Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial) (ROSS)
1 juin 2026 mis à jour par: 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
Aperçu de l'étude
Statut
Recrutement
Intervention / Traitement
Description détaillée
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.
Type d'étude
Interventionnel
Inscription (Estimé)
10
Phase
- Phase 2
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Coordonnées de l'étude
- Nom: Derek Schatz
- Numéro de téléphone: 720-848-0628
- E-mail: derek.schatz@cuanschutz.edu
Lieux d'étude
-
-
Colorado
-
Aurora, Colorado, États-Unis, 80045
- Recrutement
- University Of Colorado Hospital
-
Sous-enquêteur:
- Jonathan Gutman, MD
-
Sous-enquêteur:
- Manali Kamdar, MD
-
Sous-enquêteur:
- Daniel Sherbenou, MD
-
Sous-enquêteur:
- Christine McMahon, MD
-
Sous-enquêteur:
- Steven Bair, MD
-
Contact:
- Derek Schatz
- Numéro de téléphone: (720) 848-0628
- E-mail: DEREK.SCHATZ@CUANSCHUTZ.EDU
-
Sous-enquêteur:
- Ajay Major, MD
-
Sous-enquêteur:
- Daniel Pollyea, MD
-
Chercheur principal:
- Maria Amaya, PhD MD
-
Sous-enquêteur:
- Matthew Angelos, MD
-
Sous-enquêteur:
- Cassandra Duarte, MD
-
Sous-enquêteur:
- Bradley Haverkos, MD
-
Sous-enquêteur:
- Andrew Kent, MD
-
Sous-enquêteur:
- Jorge Monge-Urrea, MD
-
-
Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
Non
La description
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.
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Non randomisé
- Modèle interventionnel: Affectation parallèle
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: 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.
|
|
Expérimental: 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.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Preliminary Efficacy: Cytopenia Improvement
Délai: 24 weeks
|
To assess efficacy of nicotinamide riboside and pterostilbene supplementation by assessing whether it increased hemoglobin by 1g/dL in 24 weeks.
|
24 weeks
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Safety Information Collection
Délai: 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.
|
From enrollment to 30 days after last dose of study drug, approximately 390 days.
|
|
Additional Efficacy Outcomes - Platelet and Neutrophil Response
Délai: 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.
|
From start of study drug to end of treatment, approximately 360 days.
|
|
Additional Efficacy Outcomes - Decrease in Variant Allele Frequency
Délai: From start of study drug to end of treatment, approximately 360 days.
|
-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.
|
From start of study drug to end of treatment, approximately 360 days.
|
Autres mesures de résultats
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Patient Survival
Délai: 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.
|
From enrollment to end of study, approximately 390 days.
|
Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Collaborateurs
Les enquêteurs
- Chercheur principal: Maria Amaya, PhD MD, University of Colorado, Denver
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude (Réel)
20 mai 2026
Achèvement primaire (Estimé)
18 décembre 2029
Achèvement de l'étude (Estimé)
18 décembre 2030
Dates d'inscription aux études
Première soumission
5 mai 2026
Première soumission répondant aux critères de contrôle qualité
5 mai 2026
Première publication (Réel)
12 mai 2026
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
3 juin 2026
Dernière mise à jour soumise répondant aux critères de contrôle qualité
1 juin 2026
Dernière vérification
1 juin 2026
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 25-1665.cc
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Oui
Étudie un produit d'appareil réglementé par la FDA américaine
Non
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