Induction of Pluripotent Stem Cells From Human Fibroblast... | NCT02084407

Studies
Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients

NCT02084407

Sponsor

Institut de Myologie, France

Status

Completed

Last Update Posted

Sep 26, 2014Estimated

Enrollment

12Actual

Phase

Not Applicable

Conditions

Interventions

Clinical examination, skin biopsy, blood and urine sampling

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2014

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2013

Primary Completion Date

Aug 2014Actual

Completion Date

Aug 2014Actual

First Submitted Date

Mar 4, 2014

First Submission Date that Met QC Criteria

Mar 7, 2014

First Posted Date

Mar 12, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 25, 2014

Last Update Posted Date

Sep 26, 2014Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
DM1 subject with cardiopathy	Active Comparator	Clinical examination, Skin biopsy, Blood and urine sampling	Other: Clinical examination, skin biopsy, blood and urine sampling
DM1 subject without cardiopathy	Active Comparator	Clinical examination, Skin biopsy, Blood and urine sampling	Other: Clinical examination, skin biopsy, blood and urine sampling
Not DM1subject	Active Comparator	Clinical examination, Skin biopsy, Blood and urine sampling	Other: Clinical examination, skin biopsy, blood and urine sampling

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Clinical examination, skin biopsy, blood and urine sampling	Other		DM1 subject with cardiopathy DM1 subject without cardiopathy Not DM1subject

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Molecular changes in human DM1 cardiomyocytes expressing pathogenic CUGexp-RNA		At baseline

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

20 Years

Maximum Age

50 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Laurent Servais, MD PhD	Institute of Myology	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Institute of Myology		Paris		75013	France

ID	Term
D006331	Heart Diseases

ID	Term
D002318	Cardiovascular Diseases

Intervention Browse Module

MeSH Terms

ID	Term
D012149	Restraint, Physical
D001800	Blood Specimen Collection

Ancestor Terms

ID	Term
D032763	Behavior Control
D013812	Therapeutics
D007103	Immobilization
D008919	Investigative Techniques

Browse Leaves

Not provided

Browse Branches

Not provided