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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03384420
Recruitment Status : Enrolling by invitation
First Posted : December 27, 2017
Last Update Posted : March 26, 2020
Sponsor:
Information provided by (Responsible Party):
Minovia Therapeutics Ltd.

Tracking Information
First Submitted Date  ICMJE November 30, 2017
First Posted Date  ICMJE December 27, 2017
Last Update Posted Date March 26, 2020
Actual Study Start Date  ICMJE February 13, 2019
Estimated Primary Completion Date January 1, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 24, 2020)
  • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 following MNV-BM-BLD infusion. [ Time Frame: 1 year ]
    Toxicities will according to CTCAE, Version 5.0 will be assessed starting enrollment.
  • Change in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) [ Time Frame: 1 year ]
    To explore 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
Original Primary Outcome Measures  ICMJE
 (submitted: December 24, 2017)
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 following MNV-BM-BLD infusion. [ Time Frame: 1 year ]
Toxicities will according to CTCAE, Version 4.0 will be assessed starting enrollment.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 3, 2020)
Percent of mitochondrial engraftment, per participant, compared to baseline, by serial qPCR in peripheral blood performed monthly. [ Time Frame: 1 years ]
We will monitor the engraftment of infused autologous cells enriched with normal mitochondria by measuring the level of the mitochondrial DNA by a qPCR assay designed to detect and separate between the participant's mitochondria and the normal donor mitochondria. Assays will be performed at baseline, monthly during the first year following the intervention, and then every 3 months.
Original Secondary Outcome Measures  ICMJE
 (submitted: December 24, 2017)
Percent of mitochondrial engraftment, per participant, compared to baseline, by serial qPCR in peripheral blood performed monthly. [ Time Frame: 2 years ]
We will monitor the engraftment of infused autologous cells enriched with normal mitochondria by measuring the level of the mitochondrial DNA by a qPCR assay designed to detect and separate between the participant's mitochondria and the normal donor mitochondria. Assays will be performed at baseline, monthly during the first year following the intervention, and then every 3 months.
Current Other Pre-specified Outcome Measures
 (submitted: February 3, 2020)
  • To measure change from baseline in cognitive status per patient by serial neuro-developmental clinical exams. [ Time Frame: 1 years ]
    Neuro-developmental assessment will be performed by a board-certified neurologist at baseline and every 6 months
  • Number of patients with changes in brain MRI from baseline [ Time Frame: 1 years ]
    Brain MRI will be done at baseline to detect changes associated with mitochondrial disorders. Follow-up will at 1 year.
  • To measure change from baseline in aerobic activity by 5-minute walk test or exercise test, according to baseline ability. [ Time Frame: 1 years ]
    Aerobic activity will be assessed at baseline and every 6 months
  • To measure the change from baseline in weight as compared to age-specific growth charts - from baseline and monthly throughout the study [ Time Frame: 1 years ]
    Growth is delayed in many mitochondrial disorders; We will follow weight gain of participants in this study and compare to control growth charts
  • Number of patients developing anti-mitochondrial antibodies compared to baseline during the study period. [ Time Frame: 1 year ]
    For potential immune reactions, anti-mitochondrial antibodies will be determined at baseline, and every 3 months till 1 year.
  • To measure change from baseline in peripheral blood lactate level. [ Time Frame: 1 years ]
    Determination of change in basic metabolic parameters in peripheral blood as a result of mitochondrial augmentation, focusing on lactate level. Measurements will be performed at baseline and every 3 months.
Original Other Pre-specified Outcome Measures
 (submitted: December 24, 2017)
  • To measure change from baseline in cognitive status per patient by serial neuro-developmental clinical exams. [ Time Frame: 2 years ]
    Neuro-developmental assessment will be performed by a board-certified neurologist at baseline and every 6 months
  • Number of patients with changes in brain MRI from baseline [ Time Frame: 1 years ]
    Brain MRI will be done at baseline to detect changes associated with mitochondrial disorders. Follow-up will at 1 year.
  • To measure change from baseline in aerobic activity by 5-minute walk test or exercise test, according to baseline ability. [ Time Frame: 2 years ]
    Aerobic activity will be assessed at baseline and every 6 months
  • To measure the change from baseline in weight as compared to age-specific growth charts - from baseline and monthly throughout the study [ Time Frame: 2 years ]
    Growth is delayed in many mitochondrial disorders; We will follow weight gain of participants in this study and compare to control growth charts
  • Number of patients developing anti-mitochondrial antibodies compared to baseline during the study period. [ Time Frame: 1 year ]
    For potential immune reactions, anti-mitochondrial antibodies will be determined at baseline, and every 3 months till 1 year.
  • To measure change from baseline in peripheral blood lactate level. [ Time Frame: 2 years ]
    Determination of change in basic metabolic parameters in peripheral blood as a result of mitochondrial augmentation, focusing on lactate level. Measurements will be performed at baseline and every 3 months.
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mitochondrial Diseases
  • Pearson Syndrome
Intervention  ICMJE Biological: CD34+ cells enriched with MNV-BLD
Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)
Study Arms  ICMJE Experimental: Intervention 'CD34+ cells enriched with MNV-BLD'
Intervention 'CD34+ cells enriched with MNV-BLD'
Intervention: Biological: CD34+ cells enriched with MNV-BLD
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Actual Enrollment  ICMJE
 (submitted: February 13, 2019)
7
Original Estimated Enrollment  ICMJE
 (submitted: December 24, 2017)
10
Estimated Study Completion Date  ICMJE January 1, 2021
Estimated Primary Completion Date January 1, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Molecular evidence of non-inherited mitochondrial disorder with a defect identified in the mitochondrial DNA
  • Normal maternal mitochondria

Exclusion Criteria:

  • Absence of detectable mitochondrial mutation or deletion
  • Maternal condition inadequate for 1 unit of blood donation
  • HIV, Hepatitis B or C carrier in child or their mother
  • Active severe infection
  • Inability to undergo leukapheresis
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Israel
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03384420
Other Study ID Numbers  ICMJE MNV-BM-BLD-001-IL
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Minovia Therapeutics Ltd.
Study Sponsor  ICMJE Minovia Therapeutics Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Minovia Therapeutics Ltd.
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP