NCT06656494 · Beijing InnoCare Pharma Tech Co., Ltd.
ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies
What this study is about
Evaluate the safety, tolerability, how the drug moves through the body, and preliminary effectiveness of ICP-248 in combination with azacitidine in patients with acute myelogenous leukemia and Myelodysplastic Syndromes.
View original scientific description
Evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of ICP-248 in combination with azacitidine in patients with acute myelogenous leukemia and Myelodysplastic Syndromes.
Interventions
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.
Primary outcome measures
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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Eligible subjects must meet all of the following criteria:
- Subject must have confirmation of diagnosis of AML (except for acute promyelocytic leukemia \[APL\]) or MDS per 2016 World Health Organization (WHO) criteria.
- For AML (except for APL) cohort:
- Previously treated relapsed/refractory AML subjects
- 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
- For MDS cohort: Adult TN MDS and R/R MDS: revised International Prognostic Scoring System (IPSS-R) score \> 3 and bone marrow blasts ≥ 5%.
- Subject must have a projected life expectancy of at least 12 weeks.
- 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.
- Subject must have adequate liver function
Exclusion criteria
- R/R AML or R/R MDS with no response or intolerance to post azacitidine or BCL-2i.
- Subject has acute promyelocytic leukemia (French-American-British Class M3 AML) .
- Subject has known central nervous system (CNS) leukemia.
- Suggest patients with active hepatitis B or C virus infection
- History of immunodeficiency, including a positive human immunodeficiency virus (HIV) antibody test.
- Subjects have another active malignancy within the past 2 years before study entry, except for curatively treated.
Where
- Orange, California
- New Haven, Connecticut
- Tampa, Florida
- New York, New York
- Chapel Hill, North Carolina
- Milwaukee, Wisconsin
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 18, 2026 · Source of record for eligibility and locations