Clinical trial · Interventional
Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia
Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed/Refractory Acute Myeloid Leukemia
NCT06001788CI-TRIAL-00122931recruitingPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The safety, tolerability, and antileukemic response of ziftomenib in combination with standard of care treatments for patients with relapsed/refractory acute myeloid leukemia will be examined with the following agents: FLAG-IDA, low-dose cytarabine, and gilteritinib.
Conditions
Conditions (12)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Leukemia | Acute Leukemia | ONTOLOGY_EXACT | 0.90 |
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| AML | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| AML With Mutated NPM1 | Acute Myeloid Leukemia with NPM1 Mutation | ALIAS | 0.90 |
| Hematologic Malignancy | Hematopoietic and Lymphoid Cell Neoplasm | ALIAS | 0.90 |
| KMT2Ar | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Leukemia, Myeloid | Myeloid Leukemia | ALIAS | 0.90 |
| Leukemia, Myeloid, Acute | Leukemia | ONTOLOGY_EXACT | 0.85 |
| MLL Rearrangement |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Cytarabine | Drug | Cytarabine | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Gilteritinib | Drug | Gilteritinib | ALIAS |
| Granulocyte colony-stimulating factor | Biological | — | UNRESOLVED |
| Idarubicin | Drug | Idarubicin | ALIAS |
| Ziftomenib | Drug | Ziftomenib | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Phase 1a
- description
- Oral ziftomenib; sequential cohorts of escalating dose levels of ziftomenib to identify the safety and tolerability of the combination regimens. Participants will be enrolled in 1 of 5 dose escalation cohorts: A-1: Participants with a NPM1 mutation: ziftomenib plus FLAG-IDA A-2: Participants with a NPM1 mutation: ziftomenib plus low-dose cytarabine (LDAC) A-3: Participants with a NPM1 mutation: ziftomenib plus gilteritinib B-1: Participants with a KMT2A rearrangement: ziftomenib plus FLAG-IDA B-2: Participants with a KMT2A rearrangement: ziftomenib plus low-dose cytarabine (LDAC)
- interventionNames
- Drug: Ziftomenib
- Drug: Fludarabine
- Drug: Idarubicin
- Drug: Cytarabine
- Drug: Gilteritinib
- Biological: Granulocyte colony-stimulating factor
- type
- EXPERIMENTAL
- label
- Phase 1b
- description
- Oral ziftomenib; Following the determination of the maximum tolerated dose in Phase 1a, participants will be enrolled in 1 of 5 dose validation/expansion cohorts: A-1: Participants with a NPM1 mutation: ziftomenib plus FLAG-IDA A-2: Participants with a NPM1 mutation: ziftomenib plus low-dose cytarabine (LDAC) A-3: Participants with a NPM1 mutation: ziftomenib plus gilteritinib B-1: Participants with a KMT2A rearrangement: ziftomenib plus FLAG-IDA B-2: Participants with a KMT2A rearrangement: ziftomenib plus low-dose cytarabine (LDAC)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Key Inclusion Criteria: * Has been diagnosed with relapsed/refractory AML. * Has a documented NPM1 mutation or KMT2A rearrangement. * Has a documented FLT3 mutation (cA-3 only). * Has an Eastern Cooperative Oncology Group (ECOG) Performance status ≤ 2. * Has adequate hepatic and renal function as defined per protocol. * Has an ejection fraction above a protocol defined limit. * Participant, or legally authorized representative, must be able to understand and provide written informed consent prior to the first screening procedure. * Has agreed to use contraception as defined per protocol. Key Exclusion Criteria: * Has a diagnosis of acute promyelocytic leukemia or blast chronic myeloid leukemia. * Has clinically active central nervous system leukemia. * Has an active and uncontrolled infection. * Has a mean corrected QT interval (QTcF) \> 480ms. * Has uncontrolled intercurrent illness, including, but not limited to protocol defined cardiac disease. * Has received radiation, chemotherapy, immunotherapy, or any other anticancer therapy including investigational therapy \<14 days or within 5 drug half-lives prior to the first dose of study intervention. * Has had major surgery within 4 weeks prior to the first dose of study intervention. * Has received a hematopoietic stem cell transplant (HSCT) and has not previously had adequate recovery per protocol defined criteria. * Has active graft-versus-host disease (GvHD) and or on immunosuppressive drugs for the treatment of GvHD * Participant is pregnant or lactating.
References
Publications (0)
Data not yet available
No reference posted for this study.