Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome
Mitochondrial Diseases, Pearson Syndrome
About this trial
This is an interventional treatment trial for Mitochondrial Diseases focused on measuring Autologous, Mitochondrial, Pearson, Transplantation, Stem cell
Eligibility Criteria
Inclusion Criteria: Male or female participants aged from 1 to 18 years old. Diagnosis of Pearson Syndrome, mtDNA deletion verified by NGS. Body weight ≥ 10 kg. Participant has anemia or thrombocytopenia, or leukopenia and/or blood transfusion dependent (receives blood transfusions every 6 weeks or less). Participant is medically able to undergo the study interventions, as determined by the investigator. Participant's living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent. Exclusion Criteria: History of infection with HIV-1, HIV-2, or HTLV I/II. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV I/II, Treponema Pallidum or HIV I-II Participant has been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype. Participant is unable to undergo leukapheresis. Total number of CD34+ cells collected is lower than 20x106 cells Participant has known hypersensitivity to murine proteins or iron-dextran. Participant has chronic severe infection. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results. History of malignancy History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation. Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
Sites / Locations
- Sheba Medical CenterRecruiting
Arms of the Study
Arm 1
Experimental
Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria