Clinical trials: Leigh Syndrome (AR, AD, XR)

Leigh Syndrome (Maternally Inherited, MILS), Leigh Syndrome (AR, AD, XR)

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

• Recruiting
• Age: All ages
• Observational study
• Surgery or procedure

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…

Trial name

cureMILS (NCT06967831)

Testing

skin biopsy, generation of iPSCs, blood drawing

Where

2 sites in Germany

Volunteers

80 people

Sponsor

Charite University, Berlin, Germany