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)

NCT06017869 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MNV-010
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Mitochondrial Diseases; Pearson Syndrome

Keywords

Autologous; Mitochondrial; Pearson; Transplantation; Stem cell

Outcome Measures

Primary Outcomes (2)

• Occurrence of treatment-related adverse events

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

  12 months post treatment.

• Height SDS

  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.

  24 months

Secondary Outcomes (2)

• Height SDS

  12 months

• Calculated GFR Slope

  24 months

Other Outcomes (8)

• Exploratory endpoint: Organ dysfunction

  24 months

• Exploratory endpoint: IPMDS

  12 months

• Exploratory endpoint: Frequency of Hospitalizations

  24 months

Study Arms (1)

Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria

EXPERIMENTAL

Biological: MNV-201

Interventions

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.

Also known as: CD34+ cells enriched with allogeneic placenta derived mitochondria

Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Minovia Therapeutics Ltd.: lead

Study Sites (1)

Sheba Medical Center

Ramat Gan, Israel, 5266202, Israel

RECRUITING

MeSH Terms

Conditions

Mitochondrial Diseases; VLCAD deficiency

Condition Hierarchy (Ancestors)

Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Lea Bensoussan, Msc

CONTACT

+ 972 586101291; lea@minoviatx.com

Natalie Yivgi Ohana, PhD

CONTACT

+972 54 5833727; natalie@minoviatx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2023
• First Posted: August 30, 2023
• Study Start: July 31, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: June 22, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)