Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial) (ROSS)
2026年6月1日 更新者：University of Colorado, Denver
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
研究概览
详细说明
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.
研究类型
介入性
注册 (估计的)
10
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Derek Schatz
- 电话号码：720-848-0628
- 邮箱：derek.schatz@cuanschutz.edu
学习地点
-
-
Colorado
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Aurora、Colorado、美国、80045
- 招聘中
- University Of Colorado Hospital
-
副研究员：
- Jonathan Gutman, MD
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副研究员：
- Manali Kamdar, MD
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副研究员：
- Daniel Sherbenou, MD
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副研究员：
- Christine McMahon, MD
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副研究员：
- Steven Bair, MD
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接触：
- Derek Schatz
- 电话号码：(720) 848-0628
- 邮箱：DEREK.SCHATZ@CUANSCHUTZ.EDU
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副研究员：
- Ajay Major, MD
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副研究员：
- Daniel Pollyea, MD
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首席研究员：
- Maria Amaya, PhD MD
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副研究员：
- Matthew Angelos, MD
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副研究员：
- Cassandra Duarte, MD
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副研究员：
- Bradley Haverkos, MD
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副研究员：
- Andrew Kent, MD
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副研究员：
- Jorge Monge-Urrea, MD
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
不
描述
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.
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：非随机化
- 介入模型：并行分配
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Nicotinamide Riboside and Pterostilbene - Low Dose
Participants receive oral nicotinamide riboside 250 mg and pterostilbene 50 mg once daily for up to 12 months.
|
Nicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+).
Pterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol.
|
|
实验性的：Nicotinamide Riboside and Pterostilbene - High Dose
Participants receive oral nicotinamide riboside 250 mg and pterostilbene 50 mg twice daily for up to 12 months.
|
Nicotinamide riboside is an orally administered dietary supplement and a vitamin B3 derivative that serves as a precursor to nicotinamide adenine dinucleotide (NAD+).
Pterostilbene is an orally administered dietary supplement and a naturally occurring polyphenol structurally related to resveratrol.
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Preliminary Efficacy: Cytopenia Improvement
大体时间：24 weeks
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To assess efficacy of nicotinamide riboside and pterostilbene supplementation by assessing whether it increased hemoglobin by 1g/dL in 24 weeks.
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24 weeks
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Safety Information Collection
大体时间：From enrollment to 30 days after last dose of study drug, approximately 390 days.
|
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.
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From enrollment to 30 days after last dose of study drug, approximately 390 days.
|
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Additional Efficacy Outcomes - Platelet and Neutrophil Response
大体时间：From start of study drug to end of treatment, approximately 360 days.
|
-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.
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From start of study drug to end of treatment, approximately 360 days.
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Additional Efficacy Outcomes - Decrease in Variant Allele Frequency
大体时间：From start of study drug to end of treatment, approximately 360 days.
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-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.
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From start of study drug to end of treatment, approximately 360 days.
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Patient Survival
大体时间：From enrollment to end of study, approximately 390 days.
|
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.
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From enrollment to end of study, approximately 390 days.
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 首席研究员：Maria Amaya, PhD MD、University of Colorado, Denver
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2026年5月20日
初级完成 (估计的)
2029年12月18日
研究完成 (估计的)
2030年12月18日
研究注册日期
首次提交
2026年5月5日
首先提交符合 QC 标准的
2026年5月5日
首次发布 (实际的)
2026年5月12日
研究记录更新
最后更新发布 (实际的)
2026年6月3日
上次提交的符合 QC 标准的更新
2026年6月1日
最后验证
2026年6月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.