Trials / Completed
CompletedNCT03842878
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Limb-Girdle Muscular Dystrophy 2I
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 52 (actual)
- Sponsor
- Genethon · Academic / Other
- Sex
- All
- Age
- 16 Years – 99 Years
- Healthy volunteers
- Not accepted
Summary
Prospective, longitudinal, interventional, single-group, multicenter natural history study to better know the LGMD2I disease physiopathology. The duration of participation for each patient will be up to 24 months.
Detailed description
Study duration Duration from First visit of first patient (FPFV) to Last visit of last patient (LPLV) : 3 years Study objectives Primary objective: To characterize the disease course in Limb-Girdle Muscular Dystrophy 2I (LGMD2I) patients using standardized and disease appropriate evaluations. Secondary objectives: To identify clinical, imaging and/or laboratory parameters that are indicators of the disease course in LGMD2I To identify the best outcome measure for further therapeutics approaches
Conditions
Timeline
- Start date
- 2020-02-19
- Primary completion
- 2023-12-14
- Completion
- 2023-12-14
- First posted
- 2019-02-15
- Last updated
- 2026-02-12
Locations
3 sites across 3 countries: Denmark, France, United Kingdom
Source: ClinicalTrials.gov record NCT03842878. Inclusion in this directory is not an endorsement.