A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination with Venetoclax/Azacitidine, Venetoclax, or 7+3 in Patients with AML

ID#: NCT05735184

Age: 18 years - 66+

Gender: All

Healthy Subjects: No

Study Phase: Phase 1

Recruitment Status: Recruiting

Start Date: July 18, 2023

End Date: May 01, 2027

Contact Information:
Clinical Operations
858 500 8800
Summary: This Phase 1 study will assess the safety, tolerability, and preliminary antileukemic activity of ziftomenib in combination with venetoclax and azacitidine (ven/aza), ven, and 7+3 for two different molecularly-defined arms, NPM1-m and KMT2A-r.
Eligibility: Key

Inclusion Criteria:

- Patients must have a documented NPM1 mutation or KMT2A rearrangement and have either newly diagnosed or relapsed/refractory AML

- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2

- Adequate liver, renal, and cardiac function according to protocol defined criteria

- A female of childbearing potential must agree to use adequate contraception from the time of screening through 180 days following the last dose of study intervention. A male of childbearing potential must agree to use abstinence or adequate contraception from the time of screening through 90 days following the last dose of study intervention Key

Exclusion Criteria:

- Diagnosis of either acute promyelocytic leukemia or blast chronic myelomonocytic leukemia

- Known history of BCR-ABL alteration

- Advanced malignant hepatic tumor [for patients receiving ven/aza combination]

- Administration of live attenuated vaccines within 14 days prior to, during, or after treatment until B-cell recovery

- Active central nervous system (CNS) involvement by AML.

- Clinical signs/symptoms of leukostasis or WBC > 25,000 / microliter. Hydroxyurea and/or leukapheresis are permitted to meet this criterion

- Not recovered to Grade ≤1 (NCI-CTCAE v5.0) from all nonhematological toxicities except for alopecia

- Known clinically active human immunodeficiency virus, active hepatitis B or active hepatitis C infection

- For newly diagnosed cohorts: received prior chemotherapy for leukemia, except hydroxyurea and/or leukapheresis to control leukocytosis, prior treatment with all-transretinoic acid for initially suspected acute promyelocytic leukemia, or non-HMA therapy for prior myelodysplastic syndrome

- For relapsed/refractory cohorts: received chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational < 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug

- Uncontrolled intercurrent illness including, but not limited to, cardiac illness as defined in the protocol

- Mean corrected QT interval corrected for heart rate by Fredericia's formula (QTcF) >480 ms on triplicate ECGs

- Uncontrolled infection

- Women who are pregnant or lactating

- An active malignancy and currently receiving chemotherapy for that malignancy or disease that is uncontrolled/progressing