RecruitingPhase 1 / Phase 2

Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm

A Phase I/II Study of Azacitidine, Venetoclax, and Gilteritinib for Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome With an Activating FLT3 Mutation

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 the safety, best dose, and effects of gilteritinib given with azacitidine and venetoclax. It is for adults with certain blood cancers that have a gene change called FLT3 and have come back or did not respond to treatment. One study group includes adults with newly diagnosed acute myeloid leukemia (AML).

This phase I/II trial studies the side effects and best dose of gilteritinib and to see how well it works in combination with azacitidine and venetoclax in treating patients with FLT3-mutation positive acute myeloid leukemia, chronic myelomonocytic leukemia, or high-risk myelodysplastic syndrome/myeloproliferative neoplasm that has come back (recurrent) or has not responded to treatment (refractory). Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gilteritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine, venetoclax, and gilteritinib may work better compared to azacitidine and venetoclax alone in treating patients with acute myeloid leukemia, chronic myelomonocytic leukemia, or myelodysplastic syndrome/myeloproliferative neoplasm.

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

Eligibility

Who can join

  • Be age 18 or older.
  • Have AML, chronic myelomonocytic leukemia (CMML), or certain other blood cancers called MDS/MPN. The allowed diagnosis and past treatment depend on the study group; MDS/MPN must be intermediate-2 or high risk.
  • Have an FLT3-ITD or FLT3 D835 gene change in the cancer cells.
  • Have a score of 3 or lower on the ECOG scale, which measures how well you can do daily activities.
  • Have liver blood tests within study limits. Some exceptions are allowed.
  • Have kidney function of at least 30 mL/min on a test called creatinine clearance.
  • Be able to swallow.
  • Sign a form agreeing to join after learning about the study.

Who can't join

  • For the newly diagnosed AML group only: prior AML treatment, with some exceptions, or being able and willing to receive intensive starting chemotherapy.
  • Certain heart rhythm problems or severe active heart failure.
  • A serious active infection not controlled by treatment, or leukemia in the brain or spinal cord.
  • Known HIV, certain positive hepatitis B tests, or active hepatitis C. Some people with hepatitis antibodies may join if no virus is found in their blood.
  • Another cancer that could affect the study's safety or results. Other past or current cancers need review by the study leader.
  • Use of certain medicines or herbs within 3 days before joining, such as carbamazepine, phenytoin, rifampin, or St. John's wort.
  • Chemotherapy or experimental leukemia drugs within 7 days before joining, unless an exception applies. Some medicines to lower blood cell counts are allowed.
  • Pregnancy. Women who can become pregnant need a negative test and birth control during treatment and for 6 months after. Men must use birth control during treatment and for 4 months after. No breastfeeding during gilteritinib treatment or for 2 months after.

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

Official eligibility criteria

Inclusion Criteria: * Diagnosis: * Phase I cohort: Adults ≥ 18 years with relapsed/refractory FLT3-mutated AML or CMML or other MDS/MPN that is intermediate-2 or high-risk by the International Prognostic Scoring System * Phase II cohort A: Adults ≥ 18 years with newly diagnosed FLT3-mutated AML * Phase II cohort B: Adults ≥ 18 years with relapsed/refractory FLT3-mutated AML or CMML or other MDS/MPN that is intermediate-2 or high-risk by the International Prognostic Scoring System * For all cohorts, patients with either FLT3-internal tandem duplication (FLT3-ITD) or FLT3 D835 mutations will be eligible * Performance status ≤ 3 (Eastern Cooperative Oncology Group \[ECOG\] scale) * Total serum bilirubin ≤ 2.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the principal investigator (PI) * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 3 x ULN, unless due to the underlying leukemia approved by the PI * Creatinine clearance ≥ 30 mL/min * Ability to swallow * Signed informed consent Exclusion Criteria: * Prior therapies: * Phase I cohort: No restriction based on prior therapies * Phase II cohort A: Patients with prior therapy for AML are not eligible. Prior therapy for antecedent hematologic disorder is allowed. Prior hydroxyurea or cytarabine given for purposes of cytoreduction is also allowed. Prior all trans-retinoic acid given for presumed acute promyelocytic leukemia is also allowed * Phase II cohort B: No restriction on number of prior therapies * Patients suitable for and willing to receive intensive induction chemotherapy (for Phase II cohort A only) * Congenital long QT syndrome or corrected QT interval by Fridericia (QTcF) \> 450 msec. Repeat electrocardiograms (EKGs) after correction of electrolytes or discontinuation of QT prolonging medications are allowed to meet entry criteria. In cases where QTcF \> 450 msec is considered to be falsely increased due to inaccurate automated reading and not clinically significant (e.g. due to bundle branch block), patients are still eligible if cardiologist reviews and documents that QTcF is ≤ 450 msec when manually measured * Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of improvement despite antimicrobial treatment) * Active grade III-V cardiac failure as defined by the New York Heart Association criteria * Active central nervous system leukemia * Known human immunodeficiency virus (HIV) seropositive * Known hepatitis B surface antigen seropositive or known or suspected active hepatitis C infection * Note: Patients who have isolated positive hepatitis B core antibody (i.e., in the setting of negative hepatitis B surface antigen and negative hepatitis B surface antibody) must have an undetectable hepatitis B viral load. Patients who have positive hepatitis C antibody may be included if they have an undetectable hepatitis C viral load * Patients with a prior or concurrent malignancy whose natural history or treatment is not anticipated to interfere with the safety or efficacy assessment of the investigational regimen may be included only after discussion with the PI * Consumed strong inducer of cytochrome P450, family 3, subfamily A (CYP3A) or p-glycoprotein within 3 days of study enrollment. Agents include but are not limited to: carbamazepine, phenytoin, rifampin, and St. John's wort * Treatment with any investigational antileukemic agents or chemotherapy agents in the last 7 days before study entry, unless full recovery from side effects has occurred or patient has rapidly progressive disease judged to be life-threatening by the investigator. Prior recent treatment with corticosteroids, hydroxyurea and/or cytarabine (given for cytoreduction) permitted * Pregnant women will not be eligible; women of childbearing potential should have a negative pregnancy test prior to entering on the study and be willing to practice methods of contraception throughout the study period and for at least 6 months after the last dose of study drugs. Women do not have childbearing potential if they have had a hysterectomy or are postmenopausal without menses for 12 months. In addition, men enrolled on this study should understand the risks to any sexual partner of childbearing potential and should practice an effective method of birth control throughout the study period and for at least 4 months after the last dose of study drugs. Lactating women (or those planning to breastfeed) should not breastfeed during treatment of gilteritinib and for at least 2 months after the last dose of gilteritinib

Study sites (1)

TX