Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia
Kura Oncology, Inc.
Summary
Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations. Ziftomenib is a type of therapy known to target the menin pathway in cancer cells. This protocol has 2 separate studies that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) AML treatments in patients with certain genetic mutations who have not received any treatment for their AML. In the first study, the Nonintensive Therapy Study, older patients or those with serious medical problems will receive the SOC therapies venetoclax (ven) and azacitidine (aza), plus either ziftomenib or a placebo. In the second study, the Intensive Therapy Study, medically fit patients will receive (a) the SOC therapies cytarabine and daunorubicin, plus either ziftomenib or a placebo during a first treatment phase called induction, (b) cytarabine plus either ziftomenib or a placebo during a second treatment phase called consolidation, and (c) ziftomenib or a placebo during a third treatment phase called maintenance. The physician will determine which study is the appropriate treatment for the patient, but neither the patient nor their physician will know whether the patient has been assigned to receive ziftomenib or a placebo. This design is called "double-blinded".
Description
This protocol encompasses two phase 3, randomized, double-blind, placebo-controlled clinical studies to assess the efficacy, safety, and tolerability of ziftomenib in combination with: (a) the standard of care (SOC) nonintensive regimen (venetoclax \[ven\]+azacitidine \[aza\]) in untreated adults with nucleophosmin 1 mutated (NPM1-m) acute myeloid leukemia (AML); or (b) the SOC intensive regimen (cytarabine+daunorubicin induction, referred to here as 7+3, and cytarabine consolidation) in untreated adults with NPM1-m or lysine\[K\]-specific methyltransferase 2A rearranged (KMT2A-r) AML, as well…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: The following criteria apply to both the Nonintensive Therapy Study and the Intensive Therapy Study unless otherwise noted: * Age ≥18 years at time of signing the informed consent form. * Diagnosis of AML per the 2022 WHO Classification of Hematolymphoid Tumors (5th Edition). * Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2. * Adequate liver and kidney function according to protocol requirements. * A female of childbearing potential must agree to use adequate contraception from the time of screening through 180 days following the last dose…
Interventions
- DrugZiftomenib
Oral administration
- DrugPlacebo
Oral administration
- DrugVenetoclax
Oral administration
- DrugAzacitidine (AZA)
Intravenous or subcutaneous administration
- DrugDaunorubicin
Intravenous administration
- DrugCytarabine (Ara-C)
Intravenous administration
Locations (39)
- Banner MD Anderson Cancer CenterGilbert, Arizona
- University of California, FresnoClovis, California
- University of California, San DiegoLa Jolla, California
- Cedars-Sinai Medical CenterLos Angeles, California
- University of California, IrvineOrange, California
- University of ColoradoAurora, Colorado