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)

Launched by MINOVIA THERAPEUTICS LTD. · Aug 24, 2023

Keywords

ClinConnect Summary

The clinical trial titled "Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome" is investigating a new treatment for children with Pearson Syndrome, a rare condition that affects how cells produce energy and can lead to problems in multiple organs. The treatment being studied, called MNV-201, uses a special type of cell therapy that combines the participant's own stem cells with healthy mitochondria from placental tissue. The goal is to see if this therapy can help improve the health and well-being of children suffering from this condition.

To be eligible for this trial, children aged 1 to 18 who have been diagnosed with Pearson Syndrome and weigh at least 10 kg can apply. They should also have specific blood-related issues that require regular transfusions. Participants will undergo a process called leukapheresis to collect their stem cells, which will then be treated with the healthy mitochondria. Throughout the trial, participants will be closely monitored for their safety and any changes in their health. It's important to note that there are certain health conditions that would prevent participation, such as severe infections or a history of certain cancers. This trial is currently recruiting participants and aims to provide new hope for children affected by this challenging disease.

Gender

ALL

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.