Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)

Last updated: June 17, 2025
Sponsor: Minovia Therapeutics Ltd.
Overall Status: Active - Recruiting

Phase

2

Condition

Mitochondrial Diseases

Treatment

MNV-201

Clinical Study ID

NCT06017869
MNV-010
  • Ages 1-18
  • All Genders

Study Summary

Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy [LHON]), many involve multiple organs. Mitochondrial disorders may present at any age and a frequent feature is the increasing number of organs involved in the course of the disease.

Minovia Therapeutics Ltd. ("Minovia") is a biotech company developing novel therapeutics based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in Minovia's GMP facility.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Male or female participants aged from 1 to 18 years old.

  2. Diagnosis of Pearson Syndrome (current or history) as verified by molecularidentification of deletion in mtDNA of peripheral blood. Participants are diagnosedwith PS Participant can be in either the PS manifestations of the disease or mayhave transitioned to Kearns Sayre Syndrome (KSS) manifestations but has a history ofPS.

  3. Participants have failure to thrive (height SDS smaller than -1)

  4. Participants should have at least 12 months' history of body weight and height andcalculated GFR (from creatinine) before treatment.

  5. Body weight ≥ 10 kg.

  6. Participants' living parent(s) and/or legal guardian(s) able to understand andprovide voluntary written informed consent.

  7. Participants' parents or legal guardian have a good understanding of the study andnature of the procedure and are expected to be able to comply with study visitschedules and caregiver assessments without difficulty.

  8. Participants' parents or legal guardian provides written informed consent prior tostudy participation.

  9. Participants are medically able to undergo the study interventions as determined bythe Investigator.

Exclusion

Exclusion criteria:

  1. History of infection with HIV-1, HIV-2, or HTLV I/II.

  2. Participants have any active infection.

  3. Participants have been diagnosed with Myelodysplastic Syndrome, by FISH and/orkaryotype.

  4. Participants are unable to undergo apheresis.

  5. Participants have known hypersensitivity to murine proteins or iron-dextran.

  6. Participants have severe chronic infection.

  7. Participants have disease or conditions that may risk the participant or interferewith the ability to interpret the study results.

  8. History of malignancy.

  9. History of treatment with gene therapy, allogeneic bone marrow or cord bloodtransplantation.

  10. Participants have had a change in growth hormone regimen in less than 2 years priorto treatment.

  11. Participants have participated in another clinical trial or received otherexperimental medications outside a clinical trial within 1 month prior to start ofthis study.

  12. Participants who are pregnant or intend to become pregnant in the next 12 months.

  13. In the opinion of the Investigator, the participant is unsuitable for participatingin the study for any reason.

Study Design

Total Participants: 6
Treatment Group(s): 1
Primary Treatment: MNV-201
Phase: 2
Study Start date:
July 31, 2023
Estimated Completion Date:
December 31, 2027

Connect with a study center

  • Sheba Medical Center

    Ramat Gan, 5266202
    Israel

    Active - Recruiting

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