Recruiting
Phase 2

Nicotinamide Riboside & Pterostilbene

Sponsor:

University of Colorado, Denver

Code:

NCT07579429

Conditions

Clonal Cytopenia of Undetermined Significance

Myelodysplastic Syndrome

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Nicotinamide Riboside

Pterostilbene

Study Details

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

Study ID

NCT07579429

Start date

May 20, 2026

Status verified date

Jun, 2026

Completion date

Dec 18, 2030

Anticipated

Primary completion date

Dec 18, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Study Design

Enrollment

10 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Nicotinamide Riboside and Pterostilbene - Low Dose

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

experimental: Nicotinamide Riboside and Pterostilbene - High Dose

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

Interventions

Nicotinamide Riboside

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

Pterostilbene

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

Primary outcome measure

  • Preliminary Efficacy: Cytopenia Improvement [ Time Frame: 24 weeks ]

Central Contacts and Locations

Central contacts

Derek Schatz

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

Locations

University of Colorado Hospital

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Derek Schatz

(720) 848-0628DEREK.SCHATZ@CUANSCHUTZ.EDU

Principal Investigator:

Maria Amaya, PhD MD

More Information

Sponsor

University of Colorado, Denver

Last update posted

Jun 3, 2026

Last verified

Jun, 2026

Keywords

  • Lower Risk Myelodysplastic Syndromes

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Colorado, Denver on 2026-06-03.