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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
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.
Shaare Zedek Medical Center
Jerusalem, Israel, Israel
RECRUITINGOccurrence of treatment-related adverse events
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion, during a follow up period of 12 months following first dosing (Part 1) and 6 months following second dosing (Part 2), where relevant.
Time frame: 1 year
Anemia assessment
Hemoglobin rise measured by CBC of at least 1 g/dL from baseline during a follow up period of 6- and 12-months post treatment (Part 1) or 6 months post
Time frame: 1 year
Blood transfusion assessment
Reduction in frequency of blood transfusions during a follow up period of 6- and 12-months post treatment (Part 1) or 6 months post treatment (Part 2) compared to the 6 months period before treatment.
Time frame: 1 year
Assessment of Quality of Life by the Functional Assessment of Cancer Therapy - Anemia
Change in health-related quality-of-life (HRQoL) assessed by the Functional Assessment of Cancer Therapy - Anemia (FACT-An) from baseline during a follow up period of 6- and 12-months post treatment (Part 1) or 6 months post treatment (Part 2).
Time frame: 1 year
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