Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial) (ROSS)
1 giugno 2026 aggiornato da: 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
Panoramica dello studio
Stato
Reclutamento
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Interventistico
Iscrizione (Stimato)
10
Fase
- Fase 2
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Derek Schatz
- Numero di telefono: 720-848-0628
- Email: derek.schatz@cuanschutz.edu
Luoghi di studio
-
-
Colorado
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Aurora, Colorado, Stati Uniti, 80045
- Reclutamento
- University of Colorado Hospital
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Sub-investigatore:
- Jonathan Gutman, MD
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Sub-investigatore:
- Manali Kamdar, MD
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Sub-investigatore:
- Daniel Sherbenou, MD
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Sub-investigatore:
- Christine McMahon, MD
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Sub-investigatore:
- Steven Bair, MD
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Contatto:
- Derek Schatz
- Numero di telefono: (720) 848-0628
- Email: DEREK.SCHATZ@CUANSCHUTZ.EDU
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Sub-investigatore:
- Ajay Major, MD
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Sub-investigatore:
- Daniel Pollyea, MD
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Investigatore principale:
- Maria Amaya, PhD MD
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Sub-investigatore:
- Matthew Angelos, MD
-
Sub-investigatore:
- Cassandra Duarte, MD
-
Sub-investigatore:
- Bradley Haverkos, MD
-
Sub-investigatore:
- Andrew Kent, MD
-
Sub-investigatore:
- Jorge Monge-Urrea, MD
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-
Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
No
Descrizione
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.
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Non randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: 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.
|
|
Sperimentale: 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.
|
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Preliminary Efficacy: Cytopenia Improvement
Lasso di tempo: 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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Safety Information Collection
Lasso di tempo: 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
Lasso di tempo: 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.
|
|
Additional Efficacy Outcomes - Decrease in Variant Allele Frequency
Lasso di tempo: 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.
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Altre misure di risultato
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Patient Survival
Lasso di tempo: 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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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Collaboratori
Investigatori
- Investigatore principale: Maria Amaya, PhD MD, University of Colorado, Denver
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Effettivo)
20 maggio 2026
Completamento primario (Stimato)
18 dicembre 2029
Completamento dello studio (Stimato)
18 dicembre 2030
Date di iscrizione allo studio
Primo inviato
5 maggio 2026
Primo inviato che soddisfa i criteri di controllo qualità
5 maggio 2026
Primo Inserito (Effettivo)
12 maggio 2026
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
3 giugno 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
1 giugno 2026
Ultimo verificato
1 giugno 2026
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- 25-1665.cc
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
Sì
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .