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

Myelodysplastic Syndromes Clinical Trial

Official title:

A Phase Ib, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Infusion of MNV-201 in Patients With Low Risk Myelodysplastic Syndrome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06465160
• Other study ID #: MNV-012
• Status: Recruiting
• Phase: Phase 1
• Start date: May 27, 2024
• Est. completion date: December 31, 2029

Study information

• Verified date: June 2024
• Source: Minovia Therapeutics Ltd.
• Contact: Lea Bensoussan, MSc
• Phone: 0586101291
• Email: lea[@]minoviatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: December 31, 2029
• Est. primary completion date: December 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 100 Years

Eligibility

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.

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Biological:
• MNV-201 (Autologous CD34+ Cells Enriched with allogenic Placenta Derived Mitochondria)
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

Country	Name	City	State
Israel	Shaare Zedek Medical Center	Jerusalem

Sponsors (1)

Lead Sponsor	Collaborator
Minovia Therapeutics Ltd.

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence 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 post treatment.	1 year
Secondary	Anemia assessment	change in the anemia will be counted by the change in the average Hgb level in G/dL as measured monthly in balks of three months -3-1(prior to infusion), 1-3, 4-6, 7-9, 10-12 months.	1 year
Secondary	Blood transfusion assessment	Changes in frequency of blood transfusions during a follow up period of 12 months post treatment compared to the 6 months period before treatment.	1 year
Secondary	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 12 months post treatment. The higher the score, the better the Quality of Life. The minimum Score is 0 the maximum score is 4.	1 year