Inqovi for Clonal Cytopenia

Overseen By Maximilian Stahl, MD

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Lachelle D. Weeks, MD, PhD

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 21

Duration: 12 months

108 Participants Needed

Trial Summary

What is the purpose of this trial?

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).

Research Team

Lachelle Weeks, MD

Principal Investigator

Dana-Farber Cancer Institute

Eligibility Criteria

This trial is for individuals with high-risk Clonal Cytopenia of Unknown Significance (CCUS), which includes conditions like low blood cell counts, anemia, and thrombocytopenia. Participants should not have other health issues that could interfere with the study.

Inclusion Criteria

I will use effective birth control during and up to 6 months after treatment.

I am 18 years old or older.

Unexplained cytopenia(s) for at least 4 months (Hemoglobin (Hgb) <12 g/dL for women, <13g/dL for men; Absolute neutrophil count (ANC) < 1.8 × 10^9/L; Platelet count (Plt) <150 × 10^9/L)

Exclusion Criteria

I have a genetic condition that affects my bone marrow or increases my risk of blood cancer.

Laboratory evidence indicative of clinically significant red cell hemolysis

I am scheduled for cancer treatment within the next 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive oral Decitabine/cedazuridine for 12 cycles, each cycle lasting 28 days

12 months

In-clinic visits on Cycle 1 days 8, 15, 22 and Cycle 2-12 Day 1

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

In-clinic visits at Cycle 19 Day 21, Cycle 25 Day 1, Cycle 31 Day 1, and Cycle 36 Day 28

Observation

Participants in the observational cohort undergo regular assessments without active treatment

36 months

In-clinic visits with assessments at specified cycles

Treatment Details

Interventions

• Decitabine/cedazuridine

Trial Overview The trial is testing the effects of early treatment using Decitabine/cedazuridine (DEC/CED), an oral medication, compared to just observing patients without active treatment. DEC/CED inhibits certain cellular processes that may be involved in CCUS.

Participant Groups

Group I: Inqovi Cohort Experimental Treatment 1 Intervention

Participants will be enrolled and will complete: * Baseline visit. * In-clinic visits with assessments: Cycle 1 days 8, 15, and 22. * In-clinic visits with assessments and bone marrow biopsies: Cycles 2 - 12 Day 1. * Cycle 1 - 12: • Days 1 - 5 of 28 day cycle: Predetermined dose of Inqovi 1x daily. * End of Treatment visit, Cycle 13 Day 1, with assessments and bone marrow biopsy. * Follow up in-clinic visits with assessments: Cycle 19 Day 21, Cycle 25 Day 1, Cycle 31 Day 1, and Cycle 36 Day 28. Bone marrow biopsy at Cycle 25 Day 1. * End of Study visit, Cycle 36 Day 28, with assessments and bone marrow biopsy.

Group II: Observational Cohort Active Control 1 Intervention

Participants will complete: * Baseline visit. * In-clinic visits with assessments: Cycle 7 Day 1, Cycle 13 Day, Cycle 19 Day, Cycle 25 Day 1, and Cycle 31 Day 1. * Bone marrow biopsies every 12 months at Cycle 13 Day 1 and Cycle 25 Day. * End of study visit, Cycle 36 Day 28, with assessments and bone marrow biopsy.

Who Is Running the Clinical Trial?

Lachelle D. Weeks, MD, PhD

Lead Sponsor

Trials

1

Recruited

110+

Maximilian Stahl, MD

Lead Sponsor

Trials

2

Recruited

130+

Astex Pharmaceuticals, Inc.

Industry Sponsor

Trials

97

Recruited

7,400+

Dr. Harren Jhoti

Astex Pharmaceuticals, Inc.

Chief Executive Officer since 2007

PhD in Biochemistry from Birkbeck College, London

Dr. Harold N. Keer

Astex Pharmaceuticals, Inc.

Chief Medical Officer since 2020

MD

Break Through Cancer

Collaborator

Trials

2

Recruited

120+