Status:
RECRUITING
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
Lead Sponsor:
Charite University, Berlin, Germany
Collaborating Sponsors:
European Union
German Research Foundation
Conditions:
Leigh Syndrome (Maternally Inherited, MILS)
Leigh Syndrome (AR, AD, XR)
Eligibility:
All Genders
Brief Summary
In this project, the investigators are using iPSC lines derived from patients with Leigh syndrome that carry mutations in the mitochondrial (mtDNA) and in the nuclear DNA (nDNA) to reprogram them into...
Detailed Description
Leigh syndrome is a rare severe hereditary neurological disease that typically manifests during early childhood and is characterized by the progressive loss of motor and intellectual skills. A hallmar...
Eligibility Criteria
Inclusion
- Patient has a disease causing mutation in one of the genes causing Leigh syndrome if mutated, 2. Patient has the characteristic cranial MRI abnormalities of Leigh syndrome
Exclusion
- bleeding disorder that precludes a skin biopsy, 2. retraction of consent
Key Trial Info
Start Date :
March 1 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
February 28 2030
Estimated Enrollment :
80 Patients enrolled
Trial Details
Trial ID
NCT06967831
Start Date
March 1 2020
End Date
February 28 2030
Last Update
May 13 2025
Active Locations (2)
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1
Universitätsklinikum Düsseldorf
Düsseldorf, North Rhine-Westphalia, Germany, 40225
2
Charite - Universtaetsmedizin Berlin
Berlin, State of Berlin, Germany, 13353