Status:
ENROLLING_BY_INVITATION
Development of In Vitro Functional Assays From Primary Cells of Patients With Monogenic Diseases
Lead Sponsor:
Apteeus
Conditions:
Monogenic Disorders
Eligibility:
All Genders
Phase:
NA
Brief Summary
The objective of this project is to build a collection of biological samples consisting of fibroblasts and primary keratinocytes from patients with orphan monogenic diseases and to use these cells in ...
Eligibility Criteria
Inclusion
- Patients with an orphan monogenic disease (without any satisfactory therapeutic option).
- Patients with a pathology whose functional cause can be demonstrated by APTEEUS technologies in skin cells or cells derived from them.
Exclusion
- Patients whose molecular cause is not clearly identified.
- Patients contraindicated for skin biopsy.
Key Trial Info
Start Date :
September 6 2017
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 6 2025
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT03763864
Start Date
September 6 2017
End Date
September 6 2025
Last Update
August 2 2024
Active Locations (1)
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1
CHRU
Lille, France