NCT03384420

Who might be able to join this trial:

• You have been diagnosed with Pearson Syndrome, confirmed by a genetic test showing a problem in your mitochondrial DNA (the DNA found in the energy-producing parts of your cells).
• Your mother's mitochondrial DNA has been tested and shown to be normal.
• You are between 3 years old and just before your 18th birthday.
• You do not need regular blood transfusions to stay healthy.
• You have at least one of the following health issues: unusually high levels of lactic acid in your blood; episodes of serious metabolic (body chemistry) crises in the past year; kidney problems that do not require dialysis, or signs that the kidneys are not filtering properly; or poor growth or difficulty gaining weight as expected.

Who may not be able to join:

• No mitochondrial mutation or deletion can be found through testing.
• You or your mother has tested positive for a certain microbiological infection (confirm with trial site for exact details).
• You are not able to undergo a procedure called leukapheresis, which is a process that collects white blood cells from your blood.
• You have a serious long-term infection, cancer, or another condition that could put you at risk or make the study results harder to understand.
• You have previously received any cell therapy or gene therapy treatment.
• You have taken part in another clinical trial or received experimental medication within the past month.

Important: Always verify eligibility with the trial site directly before applying.