iPS Cells of Patients for Models of Retinal Dystrophies

Retinal Dystrophies Clinical Trial

— RETIPS  

Official title:

iPS Cells of Patients for Models of Retinal Dystrophies

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03853252
• Other study ID #: 9366
• Status: Recruiting
• Phase: N/A
• Start date: November 3, 2014
• Est. completion date: November 2024

Study information

• Verified date: December 2022
• Source: University Hospital, Montpellier
• Contact: Vasiliki Kalatzis, PhD
• Phone: (0)499636097
• Email: vasiliki.kalatzis[@]inserm.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: November 2024
• Est. primary completion date: November 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Retinal Dystrophies

Intervention

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

Locations

Country	Name	City	State
France	CNMR Maolya, Genetic Sensory Diseases	Montpellier	Occitanie

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital, Montpellier	Institut National de la Santé Et de la Recherche Médicale, France

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	number of human cell models obtained		10 years