Expanded Access to Ziftomenib

• Conditions: Acute Lymphoblastic Leukemia, With Appropriate Mutations; Acute Myeloid Leukemia, With NPM1 Mutations

• Registration Number: NCT05738538
• Lead Sponsor: Kura Oncology, Inc.

Brief Summary

Requests for single patient expanded access to ziftomenib monotherapy may be considered for eligible adult patients with Acute Lymphoblastic Leukemia (ALL) with KMT2A rearrangements, or Acute Myeloid Leukemia (AML), with NPM1 mutations or KMT2A rearrangements.

To request access, use Responsible Party contact information provided in this record.

Expanded access for ziftomenib is only available in the United States.

Detailed Description

• Subsection Condition:

* NPM1 mutation in AML patients

* KMT2A rearrangement in AML patients

* KMT2A rearrangement in ALL patients

Study Timeline

• Study First Submitted: 2023-02-10
• Study First Submitted QC: 2023-02-10
• Study First Posted: 2023-02-22
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-29
• Last Update Posted: 2025-06-04

Recruitment & Eligibility

• Status: AVAILABLE
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

• Diagnosed with Acute Myeloid Leukemia (AML) or Acute Lymphoblastic Leukemia (ALL).
• Adult, ages 18+.
• Has exhausted appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition. For newly diagnosed AML patients with either NPM1-mutant, or KMT2A-rearranged AML, ziftomenib therapy will be considered for patients not eligible for ongoing clinical trials. In the case of patients with KMT2A-rearranged AML, Expanded Access to ziftomenib will only be provided for use in combination with FDA-approved therapies currently under clinical investigation at Kura Oncology (e.g., venetoclax + azacitidine, cytarabine + daunorubicin).
• Is ineligible for participation in any ongoing clinical study of the investigational drug, which includes lack of access due to geographic limitations.
• Meets any other pertinent medical criteria for access to the investigational drug, as established by Kura Oncology.

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified