A Study to Evaluate the MNV-201 in Patients With Low Risk MDS
A Phase Ib, Open Label, Single or Repeated Dose Exploration Clinical Study to Evaluate the Safety and Therapeutic Effects of Infusion of MNV-201 (Autologous CD34+ Cells Enriched With Allogenic Placenta Derived Mitochondria) in Patients With Low-Risk Myelodysplastic Syndrome
Minovia Therapeutics Ltd.
15 participants
May 27, 2024
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria8
- Male or female participants aged from 18 years old and above.
- Low Risk MDS diagnosis with R-IPSS score of ≤3 with mutational burden and/or low burden of high-risk mutations as defined by IPSS-M.
- 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).
- A baseline natural history of the participant is available, including anemia and transfusions frequency at least 6 months before enrollment.
- Participant has utilized all existing treatments for low risk MDS that are approved and available to him or is not medically eligible for those treatment options.
- Participant is not eligible for Allogeneic Bone Marrow Transplantation.
- Participant is medically able to undergo the study interventions, as determined by the investigator.
- Participant and/or legal guardian(s) able to understand and provide voluntary written informed consent.
Exclusion Criteria11
- History of infection with HIV-1, HIV-2, or HTLV I/II.
- Current active infection with HBV , HCV, HTLV I/II, Treponema Pallidum or HIV I-II.
- Participant is unable to undergo apheresis.
- Participant has known hypersensitivity to murine proteins or iron-dextran.
- Participant has chronic severe infection.
- Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results.
- History of treatment for malignant disease (other than excision of non-melanoma skin cancer) in the last 2 years
- Pregnancy or breastfeeding
- History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation.
- Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment.
- In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
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Interventions
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.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT06465160