Relapsed and/or refractory Acute Myeloid Leukemia MedDRA version: 21.0 Level: LLT Classification code 10060558 Term: Acute myeloid leukemia recurrent System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: ziftomenib
Product Code: KO-539
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: ziftomenib
CAS Number: 2134675-36-6
Current Sponsor code: KO-539, C17060755-E
Concentration unit:
Sponsors
Kura Oncology, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: 1. Relapsed/refractory (R/R) acute myeloid leukemia (AML) defined as those who have also failed or are not appropriate for any approved standard-of-care (SOC) therapies or hematopoietic stem cell transplant (HSCT)with reappearance of = 5% blasts in the bone marrow (BM). 2. = 18 years of age. 3. Eastern Cooperative Oncology Group performance status of = 2, and a life expectancy of at least 2 months. 4. Peripheral white blood cell counts = 30,000/µL. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 118 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 118
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria 1. Diagnosis of acute promyelocytic leukemia or chronic myelogenous leukemia in blast crisis. 2. Donor lymphocyte infusion 480 ms on triplicate electrocardiograms.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Phase 1 Part 1a: Dose-Escalation - To determine the MTD and/or the RP2D of ziftomenib in patients with R/R AML Phase 1 Part 1b: Dose-Validation/ Cohort Expansion - Determine the safety, tolerability and MBED of ziftomenib in biomarker-specific dosing cohorts, which have demonstrated early biological activity and have been determined to be safe as part of the dose-escalation phase Phase 2: - Assess evidence of ALA of ziftomenib in patients with NPM1-m R/R AML ;Secondary Objective: Phase 1 Part 1a: Dose-Escalation - To investigate the safety and tolerability of ziftomenib in patients with R/R AML - To characterize the PK of ziftomenib and metabolites after single oral (PO) dose administration and after multiple PO dose administrations. - Explore early evidence of ALA in patients with NPM1-m R/R AML Phase 1 Part 1b: Dose-Validation/ Cohort Expansion - Explore early evidence of ALA in patients with R/R AML Phase 2: - Assess evidence of clinical activity of ziftomenib in patients with NPM1-m R/R AML - Assess safety and tolerability of ziftomenib in patients with NPM1-m R/R AML;Primary end point(s): Phase 1 Part a: - Maximum Tolerated Dose (MTD) - Recommended Phase 2 dose (RP2D) Phase 1 Part b: - Safety, tolerability, and efficacy at multiple timepoints during the study Phase 2: - Based on evaluation of CR/CRh;Timepoint(s) of evaluation of this end point: At any time during the clinical trial. Disease assessment will be done when there is clinical evidence of response. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Phase 1 Part a: - Safety/tolerability assessed at multiple timepoints during the study - From blood samples collected at specified timepoints during treatment - Plasma concentrations of ziftomenib and metabolite(s) - Plasma PK parameters (Cmax, Cmin, Tmax, AUC0-t, AUC0-8, CL/F, Vz/F, and t½) - Based on evaluation of (including but not limited to): a. Duration of CR/CRh, b. TI, c. CR/CRh MRD negativity, d. CRc (CR+ CRh + CRi), e. ORR (CR + CRh + CRi + MLFS), f. EFS, g. OS Phase 1 Part b: - Based on evaluation of (including but not limited to): a. Duration of CR/CRh, b. TI, c. CR/CRh MRD negativity, d. CRc e. EFS, f. OS Phase 2: - Based on evaluation of (including but not limited to): a. Duration of CR/CRh, b. TI, c. CR/CRh MRD negativity, d. CRc e. EFS, f. OS - Safety and tolerability assessed at multiple timepoints during the study ;Timepoint(s) of evaluation of this end point: At any time during the clinical trial. Disease assessment will be done when there is clinical evidence of response. | — |
Countries
Belgium, Canada, France, Germany, Italy, Poland, Spain, United Kingdom, United States
Contacts
Public ContactClinical Operations
Kura Oncology, Inc.
Outcome results
None listed