HPB-092 for the Treatment of Relapsed and Refractory Acute Myeloid Leukemia (NCT07137637) | Clinical Trial Compass
Not Yet RecruitingPhase 1
HPB-092 for the Treatment of Relapsed and Refractory Acute Myeloid Leukemia
60 participantsStarted 2025-12
Plain-language summary
HPB-092 effectively inhibits Fms-like tyrosine kinase 3 (FLT3) mutants with comparable or superior potency to approved FLT3 inhibitors and demonstrates improved selectivity, potentially reducing toxicity. Its highly selective and potent inhibition of Interleukin-1 Receptor-Associated Kinase 4 (IRAK4) may provide additional therapeutic benefits that could enhance treatment efficacy and durability for patients with relapsed or refractory acute myeloid leukemia (RR-AML), further improving clinical outcomes in this population. HPB-092 also has a favorable safety profile, with no major risks identified in preclinical studies.
Phas 1 Study Outline:
1. This is a multicenter, open-label, phase 1 study to evaluate the safety and efficacy of oral HPB-092 as monotherapy in patients with RR-AML.
2. The study aims to assess safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy.
3. It consists of two parts: Part A for dose escalation and Part B for dose expansion, involving single or multiple doses.
4. Patients must be diagnosed with morphologically documented RR-AML according to World Health Organization (WHO) 2022 criteria.
5. Baseline assessments will include RR-AML with FLT3 mutations, spliceosome mutations in SF3B1 and U2AF1, as well as other biomarkers, which will be monitored throughout the study.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. The patient must have a diagnosis of morphologically documented relapsed or refractory acute myeloid leukemia (AML) according to the World Health Organization (WHO) 2022 criteria, and must meet one of the following conditions:
. The patients should have a stable transfusion requirement prior to enrollment as specified in the protocol.
. Male or non-pregnant, non-lactating female patients aged 18 years or older.
. The patient is not suitable for other known therapies that have clinical benefits for the disease.
. Life expectancy of ≥12 weeks, and Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
. Renal Function: Serum creatinine \<1.5× the upper limit of normal (ULN) or Estimated creatinine clearance ≥ 60 mL/min as calculated using a standard method \[Cockcroft-Gault formula, Modification of Diet in Renal Disease (MDRD) equation, or estimated glomerular filtration rate (eGFR) calculation\]. Liver Function: Total serum bilirubin ≤1.5× ULN unless the patient has liver involvement by the primary disease (≤3× ULN); and AST and ALT ≤2.5× ULN unless the patient has hepatic metastasis (≤5× ULN). Cardiac Function: Left ventricular ejection fraction (LVEF) \>50% as measured by echocardiogram or MUGA scan.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adverse Events and Treatment-Emergent Adverse Events [Safety and Tolerability]
Timeframe: From the first dose (Cycle 1 day 1) of HPB-092 treatment until 28 days after the completion of treatment, or after early termination or withdrawal from the study, with a duration of up to 24 months
2
Dose-Limiting Toxicity [Safety and Tolerability]
Timeframe: Days 1-28 of the first cycle (each cycle is 28 days)
3
Maximum Tolerated Dose [Safety and Tolerability]
Timeframe: Days 1-28 of the first cycle (each cycle is 28 days)
. Acute effects of any prior therapy must be resolved to baseline severity or Grade ≤ 1 per CTCAE v5.0, except for adverse events (AEs) that do not constitute a safety risk according to the investigator's judgment.
. For females of childbearing potential, a serum pregnancy test must be negative within 7 days before enrollment.
Exclusion criteria
. Diagnosed with acute promyelocytic leukemia (APL) or BCR-ABL-positive leukemia.
. The patient has had a malignancy other than AML within the past five years as specified in the protocol.
. The patient has persistent non-hematological toxicities of ≥ Grade 2 (per CTCAE v5.0) from prior treatment.
. History of Hematopoietic Stem Cell Transplant (HSCT) as specified in the protocol.
. The patient has clinically active central nervous system (CNS) leukemia.
. The patient has a disseminated intravascular coagulation (DIC) abnormality.
. Recent surgery or radiation therapy as specified in the protocol.
. History of Class 3 or more severe heart failure according to NYHA classification, or left ventricular ejection fraction (LVEF) below 45%, or Fridericia-corrected QT interval (QTcF) \> 450 ms at screening.