Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity of Colorado, Denver
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Subject must voluntarily sign and date an informed consent, approved by an Institutional Review Board (IRB), prior to the initiation of any research directed procedures.
Stated willingness to comply with all study procedures and be available for the duration of the study.
Be a male or female aged ≥ 18 years of age.
For persons of reproductive potential, use of highly effective method(s) of contraception.
Disqualifiers
Subject is known to be positive for HIV with uncontrolled disease. HIV testing is not required.
Subject is known to be positive for hepatitis B or C infection with the exception of those with an undetectable viral load. Hepatitis B or C testing is not required and subjects with serologic evidence of prior vaccination to HBV may participate.
Significant active cardiac disease within the previous 6 months including: New York Heart Association heart failure > class 2, unstable angina, or myocardial infarction.
Renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or bleeding disorder independent of leukemia.
Trial design
Design model
Parallel
Treatments tested in this trial
Nicotinamide Riboside
DrugNicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+).
Pterostilbene
DrugPterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol.
Treatment groups
10 Participants
are divided into 2 treatment groupsGroup A: Nicotinamide Riboside and Pterostilbene - Low DoseExperimental treatment 2 interventions
Group B: Nicotinamide Riboside and Pterostilbene - High DoseExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
Preliminary 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 weeksSecondary outcomes
1
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.2
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.3
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.Other outcomes
1
Patient Survival
Exploratory efficacy endpoints will include survival. For MDS subjects, progression free survival will be defined as time from MDS diagnosis to progression to AML or death from any cause. Progression free survival for CCUS will be defined as time from CCUS diagnosis to progression to MDS, AML or death from any cause.
Time frame
From enrollment to end of study, approximately 390 days.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
University of Colorado, Denver
Lead sponsor
Cancer League of Colorado
Collaborator
Source ClinicalTrials.gov