This is an open-label, phase 2 study for lower risk MDS and high risk CCUS patients who are transfusion independent. There will be two cohorts enrolled at the same time to measure the effect of nicotinamide riboside and pterostilbene at different doses. The primary goals of the study are: * to assess if study drug improves cytopenias in patients * to determine safety of the study drug in patients
This is an open label, Phase 2 study for lower risk MDS and high risk CCUS patients who are transfusion independent. Ten subjects will be accrued in this protocol in two cohorts. The first five subjects in cohort 1 will be given nicotinamide riboside and pterostilbene 250mg-50mg daily for a year. The second cohort will have five subjects and nicotinamide riboside and pterostilbene will be given 250mg-50mg twice daily for a year. The two cohorts can enroll concurrently. Study visits will be every 90 days. Two bone marrow biopsies will be performed, one at the time of screening and one after 180 days of therapy. The bone marrow biopsy at 180 days will serve to assess responses such as changes in variant allele frequencies, as well as correlative studies.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Nicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+).
Pterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol.
University of Colorado Hospital
Aurora, Colorado, United States
RECRUITINGPreliminary Efficacy: Cytopenia Improvement
To assess efficacy of nicotinamide riboside and pterostilbene supplementation by assessing whether it increased hemoglobin by 1g/dL in 24 weeks.
Time frame: 24 weeks
Safety Information Collection
To determine the safety of nicotinamide riboside and pterostilbene in patients with CCUS and MDS via AE and SAE collection throughout study. Frequency and proportion of specific AEs will be calculated for the entire cohort, as well as for each individual dosing cohort.
Time frame: From enrollment to 30 days after last dose of study drug, approximately 390 days.
Additional Efficacy Outcomes - Platelet and Neutrophil Response
-To assess platelet response and neutrophil response. Platelet response will be defined as an increase of at least 30 x 109/L or an increase from \< 20 x 109/L to \> 20 x 109/L. Neutrophil response will be defined as an increase of at least 100% and an absolute increase of at least 0.5 x 109/L.
Time frame: From start of study drug to end of treatment, approximately 360 days.
Additional Efficacy Outcomes - Decrease in Variant Allele Frequency
-To assess decrease in variant allele frequency by 50%. Secondary end points will include decrease in variant allele frequency by 50%, and platelet and neutrophil response.
Time frame: From start of study drug to end of treatment, approximately 360 days.
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