Transplantation of MNV-201 for treating Pearson Syndrome in children

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

This study is testing a new cell therapy called MNV-201 to see if it can help children with Pearson Syndrome feel better and manage their symptoms.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 1 Year to 18 Years
• Sex: All
• Sponsor: Minovia Therapeutics Ltd. (Industry-sponsored)
• Locations: 1 site (Ramat Gan, Israel)
• Trial ID: NCT06017869 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 1 to 18 years with a confirmed diagnosis of Pearson Syndrome who are experiencing anemia, thrombocytopenia, or leukopenia.

Not a fit: Patients with active infections, certain blood disorders, or those unable to undergo leukapheresis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the health and quality of life for children suffering from Pearson Syndrome.

How similar studies have performed: While the approach of using mitochondrial augmentation technology is innovative, similar studies in mitochondrial diseases have shown promising results, indicating potential for success.

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.

Where this trial is running

Ramat Gan, Israel

• Sheba Medical Center — Ramat Gan, Israel, Israel (Recruiting)

Study contacts

• Study coordinator: Lea Bensoussan, Msc

Conditions Mitochondrial Diseases; Pearson Syndrome; Autologous; Mitochondrial; Pearson; Transplantation; Stem cell