Status:
UNKNOWN
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Lead Sponsor:
Genethon
Conditions:
LGMD2I
Eligibility:
All Genders
16-99 years
Brief 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 ...
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 ...
Eligibility Criteria
Inclusion
- Female and male patients
- Patients ≥ 16 years old
- Clinical diagnosis of LGMD2I and gene testing demonstrating two pathogenic mutations in fukutin-related protein gene, FKRP)
- Ambulant patients
Exclusion
- 1\. Patients presenting other disease which may significantly interfere with the interpretation of LGMD2I natural history
Key Trial Info
Start Date :
February 19 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 30 2023
Estimated Enrollment :
52 Patients enrolled
Trial Details
Trial ID
NCT03842878
Start Date
February 19 2020
End Date
December 30 2023
Last Update
April 6 2023
Active Locations (3)
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1
Pr John Vissing
Copenhagen, Denmark, DK-2100
2
Dr Tanya Stojkovic
Paris, France, 75013
3
Pr Volker Straub
Newcastle, United Kingdom, NE1 3BZ