Clinical trials: Facio-Scapulo-Humeral Dystrophy
FSHD, FSHD1
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with…
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE (NCT05747924)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 90 people
- Sponsor
- Avidity Biosciences, 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.
Facioscapulohumeral Dystrophy
Walking ANalysis Interest in Persons wiTh facioscapulohumEral Muscular Dystrophies
Facioscapulohumeral muscular dystrophy (FSH) is one of the most common genetic myopathies in adults. It is characterised by progressive asymmetric muscular atrophy affecting the skeletal muscles of the face, upper limbs and then…
- Trial name
- WANTED (NCT06600308)
- Testing
- Walk tests, ecological (real-life) assessment of walking ability
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
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
FSH Muscular Dystrophy, Muscular Dystrophy
Amino Acids and Exercise in FSHD
This study evaluates whether amino acid supplementation, combined with a structured diet and exercise program, improves body composition (increased muscle mass and reduced fat mass) and physical performance in individuals with…
- Trial name
- NCT07543016
- Testing
- Essential Amino Acids Supplementation intervention, Exercise, Diet
- Where
- Italy
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- University of Pavia
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 (FSHD)
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
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
- NCT02239224
- Testing
- ATYR1940
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 20 people
- Sponsor
- aTyr Pharma, Inc.
Facioscapulohumeral Muscular Dystrophy
The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry
Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000.
- Trial name
- NCT04001582
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,018 people
- Sponsor
- Newcastle University
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
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
Facioscapulohumeral Muscular Dystrophy
Bone Health in Facioscapulohumeral Muscular Dystrophy
This is a cross-sectional single visit study to determine bone health in individuals with FSHD.
- Trial name
- FSHD (NCT02413190)
- Where
- 2 sites in United States, Australia
- Volunteers
- 94 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Facioscapulohumeral Muscular Dystrophy
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy…
- Trial name
- NCT02927080
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 95 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]
Until twenty years ago physical exercise in muscular dystrophies was considered harmful to the muscle cells, inducing an acceleration of cell necrosis.
- Conditions
- Muscular Dystrophies, Limb Girdle Muscular Dystrophy, Facio-Scapulo-Humeral Dystrophy, Becker Muscular Dystrophy
- Trial name
- RIMUDI (NCT06378203)
- Testing
- tele-rehabilitation system with virtual reality Home Kit - Virtual Reality Rehabilitation System (VRRS KHYMEIA)
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Eugenio Medea
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
FSHD - Facioscapulohumeral Muscular Dystrophy
High Intensity Training in Patients With Facioscapulohumeral Muscular Dystrophy
The investigators aim to investigate the effect of high-intensity training in patients with facioscapulohumeral muscular dystrophy. Can patients benefit from this type of exercise without muscle damage.
- Trial name
- HIT-FSHD (NCT02159963)
- Testing
- Supervised training, Unsupervised training, Optional training
- Where
- Denmark
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Grete Andersen, MD
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
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
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
Facioscapulohumeral Muscular Dystrophy Type 2
An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common inherited myopathies in adults. It is associated with genetic and epigenetic deregulation of the D4Z4 locus on the sub-telomeric region of chromosome 4q35…
- Trial name
- INSIGHT FSHD2 (NCT06079567)
- Testing
- Validation of new COMs for FSHD2 patients
- Where
- 9 sites in Belgium, France and 3 more
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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