Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial)

NCT07579429 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 25-1665.cc
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Clonal Cytopenia of Undetermined Significance; Myelodysplastic Syndrome

Keywords

Lower Risk Myelodysplastic Syndromes

Outcome Measures

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.

  24 weeks

Secondary Outcomes (3)

• Safety Information Collection

  From enrollment to 30 days after last dose of study drug, approximately 390 days.

• Additional Efficacy Outcomes - Platelet and Neutrophil Response

  From start of study drug to end of treatment, approximately 360 days.

• Additional Efficacy Outcomes - Decrease in Variant Allele Frequency

  From start of study drug to end of treatment, approximately 360 days.

Other Outcomes (1)

• Patient Survival

  From enrollment to end of study, approximately 390 days.

Study Arms (2)

Nicotinamide Riboside and Pterostilbene - Low Dose

EXPERIMENTAL

Participants receive oral nicotinamide riboside 250 mg and pterostilbene 50 mg once daily for up to 12 months.

Drug: Nicotinamide Riboside; Drug: Pterostilbene

Nicotinamide Riboside and Pterostilbene - High Dose

EXPERIMENTAL

Participants receive oral nicotinamide riboside 250 mg and pterostilbene 50 mg twice daily for up to 12 months.

Drug: Nicotinamide Riboside; Drug: Pterostilbene

Interventions

Nicotinamide Riboside DRUG

Nicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+).

Nicotinamide Riboside and Pterostilbene - High Dose; Nicotinamide Riboside and Pterostilbene - Low Dose

Pterostilbene DRUG

Pterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol.

Nicotinamide Riboside and Pterostilbene - High Dose; Nicotinamide Riboside and Pterostilbene - Low Dose

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Subject must have confirmation of high risk CCUS (defined by CHRS score) or the diagnosis of MDS in the lower risk category as defined by IPSS-M less than 0.
• Patients must have ECOG of ≤ 2
• Subject must have adequate renal function as demonstrated by a calculated creatinine clearance ≥ 30 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula.
• Subject must have adequate liver function as demonstrated by:
• aspartate aminotransferase (AST) ≤ 3.0 × ULN
• alanine aminotransferase (ALT) ≤ 3.0 × ULN
• Unless due to Gilbert's syndrome, patients must have a total bilirubin ≤ 3 × ULN.
• Subject is informed that consumption of the following fruits is prohibited 3 days prior to the initiation of study treatment and throughout participation: grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Star fruit.

You may not qualify if:

• 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.
• Subject has any history of clinically significant condition(s) that in the opinion of the investigator would adversely affect his/her participating in this study including, but not limited to:
• 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.
• Subject has a malabsorption syndrome or other condition that precludes enteral route of administration. This includes history of inflammatory bowel disease (e.g. Crohn's disease, ulcerative colitis), celiac disease (e.g. sprue), prior gastrectomy or upper bowel removal, or any other gastrointestinal disorder or defect that would interfere with the absorption, distribution, metabolism or excretion of the study drug and/or predispose the subject to an increased risk of gastrointestinal toxicity.
• Subject exhibits evidence of uncontrolled systemic infection requiring therapy (viral, bacterial or fungal). Uncontrolled is defined as ongoing signs/symptoms related the infection without improvement despite appropriate antibiotics, antiviral therapy and/or other treatment.
• Subject has a history of other malignancies prior to study entry, with the exception of:
• Adequately treated in situ carcinoma of the breast or cervix uteri
• Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin
• Prostate cancer not requiring therapy beyond hormonal therapy
• Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent
• Any subject who is transfusion dependent and a candidate for MDS therapy such as erythroid stimulating agents, thrombopoietin receptor agonists, Lusparercept, Imetelstat or hypomethylating agents.
• Pregnant or breast-feeding females. A pregnancy test will be obtained at the time of screening if applicable.
• Known or suspected hypersensitivity to nicotinamide riboside and pterostilbene.

Sponsors & Collaborators

• University of Colorado, Denver: lead
• Cancer League of Colorado: collaborator

Study Sites (1)

University of Colorado Hospital

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

nicotinamide-beta-riboside; Pterocarpus marsupium

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Maria Amaya, PhD MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Derek Schatz

CONTACT

720-848-0628; derek.schatz@cuanschutz.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2026
• First Posted: May 12, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): December 18, 2029
• Study Completion (Estimated): December 18, 2030
• Last Updated: May 12, 2026

Record last verified: 2026-05

Locations

US (1)