Condition: Leigh Syndrome (Maternally Inherited, MILS) · Leigh Syndrome (AR, AD, XR) · Sponsor: Charite University, Berlin, Germany
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.
This description comes directly from the study's public registry record.
Markus Schuelke, MD · +49 30 4505 66112 · markus.schuelke@charite.de
Alessandro Prigione, MD · +49 211 81 17687 · alessandro.prigione@hhu.de
Always discuss trial participation with your own doctor first.
| Universitätsklinikum Düsseldorf | Düsseldorf, North Rhine-Westphalia, Germany | Recruiting |
| Charite - Universtaetsmedizin Berlin | Berlin, State of Berlin, Germany | Recruiting |
Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.
We email about this public record only. Unsubscribe anytime with one click. Never medical advice.
This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT06967831