RecruitingPhase 1 / Phase 2

Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1/2 Open Label, Multicenter, Dose Escalation and Expansion Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of HM43239 in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study looks for a safe dose of tuspetinib that may help treat certain blood cancers. It tests tuspetinib alone, with venetoclax, or with venetoclax plus azacitidine. It is for people whose AML, MDS-IB2, or CMML came back or did not respond to treatment, or who have newly diagnosed AML.

The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Parts A/B/C: Have AML, MDS-IB2, or CMML confirmed by testing. The cancer must have come back after improving or not responded to at least one round of treatment.
  • Parts A/B/C: Be able to care for yourself and be up and about for more than half the day. Be able to take medicine by mouth and be expected to live at least 3 months.
  • Parts A/B/C: Wait the required time after earlier treatments, usually 2 to 4 weeks or until certain drugs have left your body. A shorter wait may be approved.
  • Parts A/B/C: Meet the study's liver and kidney test limits.
  • Parts A/B/C: Follow pregnancy testing and highly effective birth control rules. Do not breastfeed or donate eggs or sperm during the required period, which ends 90 days after the last dose.
  • Parts A/B/C: Agree not to join another study that gives treatment while taking this study's treatment.
  • Part D: Have newly diagnosed AML that has not been treated, and be unable to receive intensive chemotherapy. Age 75 or older is listed as a reason.
  • Part D: Meet limits for daily activity and liver tests. The supplied Part D criteria are cut off, so the full requirements are not available.

