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CompletedNCT03384420Updated Aug 31, 2021

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

A Phase 1/2 interventional study of CD34+ cells enriched with MNV-BLD in Mitochondrial Diseases and Pearson Syndrome, sponsored by Minovia Therapeutics Ltd.. Completed at 1 site in Israel. Open to participants aged 3 Years to 18 Years. Per ClinicalTrials.gov, last updated 2021-08-31.

Sponsored by Minovia Therapeutics Ltd. · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Mar 2021, 5 years 8 months ago, and no results have been posted to the registry.
Phase
Phase 1/2
Study type
Interventional
Enrollment
7
Allocation
Not applicable
Ages
3 Years to 18 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Mitochondrial Diseases
  • Pearson Syndrome

Keywords

  • Autologous stem cell transplantation
  • Mitochondrial Diseases
  • Pearson Syndrome
  • Rare Diseases
03

In context

Mitochondrial Diseases

189 studies on the registry are indexed under Mitochondrial Diseases; 60 are open to participants now.

This study's enrollment of 7 is below the median of 35 across 114 interventional studies indexed under Mitochondrial Diseases.

Browse Mitochondrial Diseases studies →

Lead sponsor

Minovia Therapeutics Ltd. is the lead sponsor of 7 studies on the registry; 4 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
7 participants (actual)

Study arms

  • Experimental
    Intervention CD34+ cells enriched with MNV-BLD

    Intervention CD34+ cells enriched with MNV-BLD

    Biological: CD34+ cells enriched with MNV-BLD

Interventions

  • BiologicalCD34+ cells enriched with MNV-BLD

    Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)

06

What researchers measure

Primary outcomes

  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.

    Severity will graded according to CTCAE, Version 5.0

    Time frame: 1 year

  2. IPMDS (International Pediatric Mitochondrial Disease Scale)

    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

    Time frame: 1 year

Secondary outcomes

  1. Weight

    To compare the changes (kilograms) to Baseline

    Time frame: 1 year

  2. Quantification of levels of normal mtDNA in blood and urine

    To compare the changes to Baseline

    Time frame: 1 years

  3. Metabolic crisis events occurrence compared to two years prior to the study.

    To compare the changes during 3 years (2 years prior the study entry and 1 year follow up)

    Time frame: 3 Years

  4. Change in renal function

    Measurement of blood creatinine in a serum sample

    Time frame: 1 year

  5. Change in Brain involvement

    Lactate peak as assessed by MRS

    Time frame: 1 year

  6. Height

    To compare the changes (in meters) to Baseline

    Time frame: 1 year

  7. Change in cardiac function

    Assessment of left ventricular ejection fraction via echocardiography

    Time frame: 1 year

  8. Monitoring for liver disease

    Measurement of Aspartate Aminotransferase and Alanine aminotransferase level

    Time frame: 1 year

Other outcomes

  1. Hospitalization events

    To compare the changes from medical history to 1 year follow up

    Time frame: 1 year

  2. Change in functional status

    Distance traveled during the 6MWT (meters)

    Time frame: 1 year

  3. Change in hematological parameter

    Measurement of hemoglobin level

    Time frame: 1 year

  4. Change in hematological parameter

    Measurement of absolute neutrophil count

    Time frame: 1 year

  5. Change in hematological parameter

    Measurement of platelet count

    Time frame: 1 year

  6. Control of blood glucose concentration

    Hemoglobin A1c% in whole blood

    Time frame: 1 year

  7. ATP content.

    To compare the changes to Baseline

    Time frame: 1 year

07

Study locations

1 site
  • Sheba Medical Center Hospital- Tel Hashomer
    Ramat Gan, Israel
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Feb 2019 to primary completion in Mar 2021, 24 months.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 30, 2017
First posted
Dec 27, 2017
StartActual
Feb 13, 2019
Primary completionActual
Mar 9, 2021
CompletionActual
Mar 9, 2021
Last update
Aug 31, 2021
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 31, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT03384420
Lead sponsor
Minovia Therapeutics Ltd.
Responsible party
Sponsor
First posted
Dec 27, 2017
Start date
Feb 13, 2019
Primary completion
Mar 9, 2021
Completion
Mar 9, 2021
Last update
Aug 31, 2021

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2020. You cannot join it, but the record below documents what was studied.

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Discussion

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