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NCT03384420 | COMPLETED | Mitochondrial Diseases

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

Minovia Therapeutics Ltd.

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 diseases may be inherited from the mother (mitochondrial inheritance) or non-inherited. The latter are ultra-rare pediatric diseases caused by a mutation or deletion of mtDNA, which develop into a systemic multi organ disease and eventually death. MNV-BM-BLD is a therapeutic process for enrichment of patient's peripheral hematopoietic stem cells with normal and healthy mitochondria derived from donor blood cells. The process, called mitochondria augmentation therapy, aims to reduce the symptoms of mitochondrial diseases.

Condition or disease

Mitochondrial Diseases

Pearson Syndrome

Intervention/treatment

CD34+ cells enriched with MNV-BLD

Phase

PHASE1

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 7 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : A Phase I/II, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD (Autologous cd34+ Cells Enriched With Blood Derived Mitochondria) in Pediatric Patients With Pearson Syndrome
Actual Study Start Date : 2019-02-13
Estimated Primary Completion Date : 2021-03-09
Estimated Study Completion Date : 2021-03-09

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 3 Years to 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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.
    • A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

    Location Details

    NCT03384420

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    How to Participate

    Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

    Locations

    Not yet recruiting

    Israel,

    Sheba Medical Center Hospital- Tel Hashomer

    Ramat Gan, Israel,