Actively Recruiting
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
Led by Charite University, Berlin, Germany · Updated on 2025-05-13
80
Participants Needed
2
Research Sites
521 weeks
Total Duration
On this page
Sponsors
C
Charite University, Berlin, Germany
Lead Sponsor
E
European Union
Collaborating Sponsor
AI-Summary
What this Trial Is About
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 neural progenitor cells and into dopaminergic neurons. The researchers are using this experimental system to screen FDA (Food and Drug Administration, USA) and EMA (European Medicines Agency) approved drugs for a positive effect on Leigh patient-derived neuronal cells (drug repurposing) using various biochemical, optic, and morphological outcome measures. Confirmed positive hits may be used for compassionate off-label use in Leigh patients when no standard treatment is available.
CONDITIONS
Official Title
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient has a disease causing mutation in one of the genes causing Leigh syndrome if mutated
- Patient has the characteristic cranial MRI abnormalities of Leigh syndrome
You will not qualify if you...
- Bleeding disorder that precludes a skin biopsy
- Retraction of consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Universitätsklinikum Düsseldorf
Düsseldorf, North Rhine-Westphalia, Germany, 40225
Actively Recruiting
2
Charite - Universtaetsmedizin Berlin
Berlin, State of Berlin, Germany, 13353
Actively Recruiting
Research Team
M
Markus Schuelke, MD
CONTACT
A
Alessandro Prigione, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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