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Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Recruitingn/aAges any - anyAllCharite University, Berlin, Germany
NCT06967831 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
Inclusion Criteria: 1. 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 Criteria: 1. bleeding disorder that precludes a skin biopsy, 2. retraction of consent
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What it tests

  • skin biopsy
  • generation of iPSCs
  • blood drawing
  • off-label compassionate drug use
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Where

2 sites
Universitätsklinikum Düsseldorf
Düsseldorf, Germany
Charite - Universtaetsmedizin Berlin
Berlin, Germany
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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