Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome

Phase 1

Recruiting

• Conditions: Mitochondrial Diseases; Pearson Syndrome
• Interventions: Biological: MNV-201

• Registration Number: NCT06017869
• Lead Sponsor: Minovia Therapeutics Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-07-31
• Study First Submitted: 2023-08-24
• Study First Submitted QC: 2023-08-24
• Study First Posted: 2023-08-30
• Status Verified: 2023-10
• Last Update Submitted: 2024-06-09
• Last Update Posted: 2024-06-11
• Primary Completion: 2026-12
• Study Completion: 2027-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

1. Male or female participants aged from 1 to 18 years old.
2. Diagnosis of Pearson Syndrome
3. Body weight ≥ 10 kg.
4. Participant has anemia and/or thrombocytopenia, and/or leukopenia and/or blood transfusion dependent (receives blood transfusions every 6 weeks or less).
5. Participant is medically able to undergo the study interventions, as determined by the investigator.
6. Participant's living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.

Exclusion Criteria

1. History of infection with HIV-1, HIV-2, and HTLV I/II.
2. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV I/II, Treponema Pallidum and HIV I-II
3. Participant has been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
4. Participant is unable to undergo leukapheresis.
5. Total number of CD34+ cells collected is lower than 20x106 cells
6. Participant has known hypersensitivity to murine proteins or iron-dextran.
7. Participant has chronic severe infection.
8. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results.
9. History of malignancy
10. History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation.
11. Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment.
12. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria	MNV-201	-

Primary Outcome Measures

Name	Time	Method
Occurrence of treatment-related adverse events	12 months post treatment.	Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion.

Secondary Outcome Measures

Name	Time	Method
Change in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) scores, since Baseline and during a follow up period of 3-, 6- or 12-months post treatment.	3, 6 or 12 months post treatment.	The score is expressed as the percentage of items which were feasible to perform. The lower the score, the better is the child performance. The minimum score is 0% and the maximum score is 100%.
Changes in leukopenia during a follow up period of 12 months post treatment	3, 6 or 12 months post treatment	Changes since baseline in leukopenia during a follow up period of 3, 6 or 12 months post treatment measured by Complete Blood Count.
Changes in frequency of blood transfusions during a follow up period of 12 months post treatment.	3, 6 or 12 months post treatment	Change in frequency of blood transfusions during a follow up period of 3-, 6- or 12-months post treatment compared to the 6 months period before treatment.
Change in thrombocytopenia	3, 6 or 12 months post treatment	Changes since baseline in thrombocytopenia during a follow up period of 3, 6 or 12 months post treatment measured by Complete Blood Count.
Assessment of frequency of hospitalizations	12 months post treatment	Change in frequency of hospitalization during the 12 months after treatment compared to the 6 months period before treatment.
Assessment of lengths of hospitalizations	12 months post treatment	Change in lengths of hospitalization during the 12 months after treatment compared to the 6 months period before treatment.
Change in Anemia	3, 6 or 12 months post treatment	Change since Baseline in anemia during a follow up period of 3, 6 or 12 months post treatment measured by CBC.

Trial Locations

Locations (1)

Sheba Medical Center; 🇮🇱 Ramat Gan, Israel