Natural History Study in Patients With LGMDR5/2c

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2025-01-10

Study Completion Date

2025-09-01

Brief Summary

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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.

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Detailed Description

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Study duration Duration from First visit of first patient (FPFV) to Last visit of last patient (LPLV) : 3 years. Each patient will have a visit every 6 months over 2 years (5 visits in total).

Study primary objective is to characterize the disease course in gamma-sarcoglycanopathy (LGMDr5/2c) patients using standardized and disease appropriate evaluations.

Secondary objectives are to identify clinical, imaging and/or laboratory parameters that are indicators of the disease course in LGMD2c and to identify the best outcome measure for further therapeutics approaches

Conditions

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Gamma-sarcoglycanopathy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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

Minimum Eligible Age

6 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No