Early Intervention in High Risk CCUS
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorLachelle D. Weeks, MD, PhD
About 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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥18 years.
Hemoglobin (Hgb) <12 g/dL for women and <13g/dL for men
Absolute neutrophil count (ANC) < 1.8 × 109/L*
Platelet count (Plt) <150 × 109/L *Patients known to have a Duffy-null genotype must have anemia (Hgb < 12g/dL for women, Hgb <13g/dL for men) and/or thrombocytopenia (Plt < 150 × 109/L) to be eligible for this study.
Disqualifiers
Concurrent primary malignancy requiring active cytotoxic chemotherapy and/or ionizing radiation therapy.
Known inherited bone marrow failure disorder and/or germline predisposition to hematologic malignancy.
Receipt of anti-cancer therapy including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide, and targeted anti-cancer therapies including PARP inhibitors within the last 6 months. Patients with complete surgical resection of a tumor are not excluded from this study.
Anti-cancer therapy, including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide and targeted agents such as PARP inhibitors, planned in the next 6 months. Patients on hormonal adjuvant therapy for nonmetastatic breast and prostate cancer or other minimally-myelosuppressive maintenance therapies for non-metastatic cancer may be eligible at the discretion of the study PI.
Trial design
Design model
Parallel
Treatments tested in this trial
Inqovi
DrugCombination of a nucleoside metabolic inhibitor and cytidine deaminase inhibitor, 10mg DEC / 100mg CED tablet, taken orally per protocol.
Treatment groups
108 Participants
are divided into 2 treatment groupsGroup A: Inqovi CohortExperimental treatment 1 intervention
Group B: Observational CohortNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
Feasibility Failure Rate (FFR)
FFR is defined by the proportion of eligible subjects with higher risk CCUS who enter the study and opt to participate in the early intervention as opposed to observation only cohort.
Time frame
Treatment duration up to 12 cycles (28 days per cycle).Secondary outcomes
1
Grade 3-5 Treatment-related Toxicity Rate
All grade 3-5 adverse events (AE) with treatment attribution of possibly, probably or definite based on CTCAEv5 that are not resolved in accordance with treatment guidelines were counted. Rate is the percentage of treated participants experiencing at least one of these adverse events as defined during the time of observation.
Time frame
AE evaluated on treatment at day 1, 8, 15, 22 on cycle 1, then day 1 on cycle 2-12, and EOT. Median treatment duration for this study cohort was 6 months (range T1- T2).2
Treatment Tolerability Rate (TTR)
TTR will be measured by the percentage of participants in the early intervention treatment arm that complete ≥3 cycles of oral DEC/CED.
Time frame
Treatment up to 12 cycles, (28 days per cycle).3
Hematologic Response Rate (HRR)
HRR defined as proportion of participants achieved hematologic response by 2018 World Health Organization (WHO) International Working Group (IWG) MDS response criteria.
Time frame
Response collected every 3 months on treatment. Treatment duration up to 12 cycles, (28 days per cycle).4
Median Overt Myeloid Neoplasia (MN)-Free Survival
MN-free survival based on the Kaplan-Meier method is defined as the duration of time from study entry to overt myeloid neoplasia diagnosed per the 5th edition of the WHO Classification of Haematolymphoid Tumors.
Time frame
Disease evaluated every 3 months on treatment, then every 12 months off treatment, up to 2 years for interventional cohort, up to 3 years for observation cohort.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Lachelle D. Weeks, MD, PhD
Lead sponsor
Dana-Farber Cancer Institute
Sponsor institution
Astex Pharmaceuticals, Inc.
Collaborator
Break Through Cancer
Collaborator
Source ClinicalTrials.gov