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This study examined new clinical outcome measures for remotely assessing patients with facioscapulohumeral muscular dystrophy (FSHD) through a remote monitoring program. It enrolled patients aged 18-75 with genetically confirmed FSHD1 or characteristic findings and symptomatic limb weakness. It is a study without an assigned phase and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion criteria: * Genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring (40) * Age 18-75 years * Symptomatic limb weakness * Patient able to walk alone or with a walking aid. * Patient affiliated to the social security system * Patient giving written consent after written and oral information. * If taking over the counter supplements willing to remain consistent with supplement regimen throughout the course of the study Non inclusion criteria: * Patients with comorbidity not related to the disease that can modify the natural evolution of the disease or would interfere with safe testing in the opinion of the Investigator * Regular use of available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists * Use of an experimental drug in an FSHD clinical trial within the past 30 days * Pregnant or nursing women for women of childbearing age * Patient protected by law, under guardianship or curator ship, or not able to participate in a clinical study according to the article L.1121-16 of the French Public Health Code