Evaluating MNV-201 for patients with low risk myelodysplastic syndromes

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

This study is testing a new treatment called MNV-201 to see if it can help people with low risk myelodysplastic syndromes make more blood cells and rely less on blood transfusions.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years to 100 Years
• Sex: All
• Sponsor: Minovia Therapeutics Ltd. (industry)
• Locations: 1 site (Jerusalem, Israel)
• Trial ID: NCT06465160 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of MNV-201, a treatment involving autologous CD34+ cells enriched with allogenic placenta-derived mitochondria, in patients diagnosed with low risk myelodysplastic syndromes (MDS). The study aims to address the ineffective hematopoiesis and associated anemia in these patients, who are often dependent on blood transfusions. Participants must have a documented history of anemia and have exhausted existing treatment options for low risk MDS. The trial will assess the potential of this innovative cell therapy to improve blood cell production and reduce transfusion dependency.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a diagnosis of low risk MDS who are transfusion dependent and have not responded to existing treatments.

Not a fit: Patients who are not eligible for allogeneic bone marrow transplantation or those who have not exhausted all existing treatment options may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life for patients with low risk MDS by reducing their dependence on blood transfusions.

How similar studies have performed: While the approach of using mitochondrial therapy in MDS is relatively novel, similar cell therapy strategies have shown promise in other hematological conditions.

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 with mutational burden and/or low burden of high-risk mutations as defined by IPSS-M.
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 available to him 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 , HCV, HTLV I/II, Treponema Pallidum or HIV I-II.
3. Participant is unable to undergo apheresis.
4. Participant has known hypersensitivity to murine proteins or iron-dextran.
5. Participant has chronic severe infection.
6. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results.
7. History of treatment for malignant disease (other than excision of non-melanoma skin cancer) in the last 2 years
8. Pregnancy or breastfeeding
9. History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation.
10. Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment.
11. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.

Where this trial is running

Jerusalem, Israel

• Shaare Zedek Medical Center — Jerusalem, Israel, Israel (RECRUITING)

Study contacts

• Study coordinator: Lea Bensoussan, MSc

Conditions: Myelodysplastic Syndromes, Low Risk MDS, Anemia, Mitochondrial, Cell Therapy