Clinical trials: Facio-Scapulo-Humeral Dystrophy
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
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, 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.
Facioscapulohumeral Muscular Dystrophy
Rasch-analysis of Clinical Severity in FSHD
The purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD).
- Trial name
- ROC-FSHD (NCT02766985)
- Where
- 2 sites in United States
- Volunteers
- 44 people
- Sponsor
- University of Kansas Medical Center
Facioscapulohumeral Muscular Dystrophy
Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD)
On the basis of published data and the investigators' results indicating that oxidative stress may contribute to the peripheral skeletal muscle dysfunction in patients with FSHD, the investigators propose a study to test whether…
- Trial name
- FSHD (NCT01596803)
- Testing
- Taking of blood, needle biopsy of the vastus lateralis muscle, Vit C Vit E Zn Se
- Where
- France
- Volunteers
- 54 people
- Sponsor
- University Hospital, Montpellier
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
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, 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
Effectiveness of Upper Extremity Rehabilitation in pwFSHD (Patient With Facioscapulohumeral Dystrophia)
Facioscapulohumeral Muscular Dystrophy (FSHD) is one of the most common forms of muscular dystrophy, characterized by pronounced skeletal weakness and with a broad spectrum of diseases.
- Trial name
- NCT05178706
- Testing
- upper extremity rehabilitation program, Results of assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Istanbul University - Cerrahpasa
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
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
Facioscapulohumeral Muscular Dystrophy
Electrostimulation of Shoulder Girdle and Quadriceps Muscles in Facioscapulohumeral Muscular Dystrophy Patients
The investigators evaluated clinical tolerance, biological tolerance, feasibility and efficacy of daily electrostimulation training of shoulder girdle and quadriceps muscles in 10 patients with facioscapulohumeral muscular…
- Trial name
- NCT00821548
- Testing
- electrostimulation
- Where
- France
- Volunteers
- 9 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Inclusion Body Myositis, Myotonic Dystrophy
The Risk of Falls Index for Patients With Neuromuscular Disorders
The combination of short quantitatively assessing muscular function and balance in combination with short clinical scores, can be a new valid approach to evaluate the patient risk of fall and help to create a quick checkup test…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophies, Myasthenia Gravis
- Trial name
- NCT05890833
- Testing
- The risk of falls
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Muscular Dystrophies
Pro-inflammatory Cytokines in Facioscapulohumeral Muscular Dystrophy (CYTOKINE-FSH)
The facial-glenohumeral muscular dystrophy type 1 (DMFSH1) is characterized by a selective and asymmetrical involvement of the facial muscles, the shoulder girdle and the anterolateral lodge legs.
- Trial name
- CYTOKINE-FSH (NCT04694456)
- Testing
- cytokines dosage, test of walk, Manual muscular test
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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
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
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.
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.
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
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