RETIPS: iPS Cells of Patients for Models of Retinal Dystrophies

Sponsor
University Hospital, Montpellier (Other)
Overall Status
Recruiting
CT.gov ID
NCT03853252
Collaborator
Institut National de la Santé Et de la Recherche Médicale, France (Other)
150
1
1
120
1.3

Study Details

Study Description

Brief Summary

The investigators are focused on inherited retinal dystrophies with an aim to further understand disease pathophysiology and to elaborate novel treatments, as, to date, there is no effective treatment to prevent blindness.

The main goal of this study is to generate human cellular models of healthy and disease retinas and perform studies to evaluate the efficiency of gene therapy approaches for different diseases.

Skin biopsies of volunteers are cultured to isolate fibroblasts that are then reprogrammed into iPS cells. Healthy and disease-specific iPS cells are then differentiated into retinal models.

This study should help to elucidate disease pathways and to provide proof-of-concept for various therapeutic approaches.

Condition or Disease Intervention/Treatment Phase
  • Other: Skin biopsy
N/A

Study Design

Study Type:
Interventional
Anticipated Enrollment :
150 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Other
Official Title:
iPS Cells of Patients for Models of Retinal Dystrophies
Actual Study Start Date :
Nov 3, 2014
Anticipated Primary Completion Date :
Nov 1, 2024
Anticipated Study Completion Date :
Nov 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Other: Skin biopsy

Other: Skin biopsy
Skin biopsy on a location preliminarily anesthetized Disinfection protocol Combined required blood tests (HIV, Hepatitis B)

Outcome Measures

Primary Outcome Measures

  1. number of human cell models obtained [10 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:
5 Years to 70 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion criteria :
  • Signed informed consent and

  • Choroideremia :

  • Males

  • CHM mutation

  • With multimodal Imaging anomalies in line with CHM

  • All other presumed inherited retinal dystrophies with bilateral and symmetrical involvement with identified mutations in one of the Retnet gene

  • All presumed inherited optic neuropathy with bilateral and symmetrical involvement with identified mutations

  • And in all cases or pattern

  • Age from 5 to 70

  • with appropriate health insurance

Exclusion criteria :
  • Patient under tutorship or curatorship

Contacts and Locations

Locations

Site City State Country Postal Code
1 CNMR Maolya, Genetic Sensory Diseases Montpellier Occitanie France 34294

Sponsors and Collaborators

  • University Hospital, Montpellier
  • Institut National de la Santé Et de la Recherche Médicale, France

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
University Hospital, Montpellier
ClinicalTrials.gov Identifier:
NCT03853252
Other Study ID Numbers:
  • 9366
First Posted:
Feb 25, 2019
Last Update Posted:
Mar 31, 2020
Last Verified:
Mar 1, 2020
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Mar 31, 2020