Clinical trials: Fascioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy, FSHD
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1
- Trial name
- FORTITUDE-3 (NCT07038200)
- Testing
- AOC-1020
- Where
- 46 sites in United States, Canada and 8 more
- Volunteers
- 200 people
- Sponsor
- Avidity Biosciences, Inc.
Facioscapulohumeral Muscular Dystrophy
A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD
This study is an observational study that aims to advance our knowledge on infantile onset FSHD. The study will include 50 participants of all ages who have presented with symptoms of FSHD between birth and 10 years of age.
- Trial name
- FSHD (NCT01437345)
- Where
- 12 sites in United States, Australia and 3 more
- Volunteers
- 53 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Primary Disease Fascioscapulohumeral Dystrophy (FSHD)
Routine Health Care of Patients With FSHD
On the basis of published data and our results indicating that oxidative stress may contribute to the peripheral skeletal muscle dysfunction in patients with FSHD, investigators conducted a pilot randomized double-blind placebo…
- Trial name
- FSHD (NCT02622438)
- Testing
- Followed with an initial assessment, 12 months and followed annually corresponding to the usual follow-up of the patient.
- Where
- France
- Volunteers
- 189 people
- Sponsor
- University Hospital, Montpellier
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 Muscular Dystrophy
Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
The purpose of this research study is to identify and study changes in muscle in people with facioscapulohumeral muscular dystrophy using magnetic resonance imaging and spectroscopy.
- Trial name
- NCT01671865
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
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
Neurological Observations, Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Dystrophy in Children
This study will focus on the symptoms, natural history and clinical impact of facioscapulohumeral muscular dystrophy (FSHD) in children. Symptoms of classical FSHD start in adulthood.
- Conditions
- Neurological Observations, Facioscapulohumeral Muscular Dystrophy, Pediatric Disorder
- Trial name
- iFocus (NCT02625662)
- Where
- Netherlands
- Volunteers
- 32 people
- Sponsor
- University Medical Center Nijmegen
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
Facio-Scapulo-Humeral Dystrophy
FSHD Molecular Characterization
To characterize the clinical and molecular phenotype of FSHD.
- Trial name
- NCT06096441
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Nationwide Children's Hospital
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
Digital Biotyping of FSHD Patients and Controls
Facioscapulohumeral muscular dystrophy (FSHD) is a devastating progressive muscle dystrophy. There is no treatment. FSHD is generally characterized by asymmetrical weakness and wasting of facial, shoulder girdle and upper arm…
- Trial name
- NCT04999735
- Testing
- CHDR Monitoring Remotely (MORE), Withings Steel HR, Withings Body+ scale
- Where
- Netherlands
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Centre for Human Drug Research, Netherlands
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
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.
Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in Study ATYR1940-C-003 (Stage 1 only) or Study ATYR1940-C-004 (that is, the parent studies).
- Trial name
- FSHD (NCT02836418)
- Testing
- ATYR1940
- Where
- 5 sites in United States, Denmark and 1 more
- Volunteers
- 8 people
- Sponsor
- aTyr Pharma, Inc.
FSH, FSH Muscular Dystrophy
BetterLife FSHD: A Patient-driven Health and Research Platform
BetterLife FSHD is a registry platform built to support people living with FSHD. It connects patients with personalized resources, tools, and research opportunities that match their health experiences and needs.
- Conditions
- FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1
- Trial name
- NCT07409142
- Testing
- Observational
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- FSHD Society
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
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Biomarker Development for Muscular Dystrophies
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Conditions
- Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
- Trial name
- NCT05019625
- Where
- 5 sites in United States
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Massachusetts General 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.
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.
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