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)

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

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) is a Phase 2 interventional studying Mitochondrial Diseases and Pearson Syndrome, sponsored by Minovia Therapeutics Ltd.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

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.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Mitochondrial Diseases and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 6 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Male or female participants aged from 1 to 18 years old. 2. 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. 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 and calculated GFR (from creatinine) before treatment. 5. Body weight ≥ 10 kg. 6. Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written willing to sign a consent form. 7. 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. 8. Participants' parents or legal guardian provides written willing to sign a consent form prior to study participation. 9. Participants are medically able to undergo the study interventions as determined by the Investigator. Who Should NOT Join This Trial: 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/or karyotype. 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 interfere with the ability to interpret the study results. 8. History of malignancy. 9. History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation. 10. Participants have had a change in growth hormone regimen in less than 2 years prior to treatment. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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 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. 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 and calculated GFR (from creatinine) before treatment. 5. Body weight ≥ 10 kg. 6. Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent. 7. 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. 8. Participants' parents or legal guardian provides written informed consent prior to study participation. 9. Participants are medically able to undergo the study interventions as determined by the Investigator. 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/or karyotype. 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 interfere with the ability to interpret the study results. 8. History of malignancy. 9. History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation. 10. Participants have had a change in growth hormone regimen in less than 2 years prior to treatment. 11. 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. 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 participating in the study for any reason.

Treatments Being Tested

BIOLOGICAL

MNV-201

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.

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Sheba Medical Center

Ramat Gan, Israel, Israel

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06017869), the sponsor (Minovia Therapeutics Ltd.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06017869 clinical trial studying?

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 d… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06017869?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06017869?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06017869. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06017869. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-06-07 · Data from ClinicalTrials.gov.