Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients

Not Applicable

Completed

• Conditions: DM1
• Interventions: Other: Clinical examination, skin biopsy, blood and urine sampling

• Registration Number: NCT02084407
• Lead Sponsor: Institut de Myologie, France

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-11
• Status Verified: 2014-03
• Study First Submitted: 2014-03-04
• Study First Submitted QC: 2014-03-07
• Study First Posted: 2014-03-12
• Primary Completion: 2014-08
• Study Completion: 2014-08
• Last Update Submitted: 2014-09-25
• Last Update Posted: 2014-09-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

Exclusion Criteria

• no pragnant or nursing women
• patient unable to understand informed consent
• patient under juridic protection

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
DM1 subject without cardiopathy	Clinical examination, skin biopsy, blood and urine sampling	Clinical examination, Skin biopsy, Blood and urine sampling
Not DM1subject	Clinical examination, skin biopsy, blood and urine sampling	Clinical examination, Skin biopsy, Blood and urine sampling
DM1 subject with cardiopathy	Clinical examination, skin biopsy, blood and urine sampling	Clinical examination, Skin biopsy, Blood and urine sampling

Primary Outcome Measures

Name	Time	Method
Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells	At baseline

Secondary Outcome Measures

Name	Time	Method
Molecular changes in human DM1 cardiomyocytes expressing pathogenic CUGexp-RNA	At baseline

Trial Locations

Locations (1)

Institute of Myology; 🇫🇷 Paris, France