NCT02084407CompletedNot Applicable
Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients
Institut de Myologie, France2 sites in 1 country12 target enrollmentStarted: November 1, 2013Last updated:
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 12
- Locations
- 2
- Primary Endpoint
- Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells
Study Overview
Brief Summary
The objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Basic Science
- Masking
- None
Eligibility Criteria
- Ages
- 20 Years to 50 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •for DM1 patient with cardiomyopathy:
- •aged 20-50
- •DM1 confirmed by molecular analysis
- •Confirmed cardiomyopathy (infrahissiens conduction disorders)
- •able to sign the consent form
- •affiliated to european social security.
- •Inclusion Criteria for DM1 patient without cardiomyopathy
- •aged 20-50
- •DM1 confirmed by molecular analysis
- •No cardiomyopathy (no infrahissiens conduction disorders or other trouble linked to DM1)
- •able to sign the consent form
- •affiliated to european social security.
- •Inclusion Criteria for controls
- •aged over 18
- •brother, sister, father or mother of the DM1 patient
- •No DM1 confirmed by molecular analysis (CTG repetition under 38)
- •No cardiac disorders.
- •able to sign the consent form
- •affiliated to european social security.
Exclusion Criteria
- •no pragnant or nursing women
- •patient unable to understand informed consent
- •patient under juridic protection
Outcomes
Primary Outcomes
Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells
Time Frame: At baseline
Secondary Outcomes
- Molecular changes in human DM1 cardiomyocytes expressing pathogenic CUGexp-RNA(At baseline)
Investigators
Study Sites (2)
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