Nicotinamide Riboside and Pterostilbene in lower risk MDS and high-risk CCUS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label, two-cohort phase 2 trial enrolls a total of ten adults with lower risk myelodysplastic syndrome or high-risk clonal cytopenia of undetermined significance who are transfusion independent. Participants receive nicotinamide riboside and pterostilbene at either 250 mg/50 mg once daily or 250 mg/50 mg twice daily for 12 months, with concurrent enrollment possible for both groups. Study visits occur every 90 days, and two bone marrow biopsies are planned (at screening and after 180 days) to assess cytopenia responses and correlative molecular changes, including variant allele frequencies. Primary aims are to see if the combination improves cytopenias and to evaluate safety. The study is conducted at a single site in Colorado with lead sponsorship from the University of Colorado, Denver, in collaboration with the Cancer League of Colorado.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of Colorado Hospital
  Aurora, Colorado, United States Recruiting

Study contacts

• Principal investigator: Maria Amaya, PhD MD — University of Colorado, Denver
• Study coordinator: Derek Schatz
• Email: derek.schatz@cuanschutz.edu
• Phone: 720-848-0628

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.