Clinical trials: Facioscapulohumeral Muscular Dystrophy
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.
Primary Disease Facioscapulohumeral Dystrophy (FSHD)
Effects of NMES on Muscle Function of Patients With FSHD: a Double-blind Randomized Controled Clinical Trial
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant disease characterized by progressive weakness and atrophy of specific skeletal muscles.
- Trial name
- NEMS and FSHD (NCT02861911)
- Testing
- Current flow between sensory and motor threshold, Current intensity upper of motor threshold by Kneehab XP technology
- Where
- France
- Volunteers
- 9 people
- Sponsor
- University Hospital, Montpellier
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
Muscular Dystrophies, Scapular Dyskinesis
Evaluation of a Textile Scapula Orthosis
Shoulder instability due to muscle weakness is a common problem in disorders of the upper extremities. During arm motion, the scapula acts as a dynamic base for the humeral head.
- Conditions
- Muscular Dystrophies, Scapular Dyskinesis, Muscular Dystrophy, Facioscapulohumeral, Muscle Weakness
- Trial name
- ScapOrthosis (NCT04154098)
- Testing
- Scapula orthosis assistance, Manual scapula assistance, No assistance
- Where
- 2 sites in Germany, Switzerland
- Volunteers
- 8 people
- Sponsor
- Swiss Federal Institute of Technology
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
Facioscapulohumeral Muscular Dystrophy 1a
Muscle Inflammation and Fat Infiltration in Patients Affected by FSHD
Facioscapulohumeral muscular dystrophy (FSHD) is the second most common muscular dystrophy with approximately 500 patients in Denmark.
- Trial name
- NCT02541292
- Testing
- No intervention, observational
- Where
- Denmark
- Volunteers
- 10 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
Physical Training Introduction in Lifestyle of Facioscapulohumeral Dystrophy Patients
It is now accepted that physical activity is not deleterious in myopathies, including muscular dystrophies. In patients suffering from facioscapulohumeral dystrophy (FSHD), aerobic training has been reported to be associated to…
- Trial name
- FSHD1 (NCT01116570)
- Testing
- Physical training, Control
- Where
- 2 sites in France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Dystrophy
Intramuscular Transplantation of Muscle Derived Stem Cell and Adipose Derived Mesenchymal Stem Cells in Patients With Facioscapulohumeral Dystrophy (FSHD)
Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and myotonic dystrophy.
- Trial name
- NCT02208713
- Testing
- Intramuscular injection
- Where
- Iran
- Volunteers
- 21 people
- Sponsor
- Royan Institute
FSHD - Facioscapulohumeral Muscular Dystrophy
Imaging and Gait Analysis in FSHD Patients
Facioscapulohumeral muscular dystrophy (FSHD) is a common genetic muscle disorder characterized by progressive and often asymmetric muscle weakness, with high variability in clinical severity and disease progression.
- Trial name
- FSH-3DGait (NCT07164937)
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Muscular Dystrophy, Facioscapulohumeral
Clenbuterol to Target DUX4 in FSHD
The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three different dose levels.
- Trial name
- Target FSHD (NCT06721299)
- Testing
- Clenbuterol
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Jeffrey Statland
Facioscapulohumeral Muscular Dystrophy (FSHD)
A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult…
- Trial name
- MANOEUVRE (NCT05548556)
- Testing
- RO7204239
- Where
- 9 sites in United States, Denmark and 2 more
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
FSHD - Facioscapulohumeral Muscular Dystrophy
1 Year MRI Followup in Facioscapulohumeral Muscular Dystrophy
An investigation of disease progression in adult danish patients with facioscapulohumeral muscular dystrophy. The disease progression is followed for a year with two test-days, including functional muscle tests and a MRI scan of…
- Trial name
- FSHD (NCT02159612)
- Testing
- MRI
- Where
- Denmark
- Volunteers
- 45 people
- Sponsor
- Grete Andersen, MD
Developmental Disability, Intellectual Disability
Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort
The purpose of this research use only (RUO) study is to detect genomic structural variants (SVs) in human DNA by Optical Genome Mapping (OGM) using the Bionano Genomics Saphyr system.
- Conditions
- Developmental Disability, Intellectual Disability, Autism Spectrum Disorder, Congenital Anomaly
- Trial name
- NCT05295277
- Testing
- Standard of care genetic testing group
- Where
- 8 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Bionano Genomics
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
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 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