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

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ClinicalTrials.gov Identifier: NCT03384420
Recruitment Status : Completed
First Posted : December 27, 2017
Last Update Posted : August 31, 2021
Sponsor:
Information provided by (Responsible Party):
Minovia Therapeutics Ltd.

Study Description
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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 Intervention/treatment Phase
Mitochondrial Diseases Pearson Syndrome Biological: CD34+ cells enriched with MNV-BLD Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
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 : February 13, 2019
Actual Primary Completion Date : March 9, 2021
Actual Study Completion Date : March 9, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Intervention CD34+ cells enriched with MNV-BLD
Intervention CD34+ cells enriched with MNV-BLD
 Biological: CD34+ cells enriched with MNV-BLD
Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with Treatment-related adverse events as assessed by CTCAE v5.0 following MNV-BM-BLD during a follow up period of 12 months post treatment. [ Time Frame: 1 year ]
    Severity will graded according to CTCAE, Version 5.0

  2. IPMDS (International Pediatric Mitochondrial Disease Scale) [ Time Frame: 1 year ]
    To compare the change in International Pediatric Mitochondrial Disease Scale (IPMDS) score during a follow up period of 12 months post treatment. IPMDS total score ranges from 0 to 243. The score is expressed as the percentage of items which were feasible to perform. The lower the score is, the higher the child's function


Secondary Outcome Measures :
  1. Weight [ Time Frame: 1 year ]
    To compare the changes (kilograms) to Baseline

  2. Quantification of levels of normal mtDNA in blood and urine [ Time Frame: 1 years ]
    To compare the changes to Baseline

  3. Metabolic crisis events occurrence compared to two years prior to the study. [ Time Frame: 3 Years ]
    To compare the changes during 3 years (2 years prior the study entry and 1 year follow up)

  4. Change in renal function [ Time Frame: 1 year ]
    Measurement of blood creatinine in a serum sample

  5. Change in Brain involvement [ Time Frame: 1 year ]
    Lactate peak as assessed by MRS

  6. Height [ Time Frame: 1 year ]
    To compare the changes (in meters) to Baseline

  7. Change in cardiac function [ Time Frame: 1 year ]
    Assessment of left ventricular ejection fraction via echocardiography

  8. Monitoring for liver disease [ Time Frame: 1 year ]
    Measurement of Aspartate Aminotransferase and Alanine aminotransferase level


Other Outcome Measures:
  1. Hospitalization events [ Time Frame: 1 year ]
    To compare the changes from medical history to 1 year follow up

  2. Change in functional status [ Time Frame: 1 year ]
    Distance traveled during the 6MWT (meters)

  3. Change in hematological parameter [ Time Frame: 1 year ]
    Measurement of hemoglobin level

  4. Change in hematological parameter [ Time Frame: 1 year ]
    Measurement of absolute neutrophil count

  5. Change in hematological parameter [ Time Frame: 1 year ]
    Measurement of platelet count

  6. Control of blood glucose concentration [ Time Frame: 1 year ]
    Hemoglobin A1c% in whole blood

  7. ATP content. [ Time Frame: 1 year ]
    To compare the changes to Baseline


Eligibility Criteria
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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03384420


Locations
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Israel
Sheba Medical Center Hospital- Tel Hashomer
Ramat Gan, Israel
Sponsors and Collaborators
Minovia Therapeutics Ltd.
More Information
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Responsible Party: Minovia Therapeutics Ltd.
ClinicalTrials.gov Identifier: NCT03384420    
Other Study ID Numbers: MNV-BM-BLD-001-IL
First Posted: December 27, 2017    Key Record Dates
Last Update Posted: August 31, 2021
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Minovia Therapeutics Ltd.:
Autologous stem cell transplantation
Mitochondrial Diseases
Pearson Syndrome
Rare Diseases
Additional relevant MeSH terms:
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Muscular Diseases
Lipid Metabolism, Inborn Errors
Mitochondrial Diseases
Syndrome
Disease
Pathologic Processes
Metabolic Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases