RecruitingPhase 1

Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia

Phase 1 Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia

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 tests ziftomenib with quizartinib, two medicines taken by mouth, to find safe doses and see how well they work. It is for people with acute myeloid leukemia (AML) or myeloid mixed-phenotype acute leukemia (MPAL) that came back or did not respond to treatment and has certain gene changes.

The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r \~ 40-45%).

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

Eligibility

Who can join

  • The age rule lists 18 years, but the comparison sign is unclear. Ask the study team to confirm.
  • Have AML or myeloid MPAL that came back or did not respond to treatment, with an NPM1, KMT2A, or NUP98 gene change.
  • Be able to do most daily activities, with an ECOG activity score below 2.
  • Have a white blood cell count below 25,000 per microliter. Treatment to lower the count before joining is allowed.
  • Have a heart pumping measure above 40% and liver and kidney tests that meet study rules. Some exceptions apply when leukemia affects these tests.
  • Be able to swallow pills. You or your parent or guardian must be willing and able to give informed consent.
  • Usually, wait at least 14 days after prior treatment, or longer depending on how long the drug stays in your body. Some treatment exceptions apply for fast-growing leukemia or controlled leukemia in the brain or spinal cord.
  • Follow the study's birth control rules during treatment and afterward: at least 7 months for women who could become pregnant and 4 months for men, unless the men cannot father a child.

Who can't join

  • Past treatment with a menin inhibitor, a type of leukemia drug, or use of other leukemia drugs during the study unless specifically allowed.
  • A serious stomach, gut, or body-chemistry problem that could keep the pills from being absorbed, or another active cancer being treated.
  • Active hepatitis B, hepatitis C, or HIV infection; an infection that is not controlled; pregnancy; or breastfeeding.
  • Certain serious heart problems, a stroke, or a mini-stroke within the past 6 months.
  • A lasting heart rate below 50 beats per minute without a pacemaker; known, suspected, or family history of inherited long QT syndrome; a QTc heart rhythm test above 450 milliseconds; or very high blood pressure despite treatment.
  • Active leukemia in the brain or spinal cord.
  • Certain levels of graft-versus-host disease (GVHD), when donated cells attack the body: active acute GVHD above grade 2, moderate or severe limited chronic GVHD, or any extensive chronic GVHD.
  • Other health, mental health, legal guardianship, or drug-use issues that could make joining unsafe or prevent following study rules, as judged by the study doctor.

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

Official eligibility criteria

Inclusion Criteria: 1. Age . 18 years. 2. ECOG performance status of \< 2. 3. Relapsed or refractory AML or myeloid mixed-phenotype acute leukemia (MPAL) with NPM1mt, or KMT2Ar, or NUP98r. 4. WBC must be below 25,000/ ƒÊL at time of enrollment. Patients may receive cytoreduction prior to enrollment. 5. Baseline ejection fraction must be \> 40%. 6. Adequate hepatic function (total bilirubin \< 2x upper limit of normal (ULN) unless increase is due leukemic involvement (\<2.5 ULN), unless due to ongoing hemolysis or Gilbert's syndrome and AST and/or ALT \< 3x ULN unless considered due to leukemic involvement, in which case direct bilirubin or AST and/or ALT \< 5x ULN will be considered eligible). 7. Adequate renal function with an estimated glomerular filtration rate . 50 mL/min (using Cockcroft-Gault) unless related to disease. 8. Able to swallow pills. 9. Patient or parent/guardian is willing and able to provide informed consent. 10. In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 14 days for cytotoxic or non-cytotoxic (immunotherapy agent(s), or an interval of 5 half-lives of the prior therapy, whichever is longer. Oral hydroxyurea and/or cytarabine (up to 1 g/m2) for patients with rapidly proliferative disease is allowed before the start of study therapy, as needed, for clinical benefit and after discussion with the PI. Concurrent therapy for central nervous system (CNS) prophylaxis or continuation of therapy for controlled CNS disease is permitted. 11. Women of childbearing potential must agree to adequate methods of contraception during the study and at least 7 months for females and 4 months for males after the last treatment. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study and at least 4 months after the last treatment. Exclusion Criteria: 1. Prior treatment with a menin inhibitor. 2. The use of other chemotherapeutic agents or anti-leukemic agents is not permitted during study with the following exceptions (1) intrathecal chemotherapy for prophylactic use or for controlled CNS leukemia. (2) use of hydroxyurea for patients with rapidly proliferative disease or for control of counts during differentiation syndrome. (3) use of steroids for treatment of differentiation syndrome. 3. Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications. 4. Patients with a concurrent active malignancy under treatment. 5. Known active hepatitis B (HBV) or Hepatitis C (HCV) or HIV infection. 6. Female subjects who are pregnant or breast-feeding. 7. Patient has an active uncontrolled infection. 8. Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class .II), life-threatening, uncontrolled arrhythmia including ventricular arrythmias or torsades de pointes, cerebrovascular accident, or transient ischemic attack. 9. History of sustained bradycardia of less than 50 beats per minute unless the subject has a pacemaker. 10. Diagnosis of or suspicion of congenital long QT syndrome (including family history of congenital long QT syndrome). 11. Uncontrolled hypertension with a systolic blood pressure .180 mmHg or diastolic blood pressure .110 mmHg, sustained despite optimal medical management. 12. QTc \>450 msec using the Fridericia Formula. 13. History of or any concurrent condition, therapy, or laboratory abnormality that in the Investigator's opinion might confound the results of the study, interfere with the patient's participation for the full duration of the study, or is not in the best interest of the patient to participate. Patients under legal protection measure (guardianship, trusteeship or safeguard of justice) and/or uncontrolled psychiatric comorbidities, ongoing illicit substance abuse, inability, any impairment or unwillingness to comply with the treatments, follow-up, requirements and procedures of this clinical trial. 14. Clinically active central nervous system (CNS) leukemia. 15. Patients with Grade \> 2 active acute GVHD, moderate or severe limited chronic GVHD, or extensive chronic GVHD of any severity.

Study sites (1)

TX