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)
| NCT ID | NCT06017869 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Minovia Therapeutics Ltd. |
| Condition | Mitochondrial Diseases |
| Study Type | INTERVENTIONAL |
| Enrollment | 6 participants |
| Start Date | 2023-07-31 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 6 participants in total. It began in 2023-07-31 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06017869 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 18 Years, studying Mitochondrial Diseases. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06017869 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06017869 currently recruiting?
Yes, NCT06017869 is actively recruiting participants. Contact the research team at lea@minoviatx.com for enrollment information.
Where is the NCT06017869 trial being conducted?
This trial is being conducted at Ramat Gan, Israel.
Who is sponsoring the NCT06017869 clinical trial?
NCT06017869 is sponsored by Minovia Therapeutics Ltd.. The trial plans to enroll 6 participants.