Clonal Cytopenia of Undetermined SignificancePhase 2ROSSNCT07579429
Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial)
A Phase 2 study testing Nicotinamide Riboside and Pterostilbene in people with Clonal Cytopenia of Undetermined Significance and Myelodysplastic Syndrome, run by University of Colorado, Denver at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Clonal Cytopenia of Undetermined Significance, Myelodysplastic Syndrome
- What is being tested?
- Nicotinamide Riboside and Pterostilbene
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 20 May 2026to 18 Dec 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Colorado Hospital
Aurora, Colorado, 80045
Derek Schatz, (720) 848-0628, DEREK.SCHATZ@CUANSCHUTZ.EDU
Maria Amaya, PhD MD
About this study
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
Read the detailed description
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.
- Being tested
- Nicotinamide Riboside, Pterostilbene
- Keywords
- Lower Risk Myelodysplastic Syndromes
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nicotinamide RibosideDrug
Nicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+).
PterostilbeneDrug
Pterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Nicotinamide Riboside; Drug: Pterostilbene |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 24 weeks
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: From start of study drug to end of treatment, approximately 360 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2026
- Study start20 May 2026
- Primary completion18 Dec 2029 (estimated)
- Study completion18 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Clonal Cytopenia of Undetermined SignificanceStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Collaborators
- Cancer League of Colorado
- Responsible party
- Sponsor
- Investigators
- Maria Amaya, PhD MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07579429
- Sponsor's protocol ID
- 25-1665.cc
- Acronym
- ROSS
- Status last verified
- Jun 2026
- First submitted
- 5 May 2026
- Last updated
- 3 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial)
- ClinicalTrials.gov ID
- NCT07579429
- Acronym / sponsor ID
- ROSS / 25-1665.cc
- Keywords
- Lower Risk Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Collaborators
- Cancer League of Colorado
- Study start
- 20 May 2026 (actual)
- Primary completion
- 18 Dec 2029 (estimated)
- Study completion
- 18 Dec 2030 (estimated)
- First posted
- 12 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Nicotinamide Riboside and Pterostilbene - Low Dose | Experimental | Drug: Nicotinamide Riboside, Drug: PterostilbeneParticipants receive oral nicotinamide riboside 250 mg and pterostilbene 50 mg once daily for up to 12 months. |
| Nicotinamide Riboside and Pterostilbene - High Dose | Experimental | Drug: Nicotinamide Riboside, Drug: PterostilbeneParticipants receive oral nicotinamide riboside 250 mg and pterostilbene 50 mg twice daily for up to 12 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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.
Key exclusion criteria (15 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nicotinamide RibosideNicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+). | Drug | Nicotinamide Riboside and Pterostilbene - High Dose, Nicotinamide Riboside and Pterostilbene - Low Dose |
| PterostilbenePterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol. | Drug | Nicotinamide Riboside and Pterostilbene - High Dose, Nicotinamide Riboside and Pterostilbene - Low Dose |
Primary outcome
Preliminary Efficacy: Cytopenia Improvement
Time frame: 24 weeks
To assess efficacy of nicotinamide riboside and pterostilbene supplementation by assessing whether it increased hemoglobin by 1g/dL in 24 weeks.
3 secondary outcomes
Safety Information Collection
Time frame: From enrollment to 30 days after last dose of study drug, approximately 390 days.
Additional Efficacy Outcomes - Platelet and Neutrophil Response
Time frame: From start of study drug to end of treatment, approximately 360 days.
Additional Efficacy Outcomes - Decrease in Variant Allele Frequency
Time frame: From start of study drug to end of treatment, approximately 360 days.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Maria Amaya, PhD MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Nicotinamide Riboside and Pterostilbene in people with Clonal Cytopenia of Undetermined Significance and Myelodysplastic Syndrome, run by University of Colorado, Denver at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Derek Schatz (720-848-0628, derek.schatz@cuanschutz.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Preliminary Efficacy: Cytopenia Improvement (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2026.