrophy, Facioscapulohumeral
Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1).
- Trial name
- NCT06131983
- Testing
- SRP-1001 for Injection
- Where
- 16 sites in Australia, Canada and 5 more
- Volunteers
- 60 people
- Sponsor
- Sarepta Therapeutics, Inc.
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Muscle Diseases
Acceptance and Commitment Therapy for Muscle Disease
In adults, muscle diseases are usually chronic long-term conditions that do not have a definitive cure. Supportive care has been shown to reduce complications from muscle disease and improved survival in some cases.
- Trial name
- ACTMuS (NCT02810028)
- Testing
- Acceptance and Commitment Therapy (ACT)
- Where
- United Kingdom
- Volunteers
- 155 people
- Sponsor
- King's College Hospital NHS Trust
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Facio-Scapulo-Humeral Dystrophy
Computerized Facial Recognition for Automated Diagnosis of the Facio-Scapulo-Humeral Muscular Dystrophy (FSMHD)
The clinical diagnosis of Facio-Scapulo-Humeral Muscular Dystrophy (FSHMD) requires the movement of patients to a medical centre and a lengthy examination involving medical personnel, and may be underestimated in the most…
- Trial name
- CV4DIAGNOSIS (NCT04377217)
- Testing
- Video recording
- Where
- France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Nice
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
Unraveling Metabolic Involvement in Facioscapulohumeral Dystrophy Through Metabolomics
The pathogenesis of facioscapulohumeral dystrophy (FSHD), one of the most prevalent types of inherited muscle disease, is unknown.
- Trial name
- METIN-FSHD (NCT06086548)
- Testing
- metabolomic on plasma sample
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University Hospital, Angers
Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Study Evaluating MYO-029 in Adult Muscular Dystrophy
The purpose of this phase I/II, multicenter, safety trial is to study MYO-029 in adult patients with muscular dystrophy.
- Conditions
- Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
- Trial name
- NCT00104078
- Testing
- MYO-029
- Where
- 9 sites in United States
- Volunteers
- 108 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
FSHD, FSHD1
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE-OLE (NCT06547216)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 84 people
- Sponsor
- Avidity Biosciences, Inc.
Facioscapulohumeral Muscular Dystrophy
Arm Cycling in Facioscapulohumeral Dystrophy (FSHD) Patients
The investigators believe that arm cycling has the potential to be effective in maintaining or improving shoulder muscle functioning. However, there is no evidence for its safety or effectiveness in the upper extremity.
- Trial name
- NCT04267354
- Testing
- Arm cycling
- Volunteers
- 15 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
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
FSHD - Facioscapulohumeral Muscular Dystrophy
Ten Year Follow-up in FSHD: the FOCUS 3 Study
This study aims to assess longitudinal data in 170 adult and 30 pediatric genetically and clinically well-defined facioscapulohumeral dystrophy (FSHD) patients.
- Trial name
- FSHD-FOCUS 3 (NCT06911190)
- Where
- Netherlands
- Volunteers
- 200 people
- Sponsor
- Radboud University Medical Center
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.
Facio-Scapulo-Humeral Dystrophy
Examination of Operative Approach in pwFSHD (Patient With Facioscapulohumeral Muscular Dystrophy)
Facioscapulohumeral Muscular Dystrophy (FSHD) is one of the most common forms of muscular dystrophy, characterized by pronounced skeletal novelistic weakness and with a broad spectrum of diseases.
- Trial name
- NCT05022355
- Where
- Turkey (Türkiye)
- Volunteers
- 24 people
- Sponsor
- Istanbul University - Cerrahpasa
Facioscapulohumeral Muscular Dystrophy
Quantitative Assessment of Orofacial Muscle Function in FSHD
The goal of this study is to validate a new method for the assessment of orofacial muscles in FSHD affected individuals
- Trial name
- NCT06425445
- Testing
- Orofacial function assessment tests
- Where
- Belgium
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Mons
Muscular Dystrophy, Facioscapulohumeral
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups.
- Trial name
- NCT06517498
- Testing
- No Intervention
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Xi'an Jiaotong University
Fascioscapulohumeral Muscular Dystrophy
Dietary Protein Requirements in Adults With Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common types of muscular dystrophy, affecting about 4 out of 100,000 individuals.
- Trial name
- FSHD (NCT06785428)
- Testing
- Amino Acid Intake
- Where
- 2 sites in Canada
- Volunteers
- 10 people
- Sponsor
- McGill University