Who can't join

  • Parts A/B/C: Have acute promyelocytic leukemia (APL), leukemia with the BCR-ABL gene change, or another active cancer besides AML, MDS-IB2, or CMML.
  • Parts A/B/C: Have ongoing moderate or worse side effects not related to blood counts from earlier cancer treatment, or a history of severe such side effects from a tyrosine kinase inhibitor, a type of cancer drug.
  • Parts A/B/C: Had a stem cell transplant within 2 months or a donor immune-cell infusion within 30 days. Certain lasting transplant side effects or donor cells attacking your body and needing treatment also rule out joining.
  • Parts A/B/C: Have leukemia in the brain, spinal cord, or their coverings; another cancer-related brain or spinal cord disease; or DIC, a serious blood-clotting problem.
  • Parts A/B/C: Had major surgery or radiation treatment within 4 weeks before the first study dose.
  • Parts A/B/C: Have certain serious heart failure, heart rhythm, or heart electrical problems. Some people with past heart failure may qualify based on a heart scan.
  • Parts A/B/C: Have an active infection, including COVID-19; HIV; active hepatitis B or C; or another active liver disorder.
  • Parts A/B/C: Have any other condition that the study doctor believes makes joining unsuitable. No Part D exclusion list was provided.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria for Parts A/B/C: * Study participant is defined as having morphologically documented primary or secondary AML, MDS-IB2 (≥ 10% bone marrow blasts), or CMML by the World Health Organization (WHO) criteria (2016), and fulfills one of the following: 1. Refractory to at least 1 cycle of prior therapy 2. Relapsed after achieving remission with a prior therapy * Study participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2. * Study participant's interval from prior treatment to time of study drug administration is at least 2 weeks for cytotoxic agents (except hydroxyurea given for controlling blast cells), at 4 weeks for biologic or cellular immunotherapies, or least 5 half-lives for prior experimental agents or noncytotoxic agents, including immunosuppressive therapy post hematopoietic stem cell transplantation (HSCT). Upon discussion with the Medical Monitor, a shorter than stated washout period may be considered provided that the study participant has recovered from any clinically relevant safety issue and recovered to Grade ≤ 1 toxicity from prior therapies. * Study participant must meet the following criteria as indicated on the clinical laboratory tests. 1. Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3× institutional upper limit normal (ULN) 2. Total serum bilirubin ≤ 1.5× institutional ULN 3. Creatinine clearance as calculated by the Cockcroft-Gault formula or measured by 24-h urine collection of \> 45 mL/min. * Study participant is suitable for oral administration of study drug and has minimum life expectancy (≥ 3 months) * Female study participants must be either: * Of non-childbearing potential 1. Post-menopausal (defined as at least 1 year without any menses) prior to screening, or 2. Documented surgically sterile or status post hysterectomy (at least 1 month prior to screening) * Or, if of childbearing potential, 1. Must have a negative serum or urine pregnancy test at screening (within 72 hours prior to start of treatment), and 2. Must use an acceptable highly effective method of birth control starting at screening and throughout the study period and for 90 days after the final study drug administration. * Female study participants must not be breastfeeding at screening and during the study period, and for 90 days after the final study drug administration * Female study participants must not donate ova starting at screening and throughout the study period, and for 90 days after the final study drug administration. * Male study participants and their female spouse/partners who are of childbearing potential must be using highly effective contraception starting at screening and continue throughout the study period and for 90 days after the final study drug administration. * Male study participants must not donate sperm starting at screening and throughout the study period and for 90 days after the final study drug administration. * Study participant agrees not to participate in another interventional study while on treatment. Exclusion Criteria for Parts A/B/C: Study participants must not enter the study if any of the following exclusion criteria are met: * Study participant was diagnosed with acute promyelocytic leukemia (APL). * Study participant has known BCR-ABL-positive leukemia. * Study participant has an active malignancy other than AML, MDS-IB2, or CMML. * Study participant has persistent non-hematological toxicities of ≥ Grade 2 (CTCAE v4.03), with symptoms and objective findings, from prior AML, MDS-IB2, or CMML treatment (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, radiation, or surgery) * Study participant has had hematopoietic stem cell transplant (HSCT) and meets any of the following criteria: 1. Has undergone HSCT within the 2-month period prior to the first study dose 2. Has clinically significant graft-versus-host-disease (GVHD) requiring treatment 3. Has ≥ Grade 2 persistent non-hematological toxicity related to the transplant 4. Had a donor lymphocyte infusion (DLI) ≤ 30 days prior to the first study dose. * Study participant has meningeal or central nervous system (CNS) involvement with leukemia or other CNS disease related to underlying and secondary effects of malignancy. * Study participant has disseminated intravascular coagulation abnormality (DIC). * Study participant has had major surgery within 4 weeks prior to the first study dose. * Study participant has had radiation therapy within 4 weeks prior to the first study dose. * Study participant has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or study participant with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram or multigated acquisition (MUGA) scan performed within 3 months prior to study entry results in a left ventricular ejection fraction (LVEF) that is ≥ 45%. * Study participant has any of the following cardiac abnormalities of history: 1. Study participant has any clinically important abnormalities in rhythm, conduction or morphology of resting ECG, e.g., complete left bundle branch block, third-degree heart block, second-degree heart block, or PR interval \> 250 milliseconds (ms). 2. Study participant has a mean QT interval (QTc) by Friderica's method (QTcF) \> 450 ms in three successive screening measurements. 