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RecruitingInterventionalPhase 1

A Phase 1 Study of ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies.

NCT ID: NCT06656494Sponsor: Beijing InnoCare Pharma Tech Co., Ltd.Last updated: 2026-04-13

Summary

Evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of ICP-248 in combination with azacitidine in patients with acute myelogenous leukemia and Myelodysplastic Syndromes.

Arms & interventions

  • DrugICP-248

    Eligible patients will receive ICP-248 orally as per the protocol，once daily for every 28 days as one treatment cycle

  • DrugAzacitidine

    Eligible patients will receive azacitidine subcutaneously or intravenously as per the protocol，once daily on days 1-7 of each 28-day cycle.

Outcome measures

Primary

  • Incidence, type, and severity of dose-limiting toxicity (DLT).

    Time frame: 2.5 years

  • Recommended phase II dose (RP2D) and/or maximum tolerated dose (MTD).

    Time frame: 2.5 years

  • The incidence, nature, and severity of adverse events (AEs) as assessed per National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE v5.0) criteria.

    Time frame: 2.5 years

  • AML cohort:Composite complete remission rate by Investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Composite complete remission rate by completion of cycle 2 by Investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • MDS cohort：mOR rate, including CR, mCR, and PR, assessed by Investigator at any time point during the study per revised IWG 2006 MDS Criteria.

    Time frame: 2.5 years

Secondary

  • AML cohort：Composite complete remission rate: The proportion of subjects with complete remission (CR) and CR with incomplete hematologic recovery (CRi) by Investigator per European Leukemia Net (ELN) 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Composite complete remission rate by completion of cycle 2 by Investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • The incidence, nature, and severity of adverse events (AEs) as assessed per NCI-CTCAE v5.0 criteria.

    Time frame: 2.5 years

  • Maximum concentration (Cmax)of ICP-248.

    Time frame: 2.5 years

  • Area under the curve (AUC) of ICP-248.

    Time frame: 2.5 years

  • Time of maximum observed plasma(Tmax)of ICP-248.

    Time frame: 2.5 years

  • Trough concentration(Ctrough) of ICP-248.

    Time frame: 2.5 years

  • Apparent clearance (CL/F) of ICP-248.

    Time frame: 2.5 years

  • AML cohort：Partial Response (PR) by investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Overall survival (OS) by investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Duration of Response (DOR) by investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Event-free Survival (EFS) by investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Relapse-free Survival (RFS) by investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • AML cohort：Morphologic leukemia-free state (MLFS) by investigator per ELN 2017 criteria.

    Time frame: 2.5 years

  • MDS cohort：Modified overall response (mOR) rate, including CR, marrow complete response (mCR), and PR, assessed by Investigator at any time point during the study per revised International Working Group (IWG) 2006 MDS Criteria

    Time frame: 2.5 years

  • MDS cohort：Complete remission(CR) rate by Investigator per revised IWG 2006 MDS Criteria

    Time frame: 2.5 years

  • MDS cohort：Event-free survival (EFS) by Investigator per revised IWG 2006 MDS Criteria

    Time frame: 2.5 years

  • MDS cohort：Duration of modified overall response (DmOR) by Investigator per revised IWG 2006 MDS Criteria

    Time frame: 2.5 years

  • MDS cohort：Overall survival(OS) by Investigator per revised IWG 2006 MDS Criteria

    Time frame: 2.5 years

  • MDS cohort：Marrow complete response (mCR) rate by Investigator per revised IWG 2006 MDS Criteria

    Time frame: 2.5 years

Eligibility criteria

Sex: AllAge: 18 Years and olderHealthy volunteers: No
Inclusion Criteria: Eligible subjects must meet all of the following criteria: 1. Subject must have confirmation of diagnosis of AML (except for acute promyelocytic leukemia \[APL\]) or MDS per 2016 World Health Organization (WHO) criteria. 2. For AML (except for APL) cohort: 1. Previously treated relapsed/refractory AML subjects 2. Treatment-naïve AML subjects should be: ≥60 years of age OR ≥18 years and \<60 years will be eligible if the subject has at least one of the following co-morbidities, which make the subject unfit for intensive chemotherapy 3. For MDS cohort: Adult TN MDS and R/R MDS: revised International Prognostic Scoring System (IPSS-R) score \> 3 and bone marrow blasts ≥ 5%. 4. Subject must have a projected life expectancy of at least 12 weeks. 5. Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 30 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft-Gault formula. 6. Subject must have adequate liver function Exclusion Criteria: 1. R/R AML or R/R MDS with no response or intolerance to post azacitidine or BCL-2i. 2. Subject has acute promyelocytic leukemia (French-American-British Class M3 AML) . 3. Subject has known central nervous system (CNS) leukemia. 4. Suggest patients with active hepatitis B or C virus infection 5. History of immunodeficiency, including a positive human immunodeficiency virus (HIV) antibody test. 6. Subjects have another active malignancy within the past 2 years before study entry, except for curatively treated.

Study locations (2)

Yale University, Yale Cancer Center

New Haven, Connecticut, 06520

Recruiting
Farah Fasihuddin · Contact
(203) 494-4610farah.fasihuddin@yale.edu

NYU Langone Health

New York, New York, 10016

Recruiting
Antoine Mesidor · Contact
212-263-4403antoine.mesidor@nyulangone.org