Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorKirsten Grønbæk

About this trial

This is a single-arm pilot study of the feasibility and safety of metformin in patients with clonal cytopenia of undetermined significance (CCUS) or lower-risk myelodysplastic neoplasms (LR-MDS).

Eligibility criteria

Qualifiers

LR-MDS according to the revised international prognostic scoring system (IPSS-R), i.e., very low- or low-risk disease (IPSS-R score ≤3) in addition to a bone marrow blast percentage <5 OR

CCUS defined as the presence of somatic mutation(s) or cytogenetic abnormality not diagnostic of MDS or any other malignancy in the context of persistent cytopenia (>6 months) with other common causes of cytopenia ruled out in the setting of bone marrow morphology that is not diagnostic of MDS or any other malignancy, and hematolytic conditions have been ruled out. Peripheral blood cytopenia is defined as hemoglobin (hgb) <11.3 g/dL (7 mmol/L) in women and hgb <12.9 g/dL (8 mmol/L) in men, platelet count <150 x 109/L, or neutrophil count <1.8 x 109/L

Menopause, if being a female, defined as females >45 years of age who have experienced amenorrhea for minimum 12 months, without any other obvious pathological or physiological cause

≥18 years of age

Disqualifiers

Any prior treatment with metformin

A diagnosis of diabetes mellitus

Therapeutic radiation, immunosuppressive therapy (with the exception of corticosteroids), or chemotherapy within the past year

Treatment with granulocyte colony-stimulating factor within the past 30 days

Trial design

Treatments tested in this trial

  • Metformin

Treatment groups

40 Participants
are divided into 1 treatment group

Sponsors and collaborators

Kirsten Grønbæk

Lead sponsor

Rigshospitalet, Denmark

Sponsor institution

Steno Diabetes Center Copenhagen

Collaborator

Zealand University Hospital

Collaborator

Van Andel Research Institute

Collaborator

Herlev Hospital

Collaborator

Technical University of Denmark

Collaborator

Region Hovedstadens Apotek

Collaborator

University of Copenhagen

Collaborator