Clinical trials: Facioscapulohumeral Muscular Dystrophy (FSHD)
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1)
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD).
- Conditions
- Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- NCT04907162
- Testing
- Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory
- Where
- Germany
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- LMU Klinikum
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
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.
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
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
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
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
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.
Muscular Dystrophy, Facioscapulohumeral
Magnetic Resonance Imaging and Ultrasound Comparison With Load Evaluation
Facioscapulohumeral dystrophy (FSHD) is one of the most common hereditary neuromuscular disorders (NMD), with an estimated prevalence of 2000 patients in the Netherlands.
- Trial name
- MUSCLE+ (NCT06227182)
- Testing
- Muscle ultrasound with surface electromyography and dynamometry, MRI scan
- Where
- Netherlands
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Radboud University Medical Center
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.
Facioscapulohumeral Muscular Dystrophy (FSHD)
Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
This is a study to evaluate the safety and efficacy of losmapimod in treating participants with Facioscapulohumeral Muscular Dystrophy (FSHD).
- Trial name
- NCT05397470
- Testing
- Losmapimod
- Where
- 33 sites in United States, Canada and 7 more
- Volunteers
- 260 people
- Sponsor
- Fulcrum Therapeutics
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.
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
Facioscapulohumeral Muscular Dystrophy
Study of Testosterone and rHGH in FSHD
The purpose of this study is to investigate the safety and tolerability of combination therapy with recombinant human growth hormone (rHGH) and testosterone in adult male patients with facioscapulohumeral muscular dystrophy…
- Trial name
- STARFISH (NCT03123913)
- Testing
- Testosterone Enanthate, Somatropin
- Where
- United States
- Volunteers
- 20 people
- 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
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
Muscular Dystrophy, Facioscapulohumeral
Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy
OBJECTIVES: I. Determine whether albuterol increases strength in patients with facioscapulohumeral dystrophy as measured by quantitative voluntary isometric contraction testing. II.
- Trial name
- NCT00004685
- Testing
- albuterol
- Volunteers
- 90 people
- Sponsor
- Ohio State University