Actively Recruiting
A Multi-Site Pilot Study Testing the Feasibility and Safety of Decitabine/Cedazuridine Treatment for Patients With High-risk Clonal Cytopenia of Undetermined Significance (CCUS)
Led by Lachelle D. Weeks, MD, PhD ยท Updated on 2026-04-23
108
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
L
Lachelle D. Weeks, MD, PhD
Lead Sponsor
A
Astex Pharmaceuticals, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying high-risk Clonal Cytopenia of Unknown Significance (CCUS) to understand the potential risks and benefits of early treatment. This open-label, multicenter pilot trial evaluates the feasibility and safety of using the oral drug decitabine/cedazuridine (DEC/CED), which is approved for other conditions but not yet for CCUS. The study is funded by Astex Oncology and supported by lead investigator Dr. Lachelle D. Weeks, MD, PhD. Participants can choose between two groups: one receiving the oral drug Inqovi (decitabine/cedazuridine) daily for five days in each 28-day cycle over 12 cycles (approximately one year), and another observational group receiving no intervention. The intervention group has frequent clinic visits including bone marrow biopsies and assessments, continuing with a post-treatment observation period for two years. The observation group undergoes periodic assessments and bone marrow biopsies over three years. Participants will have multiple visits for clinical assessments, blood tests, electrocardiograms, echocardiograms, and bone marrow biopsies. Researchers will monitor treatment feasibility, tolerability, hematologic response, mutation changes, inflammatory markers, and quality of life surveys over the course of three years. Safety and disease progression are evaluated regularly during treatment and follow-up periods, with the total participation lasting up to three years.
CONDITIONS
Brief Title
Early Intervention in High Risk CCUS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Unexplained cytopenia(s) lasting at least 4 months confirmed by at least two lab tests
- Low hemoglobin, neutrophil count, or platelet count as defined
- At least one pathogenic variant in a myeloid driver gene with variant allele frequency of 2% or more
- High risk score according to Clonal Hematopoiesis Risk Calculator
- Bone marrow biopsy consistent with CCUS and no overt blood cancer
- ECOG performance status between 0 and 2
- Adequate liver and kidney function
- Ability to understand and sign informed consent
- For treatment group, women of childbearing potential and males with partners must use effective contraception during and after treatment
You will not qualify if you...
- Active cancer requiring chemotherapy or radiation
- Known inherited bone marrow failure or genetic risk for blood cancers
- Anti-cancer therapy within the last 6 months or planned in next 6 months, except certain maintenance therapies
- Diagnosis of myelodysplastic syndromes, myeloproliferative neoplasms, chronic myelomonocytic leukemia, acute myeloid leukemia, or other blood cancers
- Presence of other blood cancer precursor conditions like smoldering multiple myeloma, MGUS, or monoclonal B cell lymphocytosis
- Active uncontrolled infections
- Recent or planned live attenuated vaccinations
- Evidence of significant red cell destruction
- Hypersplenism or portal hypertension
- Pregnancy or breastfeeding
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 cycles of 28 days each
Participants in the intervention cohort receive oral Inqovi daily for up to 12 cycles (28 days each) with in-clinic visits for assessments and bone marrow biopsies throughout treatment.
Multiple in-clinic visits during treatment cycles, including Cycle 1 Days 8, 15, 22; Cycles 2-12 Day 1; and End of Treatment visit
Duration - Up to 2 years after treatment
Participants continue in post-treatment observation with periodic assessments and bone marrow biopsies for up to 2 years after treatment ends.
In-clinic visits at Cycle 19 Day 21, Cycle 25 Day 1, Cycle 31 Day 1, and Cycle 36 Day 28 with bone marrow biopsy at Cycle 25 Day 1
Duration - 3 years total participation
Participants in the observational cohort are monitored with periodic visits and bone marrow biopsies over the 3-year study period.
In-clinic visits at baseline, Cycle 7 Day 1, Cycle 13 Day 1, Cycle 19 Day 1, Cycle 25 Day 1, and Cycle 31 Day 1 with bone marrow biopsies at Cycle 13 Day 1 and Cycle 25 Day 1
Trial Site Locations
Total: 1 location
1
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
Research Team
L
Lachelle Weeks, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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