Status:
COMPLETED
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Lead Sponsor:
Minovia Therapeutics Ltd.
Conditions:
Mitochondrial Diseases
Pearson Syndrome
Eligibility:
All Genders
3-18 years
Phase:
PHASE1
PHASE2
Brief Summary
Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes. These diseas...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Patient diagnosed with Pearson Syndrome, as verified by molecular identification of a defect in the mitochondrial DNA.
- Normal maternal mitochondria as verified by mtDNA sequencing.
- Males and females between 3 years or older and up to 18th birthday.
- Patient is transfusion independent.
- Patient has at least one of the following systematic involvements:
- High baseline lactate levels
- Episodes of metabolic crisis in the last year before pre-screening
- Renal failure (not dependent on dialysis) or evidence of proximal tubulopathy
- Growth retardation or failure to thrive
- Exclusion Criteria
- Absence of detectable mitochondria mutation or deletion.
- Patient or patient's mother have a positive test for microbiologic
- Patient is unable to undergo leukapheresis.
- 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.
- Patient has been treated previously with any cell or gene therapy.
- 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.
Exclusion
Key Trial Info
Start Date :
February 13 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 9 2021
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT03384420
Start Date
February 13 2019
End Date
March 9 2021
Last Update
August 31 2021
Active Locations (1)
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1
Sheba Medical Center Hospital- Tel Hashomer
Ramat Gan, Israel