Transplantation of MNV-201 for treating Pearson Syndrome in children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and therapeutic effects of MNV-201, a cell therapy developed by Minovia Therapeutics, in pediatric patients diagnosed with Pearson Syndrome. The therapy involves the transplantation of the participant's own enriched hematopoietic stem and progenitor cells, combined with allogeneic placental-derived mitochondria. The study aims to assess the potential benefits of this innovative approach in addressing the complications associated with mitochondrial diseases. Participants will be monitored for safety and efficacy throughout the trial.

Why it matters

Who can join: the full rules

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.

Where it's running

• Sheba Medical Center
  Ramat Gan, Israel, Israel Recruiting

Study contacts

• Study coordinator: Lea Bensoussan, Msc
• Email: lea@minoviatx.com
• Phone: + 972 586101291

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.