NCT02084407

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Nov 2013

Shorter than P25 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2013

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

March 4, 2014

Completed
8 days until next milestone

First Posted

Study publicly available on registry

March 12, 2014

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2014

Completed
Last Updated

September 26, 2014

Status Verified

March 1, 2014

Enrollment Period

9 months

First QC Date

March 4, 2014

Last Update Submit

September 25, 2014

Conditions

DM1

Keywords

DM1CardiopathyAdult

Outcome Measures

Primary Outcomes (1)

  • Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells

    At baseline

Secondary Outcomes (1)

  • Molecular changes in human DM1 cardiomyocytes expressing pathogenic CUGexp-RNA

    At baseline

Study Arms (3)

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

Clinical examination, skin biopsy, blood and urine samplingOTHER
DM1 subject with cardiopathyDM1 subject without cardiopathyNot DM1subject

Eligibility Criteria

Age20 Years - 50 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • aged 20-50
  • DM1 confirmed by molecular analysis
  • Confirmed cardiomyopathy (infrahissiens conduction disorders)
  • able to sign the consent form
  • affiliated to european social security.
  • 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.
  • 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
  • +1 more criteria

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Institute of Myology

Paris, 75013, France

Location

MeSH Terms

Conditions

Heart Diseases

Interventions

Restraint, PhysicalBlood Specimen Collection

Condition Hierarchy (Ancestors)

Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Behavior ControlTherapeuticsImmobilizationInvestigative TechniquesSpecimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, Operative

Study Officials

  • Laurent Servais, MD PhD

    Institute of Myology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 4, 2014

First Posted

March 12, 2014

Study Start

November 1, 2013

Primary Completion

August 1, 2014

Study Completion

August 1, 2014

Last Updated

September 26, 2014

Record last verified: 2014-03

Locations

FR(1)