A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

This is an interventional treatment trial for Mitochondrial Diseases focused on measuring Autologous stem cell transplantation, Mitochondrial Diseases, Pearson Syndrome, Rare Diseases Read More

Inclusion Criteria

1. Patient diagnosed with Pearson Syndrome, as verified by molecular identification of a defect in the mitochondrial DNA.
2. Normal maternal mitochondria as verified by mtDNA sequencing.
3. Males and females between 3 years or older and up to 18th birthday.
4. Patient is transfusion independent.

5. Patient has at least one of the following systematic involvements:

   1. High baseline lactate levels
   2. Episodes of metabolic crisis in the last year before pre-screening
   3. Renal failure (not dependent on dialysis) or evidence of proximal tubulopathy
   4. Growth retardation or failure to thrive

Exclusion Criteria

1. Absence of detectable mitochondria mutation or deletion.
2. Patient or patient's mother have a positive test for microbiologic
3. Patient is unable to undergo leukapheresis.
4. Patient suffers from chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results.
5. Patient has been treated previously with any cell or gene therapy.
6. Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.