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NCT Number: NCT06017869

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)

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.

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Key information

Conditions

Mitochondrial Diseases Metabolic Diseases Nutritional and Metabolic Diseases Pearson Syndrome VLCAD deficiency

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or female participants aged from 1 to 18 years old.
  • Diagnosis of Pearson Syndrome (current or history) as verified by molecular identification of deletion in mtDNA of peripheral blood. Participants are diagnosed with PS Participant can be in either the PS manifestations of the disease or may have transitioned to Kearns Sayre Syndrome (KSS) manifestations but has a history of PS.
  • Participants have failure to thrive (height SDS smaller than -1)
  • Participants should have at least 12 months' history of body weight and height and calculated GFR (from creatinine) before treatment.
  • Body weight ≥ 10 kg.
  • Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.
  • Participants' parents or legal guardian have a good understanding of the study and nature of the procedure and are expected to be able to comply with study visit schedules and caregiver assessments without difficulty.
  • Participants' parents or legal guardian provides written informed consent prior to study participation.
  • Participants are medically able to undergo the study interventions as determined by the Investigator.

Exclusion criteria

  • History of infection with HIV-1, HIV-2, or HTLV I/II.
  • Participants have any active infection.
  • Participants have been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
  • Participants are unable to undergo apheresis.
  • Participants have known hypersensitivity to murine proteins or iron-dextran.
  • Participants have severe chronic infection.
  • Participants have disease or conditions that may risk the participant or interfere with the ability to interpret the study results.
  • History of malignancy.
  • History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation.
  • Participants have had a change in growth hormone regimen in less than 2 years prior to treatment.
  • Participants have participated in another clinical trial or received other experimental medications outside a clinical trial within 1 month prior to start of this study.
  • Participants who are pregnant or intend to become pregnant in the next 12 months.
  • In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.

Treatment and study plan

MNV-201

Biological

Autologous CD34+ cells are isolated from the participant's peripheral blood after mobilization by leukapheresis. Allogeneic mitochondria are isolated under aseptic conditions from healthy donor placenta, cryopreserved and qualified before use.

Other names: CD34+ cells enriched with allogeneic placenta derived mitochondria

Primary outcomes

  1. Occurrence of treatment-related adverse events

    Time frame: 12 months post treatment.

    Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion

  2. Height SDS

    Time frame: 24 months

    Improvement from baseline to 12 months post treatment in height SDS compared to the calculated change in height SDS in the 12 months prior to treatment.

Secondary outcomes

  1. Height SDS

    Time frame: 12 months

    Improvement from baseline to 6 months post treatment in height SDS compared to the calculated change in height SDS in the 6 months prior to treatment.

  2. Calculated GFR Slope

    Time frame: 24 months

    Improvement in calculated GFR slope 6- and/or 12-months post treatment relative to 6 and/or 12 months prior to treatment (respectively)

Other outcomes

  1. Exploratory endpoint: Organ dysfunction

    Time frame: 24 months

    Improvement in any of the measurements below of organ dysfunction at 6- and/or 12-month post-treatment compared to Baseline

    • Renal glomerulopathy measured by level of cystatin-C or creatinine
    • Renal tubulopathy measured by urine amino acids
    • Renal tubulopathy measured by blood bicarbonate
    • Gastrointestinal activity measured by albumin
    • Gastrointestinal activity measured by pediatric PedsQL 3.0 Gastrointestinal Symptoms Module
    • Pancreas activity measured by insulin
    • Pancreas activity measured by C-peptide
    • Heart activity measured by ECG
    • Growth measured by GDF15
    • Growth measured by IGF1
    • Growth measured by IGFBP
    • Change in physician organ involvement score (see appendix C)
    • Renal glomerulopathy measured by slope of change in eGFR calculated from cystatin-C at 6- or 12-month post-treatment compared to that calculated in the 6- or 12- month period prior to baseline.

  2. Exploratory endpoint: IPMDS

    Time frame: 12 months

    Improvement from baseline in normalized International Pediatric Mitochondrial Disease Scale (IPMDS) scores (total score or each of three separate components) at 6- or 12-month post-treatment compared to Baseline.

  3. Exploratory endpoint: Frequency of Hospitalizations

    Time frame: 24 months

    Reduction in frequency of hospitalization(s) during the 6- and/or 12-months post treatment relative to the 6 and/or 12 months (respectively) prior to treatment

  4. Exploratory endpoint: Length of Hospitalizations

    Time frame: 24 months

    Reduction of length of hospitalization(s) during the 6- and/or 12-months post treatment relative to the 6 and/or 12 months (respectively) prior to treatment.

  5. Exploratory endpoint: exogenous mtDNA

    Time frame: 12 months

    Pharmacokinetic measurements: exogenous mtDNA analysis in PBMCs at 6- and/or 12-month time points

  6. Exploratory endpoint: Blood based biomarkers

    Time frame: 12 months

    Change in blood-based biomarkers at 6- and/or 12-month post-treatment compared to Baseline

    • Mitochondrial quantity and activity (full length mtDNA copy number in PBMC and/or whole blood); ATP content of PBMCs;
    • Anti-mitochondrial antibody level
    • PBMC subset analysis.

  7. Exploratory endpoint: Height SDS

    Time frame: 12 months

    Improvement in height SDS at 6 and/or 12 months relative to natural history available (prospective and/or retrospective)

  8. Exploratory endpoint: weight SDS

    Time frame: 24 months

    Improvement in weight SDS in 6-month and/or 12-month period after treatment relative to 6- and/or 12-month period (respectively) prior to treatment.

Study contacts

Contact information is provided by the study sponsor or research team.

Lea Bensoussan, Msc

CONTACT

[email protected]

+ 972 586101291 ext. +972

Natalie Yivgi Ohana, PhD

CONTACT

[email protected]

+972 54 5833727 ext. +972

Sponsors and collaborators

Lead sponsor

Minovia Therapeutics Ltd.

Industry

Registry information

Official study title

PHASE II, OPEN LABEL, SINGLE DOSE STUDY OF THE SAFETY AND EFFICACY OF MNV-201 FOR THE TREATMENT OF PEARSON SYNDROME

Important dates

Study start
2023
Primary completion
2026
Study completion
2027
First posted
Aug 30, 2023
Registry last updated
Jun 22, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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