This phase II trial tests how well nicotinamide riboside and pterostilbene work to improve cytopenia and delay disease progression in patients with clonal cytopenia of undetermined significance (CCUS) or myelodysplastic syndrome (MDS). Cytopenia is a condition in which a patient has a lower-than-normal number of blood cells, including red blood cells, white blood cells, and platelets. CCUS is a condition characterized by cytopenia but without changes in the bone marrow. CCUS can progress to MDS, which is a group of diseases in which the bone marrow does not make enough healthy blood cells. Most patients with CCUS only require observation, but some patients can be at higher risk for progression to MDS, and patients with MDS are at risk for progression to acute myeloid leukemia. Nicotinamide riboside is a form of vitamin B3. It helps with the production of an enzyme required for cellular energy production, and research has shown it may help support the formation of new blood cells. Pterostilbene is a nutrient with anti-oxidant and anti-inflammatory properties with the potential to prevent oxidative stress and cell damage. It also alters gene expression and prevents the activation of many signaling pathways involved in cancer formation. Nicotinamide riboside and pterostilbene may improve cytopenia and delay disease progression in patients with CCUS or MDS.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT07579429.
Locations matching your search criteria
United States
Colorado
Aurora
UCHealth University of Colorado HospitalStatus: Active
Contact: Maria Laura Amaya
Phone: 720-766-9786
PRIMARY OBJECTIVE:
I. To assess preliminary efficacy information from 10 subjects on whether nicotinamide riboside and pterostilbene supplementation improves cytopenias in patients with lower risk MDS or high risk CCUS.
SECONDARY OBJECTIVE:
I. To assess feasibility of treatment, further assess efficacy, and collect additional safety information of nicotinamide riboside and pterostilbene in the low risk MDS and high risk CCUS population.
TERTIARY/EXPLORATORY OBJECTIVES:
I. To obtain correlative data on the effects of nicotinamide riboside and pterostilbene in the bone marrow cells of subjects taking nicotinamide riboside and pterostilbene, including clonal tracking, and to understand its efficacy in different subgroups of MDS or CCUS patients.
II. To obtain and compare efficacy data from the two dosing cohorts.
III. To obtain quality of life information on patients taking nicotinamide riboside and pterostilbene with Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaires.
OUTLINE: Patients are assigned to 1 of 2 cohorts.
COHORT 1: Patients receive nicotinamide riboside orally (PO) once daily (QD) and pterostilbene PO QD for 1 year in the absence of disease progression or unacceptable toxicity. Patients also undergo bone marrow aspiration and biopsy, as well as collection of blood samples throughout the trial.
COHORT 2: Patients receive nicotinamide riboside PO twice daily (BID) and pterostilbene PO BID for 1 year in the absence of disease progression or unacceptable toxicity. Patients also undergo bone marrow aspiration and biopsy, as well as collection of blood samples throughout the trial.
After completion of study treatment, patients are followed up at 30 days.
Lead OrganizationUCHealth University of Colorado Hospital
Principal InvestigatorMaria Laura Amaya
