Clinical trials: FSHD, active
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.
FSHD1, FSHD2
Longitudinal Study on Diaframmatic Ultrasound in FSHD Patients
The goal of this prospective, longitudinal, single-center study is to describe respiratory function in patients affected by FSHD at baseline and after one year using both diaphragmatic ultrasound and pulmonary function test.
- Trial name
- NCT06078852
- Testing
- Diaphragmatic ultrasound, Pulmonary function test
- Where
- Italy
- Volunteers
- 34 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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.
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
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.
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
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.
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
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy…
- Trial name
- FSHD (NCT06907875)
- Testing
- EPI-321
- Where
- 7 sites in United States, Australia and 1 more
- Volunteers
- 12 people
- Sponsor
- Epicrispr Biotechnologies, Inc.
Facioscapulohumeral Muscular Dystrophy Type 2
An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common inherited myopathies in adults. It is associated with genetic and epigenetic deregulation of the D4Z4 locus on the sub-telomeric region of chromosome 4q35…
- Trial name
- INSIGHT FSHD2 (NCT06079567)
- Testing
- Validation of new COMs for FSHD2 patients
- Where
- 9 sites in Belgium, France and 3 more
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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.
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
FSHD
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided.
- Trial name
- MOVE FSHD (NCT04635891)
- Where
- 21 sites in United States, Brazil and 2 more
- Volunteers
- 450 people
- Sponsor
- University of Kansas Medical Center
Facioscapulohumeral Muscular Dystrophy, FSHD
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
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
FSHD
Testing a Tailored Home Exercise Program to Reduce Pain and Fatigue in Patients with FSHD.
The goal of this intervention study is to determine the effect of a tailored exercise program (with aerobic, strength and balance training) in patients with FSHD.
- Trial name
- GRIPonFSHD (NCT06712043)
- Testing
- Personalised Exercise, Historical control group
- Volunteers
- 50 people
- Sponsor
- Radboud University Medical Center
Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy
Muscle MRI Outlining of Neuromuscular Diseases Using Artificial Intelligence
Background and aim: Neuromuscular diseases encompass a range of conditions affecting muscle cells, nerves, or the interaction between the two.
- Conditions
- Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, Hypokalemic Periodic Paralysis
- Trial name
- NCT06917430
- Testing
- No intervention
- Volunteers
- 120 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
Muscular Dystrophy, Facioscapulohumeral
Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study
The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a…
- Trial name
- ReSOLVE_France (NCT04038138)
- Testing
- Validation of new COA for FSHD patients
- Where
- 3 sites in France
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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