NCT06802146

Early Intervention in High Risk CCUS

A Multi-Site Break Through Cancer Pilot Study Testing the Feasibility and Safety of Therapeutic Intervention for Patients With High-risk Clonal Cytopenia of Undetermined Significance (CCUS)

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 108 (estimated)
• Sponsor: Lachelle D. Weeks, MD, PhD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This research is being done to find out more about the potential risks and benefits of early treatment in participants with high risk Clonal Cytopenia of Unknown Significance (CCUS). This study will give eligible CCUS participants the option of either being observed or taking an oral drug as treatment. The names of the study drug involved in this study is: -Decitabine/cedazuridine (DEC/CED) (a nucleoside metabolic inhibitor and cytidine deaminase inhibitor).

Detailed description

This is an open-label, multicenter pilot study testing the feasibility and safety of early pharmacologic intervention, decitabine/cedazuridine, in participants with higher-risk clonal cytopenia of unknown significance (CCUS). The U.S. Food and Drug Administration (FDA) has not approved DEC/CED for CCUS but it has been approved for other uses. The research study procedures include screening for eligibility, in-clinic treatment visits, electrocardiograms, echocardiograms, bone marrow biopsies, and blood tests. Participants who choose to enroll in the intervention cohort will receive the oral drug for 1 year and will continue in a post-treatment observation period for 2 years after treatment. Participants in both groups will each participate in the study for 3 years total. It is expected that the study will continue to enroll up to 108 participants in total or until there are 30 participants enrolled in the early intervention cohort, whichever occurs first. Astex Oncology is funding this research study by providing the drug Decitabine/cedazuridine.

Conditions

• Clonal Cytopenia of Undetermined Significance
• Cytopenia

Interventions

Type	Name	Description
DRUG	Inqovi	Combination of a nucleoside metabolic inhibitor and cytidine deaminase inhibitor, 10mg DEC / 100mg CED tablet, taken orally per protocol.

Timeline

• Start date: 2025-02-07
• Primary completion: 2026-12-01
• Completion: 2028-12-01
• First posted: 2025-01-31
• Last updated: 2025-03-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study