Trial record 18 of 185 for:    "Mitochondrial Diseases" OR "mitochondrial neurogastrointestinal encephalopathy disease"

Mitochondrial-enriched Autologous CD34+ Cells for Non-inherited Mitochondrial Disorders (MNV-BLD-1)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03384420
Recruitment Status : Not yet recruiting
First Posted : December 27, 2017
Last Update Posted : December 18, 2018
Information provided by (Responsible Party):
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 Intervention/treatment Phase
Mitochondrial Diseases Pearson Syndrome Biological: CD34+ cells enriched with MNV-BLD Early Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Autologous CD34+ Cells Enriched With MNV-BLD (Blood Derived Mitochondria) for Non-inherited Mitochondrial Disorders
Estimated Study Start Date : January 1, 2019
Estimated Primary Completion Date : January 1, 2020
Estimated Study Completion Date : January 1, 2021

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)

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

Other Outcome Measures:
  1. 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

  2. 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.

  3. 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

  4. 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

  5. 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.

  6. 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.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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 identifier (NCT number): NCT03384420

Contact: Vered Nusboim, MS 972-3-5307145
Contact: Elad Jacoby, MD 972-3-5303037

Sponsors and Collaborators
Minovia Therapeutics Ltd.

Responsible Party: Minovia Therapeutics Ltd. Identifier: NCT03384420     History of Changes
Other Study ID Numbers: 3428-16-SMC
First Posted: December 27, 2017    Key Record Dates
Last Update Posted: December 18, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Minovia Therapeutics Ltd.:
Autologous stem cell transplantation

Additional relevant MeSH terms:
Mitochondrial Diseases
Metabolic Diseases