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 range

6 Years – 35 Years

Sex

ALL

See this in plain English?

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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

Who can participate

Age range

6 Years – 35 Years

Sex

ALL

See this in plain English?

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