Modulation of Stem Cell Differentiation in Individuals With High Risk Clonal Haematopoiesis (NCT07435636) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Modulation of Stem Cell Differentiation in Individuals With High Risk Clonal Haematopoiesis
Australia80 participantsStarted 2026-04-13
Plain-language 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.
Who can participate
Age range60 Years – 85 Years
SexALL
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Inclusion criteria
✓. Age ≥ 60 and ≤ 85 years old
✓. Clonal Cytopenia of Uncertain Significance (CCUS), defined by all of the following:
✓. Receiving and adherent to suppressive antiretroviral therapy for at least 12 months
✓. CD4+T cell count ≥ 0.35 x 109/L
✓. HIV viral load \< 50 copies/mL
✓. 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
Exclusion criteria
✕. ANC \< 0.5 x109/L
✕. Serum AST (Aspartate transaminase) or ALT (Alanine aminotransaminase) \> 3 times of upper limit of normal
What they're measuring
1
Change in variant allele fraction (VAF) in peripheral blood mononuclear cells (PBMC) at the completion of 24 weeks of treatment.
Timeframe: End of Treatment (Week 24)
Trial details
NCT IDNCT07435636
SponsorClinical Hub for Interventional Research (CHOIR)