ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies

April 8, 2026 updated by: Beijing InnoCare Pharma Tech Co., Ltd.

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

266

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2010
        • Recruiting
        • St Vincent's Hospital
        • Contact:
    • Western Australia
      • Perth, Western Australia, Australia, 6000
  • China
    • Anhui
      • Hefei, Anhui, China, 230001
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100044
        • Recruiting
        • Peking University People's Hospital
        • Contact:
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, China, 400042
        • Recruiting
        • The First Affiliated Hospital of Chongqing Medical University
        • Contact:
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Recruiting
        • Nanfang Hospital Southern Medical University
        • Contact:
      • Guangzhou, Guangdong, China, 510030
    • Henan
      • Zhengzhou, Henan, China, 450000
        • Recruiting
        • Henan Cancer Hospital
        • Contact:
    • Hubei
      • Wuhan, Hubei, China, 430000
        • Recruiting
        • Union Hospital Tongji Medical College Huazhong University of Science and Technology
        • Contact:
    • Jiangsu
      • Suzhou, Jiangsu, China, 215006
        • Recruiting
        • the First Affiliated Hospital of Soochow University
        • Contact:
    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Recruiting
        • The First Affiliated Hospital of Nanchang University
        • Contact:
    • Jilin
      • Changchun, Jilin, China, 130000
        • Recruiting
        • The First Hospital of Jilin University
        • Contact:
    • Liaoning
      • Shengyang, Liaoning, China, 110004
        • Recruiting
        • Shengjing Hospital of China Medical University
        • Contact:
    • Sichuan
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, China, 300192
        • Recruiting
        • Tianjin People's Hospital
        • Contact:
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310012
        • Recruiting
        • The First Affiliated Hospital, Zhejiang University School of Medicine
        • Contact:
  • United States
    • Connecticut
      • New Haven, Connecticut, United States, 06520
        • Recruiting
        • Yale University, Yale Cancer Center
        • Contact:
    • New York
      • New York, New York, United States, 10016

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ICP-248 in combination with azacitidine
Drug: ICP-248
Eligible patients will receive ICP-248 orally as per the protocol，once daily for every 28 days as one treatment cycle
Drug: Azacitidine
Eligible patients will receive azacitidine subcutaneously or intravenously as per the protocol，once daily on days 1-7 of each 28-day cycle.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence, type, and severity of dose-limiting toxicity (DLT).
Time Frame: 2.5 years
2.5 years
Recommended phase II dose (RP2D) and/or maximum tolerated dose (MTD).
Time Frame: 2.5 years
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
2.5 years
AML cohort:Composite complete remission rate by Investigator per ELN 2017 criteria.
Time Frame: 2.5 years
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
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
2.5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
The incidence, nature, and severity of adverse events (AEs) as assessed per NCI-CTCAE v5.0 criteria.
Time Frame: 2.5 years
2.5 years
Maximum concentration (Cmax)of ICP-248.
Time Frame: 2.5 years
2.5 years
Area under the curve (AUC) of ICP-248.
Time Frame: 2.5 years
2.5 years
Time of maximum observed plasma(Tmax)of ICP-248.
Time Frame: 2.5 years
2.5 years
Trough concentration(Ctrough) of ICP-248.
Time Frame: 2.5 years
2.5 years
Apparent clearance (CL/F) of ICP-248.
Time Frame: 2.5 years
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
2.5 years
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
2.5 years
AML cohort：Partial Response (PR) by investigator per ELN 2017 criteria.
Time Frame: 2.5 years
2.5 years
AML cohort：Overall survival (OS) by investigator per ELN 2017 criteria.
Time Frame: 2.5 years
2.5 years
AML cohort：Duration of Response (DOR) by investigator per ELN 2017 criteria.
Time Frame: 2.5 years
2.5 years
AML cohort：Event-free Survival (EFS) by investigator per ELN 2017 criteria.
Time Frame: 2.5 years
2.5 years
AML cohort：Relapse-free Survival (RFS) by investigator per ELN 2017 criteria.
Time Frame: 2.5 years
2.5 years
AML cohort：Morphologic leukemia-free state (MLFS) by investigator per ELN 2017 criteria.
Time Frame: 2.5 years
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
2.5 years
MDS cohort：Complete remission(CR) rate by Investigator per revised IWG 2006 MDS Criteria
Time Frame: 2.5 years
2.5 years
MDS cohort：Event-free survival (EFS) by Investigator per revised IWG 2006 MDS Criteria
Time Frame: 2.5 years
2.5 years
MDS cohort：Duration of modified overall response (DmOR) by Investigator per revised IWG 2006 MDS Criteria
Time Frame: 2.5 years
2.5 years
MDS cohort：Overall survival(OS) by Investigator per revised IWG 2006 MDS Criteria
Time Frame: 2.5 years
2.5 years
MDS cohort：Marrow complete response (mCR) rate by Investigator per revised IWG 2006 MDS Criteria
Time Frame: 2.5 years
2.5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Beijing InnoCare Pharma Tech Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 18, 2024

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

January 1, 2028

Study Registration Dates

First Submitted

October 21, 2024

First Submitted That Met QC Criteria

October 22, 2024

First Posted (Actual)

October 24, 2024

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 8, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ICP-CL-01205

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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