3. Study participant has any factors that increase the risk of QTc prolongation or risk of arrhythmic events, such as congenital long QT, syndrome, family history of long QT syndrome. * Study participant is known to have active infection including any identified active COVID-19 infection. * Study participant is known to have human immunodeficiency virus infection. * Study participant has known active hepatitis B or C, or other active hepatic disorder. * Study participant has any condition which, in the Investigator's opinion, makes the study participant unsuitable for study participation. * Study participant has a history of Grade 3 or 4 non-hematologic toxicity related to tyrosine kinase inhibitor. Inclusion Criteria for Part D: * Study participant is defined as having morphologically documented newly diagnosed previously untreated primary or secondary AML by the World Health Organization (WHO) criteria (2016) and considered ineligible to receive intensive chemotherapy. Study participants ≥ 75 years of age are considered ineligible to receive intensive chemotherapy if they meet any of the following criteria: 1. ECOG Performance Status of 2 or 3; 2. Cardiac history of congestive heart failure (CHF) requiring treatment or ejection fraction ≤ 50% or chronic stable angina; 3. Diffusing capacity of the lung for carbon monoxide (DLCO) ≤ 65% or forced expiratory volume during the first second (FEV1) ≤ 65%; 4. Creatinine clearance ≥ 30 mL/min to \< 45 ml/min; 5. Moderate hepatic impairment with total bilirubin \> 1.5 to ≤ 3.0×ULN; 6. Any other comorbidity that the Investigator judges to be incompatible with intensive chemotherapy must be reviewed and approved by the Medical Monitor during screening and prior to treatment. Study participants are considered ineligible to receive intensive chemotherapy based on their age being ≥ 75 years. * Study participant \< 75 years has an ECOG performance status ≤ 2; study participant ≥ 75 years has an ECOG performance status 0-2. * Study participant must meet the following criteria as indicated on the clinical laboratory tests: 1. Serum AST and ALT ≤ 3× institutional ULN unless considered due to leukemic organ involvement. 2. Total serum bilirubin ≤ 3x institutional ULN unless considered due to leukemic organ involvement for study participants \< 75 years; total serum bilirubin ≤ 1.5x institutional ULN (or ≤ 3x institutional ULN if documented history of Gilbert's syndrome) unless considered due to leukemic organ involvement for study participants ≥ 75 years. 3. Creatinine clearance as calculated by the Cockcroft-Gault formula or measured by 24-h urine collection of ≥ 30 mL/min for study participants \< 75 years; creatinine clearance as calculated by the Cockcroft-Gault formula or measured by 24-h urine collection of ≥ 45 mL/min for study participants ≥ 75 years. * Study participant is suitable for oral administration of study drug and has minimum life expectancy (≥ 3 months) * Female study participants must be either: * Of non-childbearing potential 1. Post-menopausal (defined as at least 1 year without any menses) prior to screening, or 2. Documented surgically sterile or status post hysterectomy (at least 1 month prior to screening) * Or, if of childbearing potential, 1. Must have a negative serum or urine pregnancy test at screening (within 72 hours prior to start of treatment), and 2. Must use an acceptable highly effective contraception starting at screening and throughout the study period and for 90 days after the final study drug administration. * Female study participants must not be breastfeeding at screening and during the study period, and for 90 days after the final study drug administration. * Female study participants must not donate ova starting at screening and throughout the study period, and for 90 days after the final study drug administration. * Male study participants and their female spouse/partners who are of childbearing potential must be using highly effective contraception starting at screening and continue throughout the study period and for 90 days after the final study drug administration. * Male study participants must not donate sperm starting at screening and throughout the study period and for 90 days after the final study drug administration. * Study participant agrees not to participate in another interventional study while on treatment. Exclusion Criteria for Part D: Study participants must not enter the study if any of the following exclusion criteria are met: * Study participant was diagnosed with acute promyelocytic leukemia (APL). * Study participant has known BCR-ABL-positive leukemia. * Study participant has an active malignancy other than AML. * Study participant has received treatment with the following: 1. An HMA, VEN (or other BCL-2 inhibitor), a tyrosine kinase inhibitor (TKI), a FLT3 inhibitor (FLT3i), a hematopoietic stem cell transplant (HSCT), and/or a chemotherapeutic agent for antecedent myeloid neoplasm (Note: Prior chemotherapy for solid tumors considered in remission is allowed.) 2. CAR-T cell therapy 3. Experimental therapies for antecedent myeloid neoplasms 4. Current participation in another research or observational study * Study participant has persistent non-hematological toxicities of ≥ Grade 2 (CTCAE v4.03), with symptoms and objective findings, from treatment for antecedent myeloid neoplasms (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, immunosuppressive therapy, radiation, or surgery). * Study participant has meningeal or central nervous system (CNS) involvement with leukemia or other CNS disease related to underlying and secondary effects of malignancy. * Study participant has disseminated intravascular coagulation abnormality (DIC). * Study participant has had major surgery within 4 weeks prior to the first study dose. * Study participant has had radiation therapy within 4 weeks prior to the first study dose. * Study participant has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or study participant with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram or multigated acquisition (MUGA) scan performed within 3 months prior to study entry results in a left ventricular ejection fraction (LVEF) that is ≥ 45%. * Study participant has any of the following cardiac abnormalities of history: 1. Study participant has any clinically important abnormalities in rhythm, conduction or morphology of resting ECG, e.g., complete left bundle branch block, third-degree heart block, second-degree heart block, or PR interval \> 250 milliseconds (ms). 2. Study participant has a mean QT interval (QTc) by Friderica's method (QTcF) \> 450 ms in three successive screening measurements. 3. Study participant has any factors that increase the risk of QTc prolongation or risk of arrhythmic events, such as congenital long QT, syndrome, family history of long QT syndrome. * Study participant is known to have active infection, including any identified active COVID-19 infection. * Study participant is known to have human immunodeficiency virus infection. * Study participant has known active hepatitis B or C, or other active hepatic disorder. * Study participant has any condition which, in the Investigator's opinion, makes the study participant unsuitable for study participation, including a chronic respiratory disease that requires continuous oxygen, or a significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, or cardiovascular disease, or any other medical condition or known hypersensitivity to any of the study medications including excipients of VEN and AZA that in the opinion of the Investigator would adversely affect participating in this study. * Study participant exhibits evidence of other clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal) or other medical conditions (e.g., infection, heart failure, COPD flare, etc.). * Study participant has a white blood cell count \> 25 × 10\^9/L. (Treatment with hydroxyurea or use of leukapheresis are permitted to meet this criterion.)

Study sites (15)

AL

CA

CT

FL

GA

MA

NC

OH

TX