Clinical trials: FSHD
Facioscapulohumeral Muscular Dystrophy 1
Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1
Facioscapulohumeral muscular dystrophy (FSHD) is characterized by clinical diversity, with FSHD1 being the most common form. It is associated with a toxic gain of function of the Double homeobox 4 (DUX4) gene, leading to muscle…
- Trial name
- REINFORCE (NCT06222827)
- Testing
- Satralizumab Prefilled Syringe
- Where
- 2 sites in Canada, France
- Volunteers
- 46 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
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.
FSHD
New Biomarkers in Facioscapulohumeral Muscular Dystrophy, Multispectral Optoacoustic Tomography.
Facioscapulohumeral Muscular Dystrophy (FSHD) is one of the most frequent muscular dystrophies in the adulthood. Multi-spectral optoacoustic tomography (MSOT) is an innovative imaging technique able to non-invasively characterize…
- Trial name
- MSOT-FSHD (NCT05902884)
- Testing
- MSOT Acuity Echo
- Where
- Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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
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
Facioscapulohumeral Muscle Dystrophy
Protein Supplementation and Exercise in Patients With FSH Muscular Dystrophy- a Randomized Placebo Controlled Study
The hypotheses is that regular post exercise supplementation increase fitness and daily activity level in patients with Fascioscapulohumeral (FSH) muscular dystrophy.
- Trial name
- FSHD (NCT01618331)
- Testing
- Regular exercise, Protein-carbohydrate supplementation
- Where
- Denmark
- Volunteers
- 42 people
- Sponsor
- Grete Andersen, MD
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
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 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 (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.
Shoulder Pain, Neuromuscular Diseases
Understanding Control and Mechanisms of Shoulder Instability in FSHD
The aim of this study is to identify factors for shoulder instability in people with Facioscapulohumeral dystrophy (FSHD). FSHD is a non-life limiting condition with symptoms presenting in the second decade of life (Evangelista…
- Conditions
- Shoulder Pain, Neuromuscular Diseases, Shoulder Injuries, Facio-Scapulo-Humeral Dystrophy
- Trial name
- NCT05239520
- Testing
- 3D movement analysis with surface electromyography and ultrasound
- Where
- United Kingdom
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- University of Liverpool
Muscular Dystrophy, Facioscapulohumeral
Prospective Study for 24-months of Physical Training Introduced in Lifestyle of Patients With FSHD : Tolerance, Sustainability and Efficiency of Unsupervised Training Program.
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 with…
- Trial name
- FSHD2 (NCT01689480)
- Testing
- FSHD training
- Where
- 2 sites in France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
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
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue.
- Conditions
- Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1
- Trial name
- NCT00082108
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- University of Rochester
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.
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
Muscular Dystrophy, Facioscapulohumeral
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
- Trial name
- MOVE Peds (NCT06847282)
- Where
- 7 sites in United States, Australia
- Volunteers
- 80 people
- Sponsor
- University of Kansas Medical Center
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