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Modulation of Stem Cell Differentiation in Individuals With High Risk Clonal Haematopoiesis | Clinical Trials | Clareo Health

NOT_YET_RECRUITINGPHASE2

Modulation of Stem Cell Differentiation in Individuals With High Risk Clonal Haematopoiesis

A Multi-centre, Double-blind, Placebo-controlled Randomised Phase II Trial to Evaluate the Effect of Low Dose Decitabine and Tetrahydrouridine in Individuals With High-risk Clonal Haematopoiesis

NCT07435636•Clinical Hub for Interventional Research (CHOIR)

View on ClinicalTrials.gov

Summary

Clonal hematopoiesis (CH) is characterized by the overproduction of blood cells derived from a single hematopoietic stem and progenitor cell (HSPC) harboring certain somatic mutations. It is linked to serious outcomes, including cardiovascular disease, myeloid neoplasm (MN), and increased mortality. Clonal Cytopenia of Uncertain Significance (CCUS) is a CH subtype characterized by associated persistent cytopenia. It affects approximately 10 % of people over 70 and is the most advanced precursor state with the highest risk of progressing to MN. There is an unmet need to determine whether modifying CH can prevent adverse outcomes. Current blood cancer therapies are too toxic for precursor conditions like CH. MOSAIC is a randomized double-blind placebo-controlled trial that will test a novel low-dose oral epigenetic therapy-decitabine with tetrahydrouridine (Dec+THU) in CCUS. It has shown targeted, non-cytotoxic reversal of common CH mutations in preclinical and early-phase studies. The goal is to develop a safe and effective therapy in CCUS that restores normal blood cell production and prevents progression.

Eligibility

Age

60 - 85 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Age ≥ 60 and ≤ 85 years old
•2. Clonal Cytopenia of Uncertain Significance (CCUS), defined by all of the following:
•a. Persistent cytopenia, present on at least two occasions, at least four months apart, with no other cause identified: i. Hemoglobin (Hb) \< 120 g/L in people born female, and \< 130 g/L in people born male ii. Platelet count \< 150 x 109/L iii. Absolute Neutrophil Count (ANC) \< 1.8 x109/L b. Clonal hematopoiesis (CH) driver mutation confirmed by custom gene panel mutation analysis c. absence of features diagnostic for defined myeloid neoplasm (MN) on bone marrow examination
•3. CH driver mutation variant allele fraction (VAF) of ≥ 10%
•4. For participants living with HIV:
•1. Receiving and adherent to suppressive antiretroviral therapy for at least 12 months
•2. CD4+T cell count ≥ 0.35 x 109/L
•3. HIV viral load \< 50 copies/mL
•6\. Performance status by Eastern Cooperative Oncology Group (ECOG) Criteria of 0 or 1 7. For participants who are of childbearing potential, or whose partners are of childbearing potential:
•1. Agreement to use at least two highly effective (per Clinical Trial Facilitation Group) contraceptive methods throughout the course of the trial, and for 6 months following the last dose of trial drug
+11 more criteria

Exclusion Criteria

•1. ANC \< 0.5 x109/L
•2. Serum AST (Aspartate transaminase) or ALT (Alanine aminotransaminase) \> 3 times of upper limit of normal
•3. Calculated or measured creatinine clearance ≤ 50 mL/min
•4. Significant active cardiac disease within the previous 6 months, including:
•1. New York Heart Association (NYHA) class III or IV congestive heart failure
•2. Unstable angina or angina requiring surgical or medical intervention
•3. Myocardial infarction
•4. New or unstable cardiac arrhythmia. Stable or controlled arrhythmias are permitted
•5. Active systemic infections:
•1. Infection with ongoing signs/symptoms related to the infection without improvement despite appropriate anti-infectives
+1 more criteria

Locations (3)

Canberra Health Services

Canberra, Australian Capital Territory, Australia

Prince of Wales Hospital

Randwick, New South Wales, Australia

Westmead Hospital

Westmead, New South Wales, Australia

Key Dates

Start Date

April 13, 2026

Primary Completion Date

April 14, 2030

Completion Date

October 1, 2030

Last Updated

February 27, 2026

Enrollment

ESTIMATED participants

Conditions

Clonal Cytopenia of Uncertain SignificanceCCUS Clonal Cytopenia of Undetermined SignificanceClonal Hematopoiesis

Interventions

Oral Decitabine and Tetrahydrouridine

DRUG

Matched Placebo (Capsules)

DRUG

Contacts

Professor Mark Polizzotto

CONTACT

+61000000000 mosaic.jcsmr@anu.edu.au

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 27, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Modulation of Stem Cell Differentiation in Individuals With High Risk Clonal Haematopoiesis

Inclusion Criteria

Exclusion Criteria

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