Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Launched by CHARITE UNIVERSITY, BERLIN, GERMANY · May 8, 2025

Keywords

ClinConnect Summary

This clinical trial is exploring new treatment options for Leigh syndrome, a serious genetic condition that affects how the body produces energy. The researchers are using specialized cells from patients with Leigh syndrome to test existing FDA-approved medications and see if they can help improve the health of brain cells affected by the disease. This process, called drug repurposing, could provide new hope for patients who currently have no standard treatment available.

To participate in the trial, individuals must have a genetic mutation linked to Leigh syndrome and show specific brain abnormalities on an MRI scan. The trial is open to all genders, and participants can expect to contribute to important research that could lead to new therapies. If successful, the drugs identified in this study may be used compassionately for patients who need them. This means that even though these drugs are not officially approved for Leigh syndrome yet, they could still be offered to patients in need.

Gender

ALL

Eligibility criteria

• • 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