WithdrawnNot Applicable

Natural History Study in Patients With LGMDR5/2c

Stopped: lack of financing

France, Tunisia0Started 2025-01-10

Plain-language summary

Prospective, longitudinal, interventional, multicenter natural history study to better understand the LGMDR5/2c disease physiopathology. The duration of participation for each patient will be up to 24 months.

Who can participate

Age range6 Years – 35 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male and female patients 6 to less than 35 years of age * Ambulant patients should be less than 12 years of age and able to walk/run 10 meters (item 29 of NSAD) in less than 15 sec * Confirmed diagnosis of LGMDR5 (genotyping) * FVC \> 40% Exclusion Criteria: * Subjects with co-morbidity which may interfere with LGMDR5 natural evolution and/or evaluation of outcome measures * Need of non-invasive ventilation \> 16h per 24h or any invasive ventilation * Left ventricular ejection fraction (LVEF) \< 30% or prior heart failure decompensation requiring hospitalization * Past participation in a gene therapy or cell therapy trial

What they're measuring

North Star Assessment for Neuromuscular Disorders (NSAD)

Timeframe: Baseline through 24 months

Performance of Upper Limb (PUL)

Timeframe: Baseline through 24 months

Timed Up and Go (TUG)

Timeframe: Baseline through 24 months

100-meter walk/run test (100MWT)

Timeframe: Baseline through 24 months

Trial details

NCT IDNCT06210672

SponsorAtamyo Therapeutics

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-09-01

View on ClinicalTrials.gov More Gamma-sarcoglycanopathy trials