A Study to Evaluate the MNV-201 in Patients With Low Risk MDS

Trial Title: A Study to Evaluate the MNV-201 in Patients With Low Risk MDS

NCT ID: NCT06465160

Condition: Myelodysplastic Syndromes

Conditions: Official terms:
Preleukemia
Myelodysplastic Syndromes
Syndrome

Conditions: Keywords:
Low Risk MDS
Anemia
Mitochondrial
Cell Therapy

Study type: Interventional

Study phase: Phase 1

Overall status: Recruiting

Study design:

Allocation: N/A

Intervention model: Single Group Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Intervention:

Intervention type: Biological
Intervention name: MNV-201 (Autologous CD34+ Cells Enriched with allogenic Placenta Derived Mitochondria)
Description: The participant will undergo 5 days of mobilization by G-CSF administration (Neupogen) once a day during 5 days. On the 5th day, and after receiving the last dose of Neupogen, the participant will undergo Apheresis to collect CD34+ cells. MNV-201 consists of autologous CD34+ cells enriched with allogeneic placenta derived mitochondria. Autologous CD34+ cells are isolated from the participant's peripheral blood after mobilization by apheresis. Allogeneic mitochondria are isolated under aseptic conditions from healthy donor placenta, cryopreserved and qualified before use. Each product package will consist of a ready-for-injection sterile infusion bag containing clinical grade MNV-201 product for IV infusion for a single specified (autologous) participant.
Arm group label: Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria

Summary: Myelodysplastic syndromes (MDS) are a group of bone marrow failures that occur when the blood-forming cells in the bone marrow become abnormal leading to an abnormal differentiation and production of one or more blood cell types. According to the American Cancer Society, in the United States, MDS occurs at a rate of 4.8 cases for every 100,000 people; MDS affects an estimated 60,000 persons in the United States, with 10,000-15,000 new cases recorded each year. MDS is defined by ineffective haematopoiesis resulting in blood cytopenias (a reduction in the number of mature blood cells), and clonal instability with a risk of evolution to acute myeloid leukaemia (AML). Patients with MDS collectively have a high symptom burden and are also at risk of death from complications of cytopenias and AML. MDS is generally a disease that develops with ageing; the median age at diagnosis of MDS is ~70 years, and patients frequently have comorbid conditions. The goals of therapy for patients with MDS are to reduce disease-associated symptoms and the risk of disease progression and death, thereby improving both quality and quantity of life. 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.

Criteria for eligibility:
Criteria:
Inclusion Criteria: 1. Male or female participants aged from 18 years old and above. 2. Low Risk MDS diagnosis with R-IPSS score of ≤3. 3. Participant has anemia and is blood transfusion dependent (received 2 or more units of packed blood per /4 weeks for at least 8 weeks before enrollment). 4. A baseline natural history of the participant is available, including anemia and transfusions frequency at least 6 months before enrollment. 5. Participant has utilized all existing treatments for low risk MDS that are approved and reimbursed in Israel, or is not medically eligible for those treatment options. 6. Participant is not eligible for Allogeneic Bone Marrow Transplantation. 7. Participant is medically able to undergo the study interventions, as determined by the investigator. 8. Participant 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, or HTLV I/II. 2. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV I/II, Treponema Pallidum or HIV I-II 3. Participant is unable to undergo apheresis. 4. Total number of CD34+ cells collected is lower than 20x106 cells. 5. Participant has known hypersensitivity to murine proteins or iron-dextran. 6. Participant has chronic severe infection. 7. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results. 8. History of treatment for malignant disease (other than excision of non-melanoma skin cancer) in the last 2 years 9. Pregnancy or breastfeeding 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.

Gender: All

Minimum age: 18 Years

Maximum age: 100 Years

Healthy volunteers: No

Locations:

Facility:
Name: Shaare Zedek Medical Center

Address:
City: Jerusalem
Zip: 9103102
Country: Israel

Status: Recruiting

Contact:
Last name: Yishai Ofran, MD

Phone: (0)2-5645462

Phone ext: +972
Email: yofran@szmc.org.il

Contact backup:
Last name: Dana Darel

Phone: 0503855514

Phone ext: +972
Email: danada@szmc.org.il

Start date: May 27, 2024

Completion date: December 31, 2029

Lead sponsor:
Agency: Minovia Therapeutics Ltd.
Agency class: Industry

Source: Minovia Therapeutics Ltd.

Record processing date: ClinicalTrials.gov processed this data on November 12, 2024

Source: ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT06465160