Clinical trial NCT06017869: Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome
Official title: A Phase I, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Mitochondrial Diseases
Pearson Syndrome
Keywords: Autologous
Mitochondrial
Pearson
Transplantation
Stem cell
Lead sponsor: Minovia Therapeutics Ltd.
Sponsor class: INDUSTRY
First posted: 2023-08-30
Enrollment: 5
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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
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.

Primary outcomes:
measure: Occurrence of treatment-related adverse events
timeFrame: 12 months post treatment.
Secondary outcomes:
measure: 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.
timeFrame: 3, 6 or 12 months post treatment.
measure: Assessment of frequency of hospitalizations
timeFrame: 12 months post treatment
measure: Assessment of lengths of hospitalizations
timeFrame: 12 months post treatment
measure: Change in Anemia
timeFrame: 3, 6 or 12 months post treatment
measure: Change in thrombocytopenia
timeFrame: 3, 6 or 12 months post treatment
measure: Changes in leukopenia during a follow up period of 12 months post treatment
timeFrame: 3, 6 or 12 months post treatment
measure: Changes in frequency of blood transfusions during a follow up period of 12 months post treatment.
timeFrame: 3, 6 or 12 months post treatment
Other outcomes:
Locations:
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
Study officials:
References: