14 and older, any sex, with Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Dose Limiting Toxicity (Phase Ib)Primary· Up to 35 days
Dose escalation for phase 1b will be in the usual 3+3 fashion
Group
Value
95% CI
Cohort I (Higher and Lower Dose Pinometostat)
0
Cohort II (Higher Dose Pinometostat)
0
Complete Response (CR) or Complete Response With Incomplete Count Recovery (CRi) RatePrimary· Up to 3 years
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Group
Value
95% CI
Cohort I (Higher and Lower Dose Pinometostat)
3
Cohort II (Higher Dose Pinometostat)
2
Number of Participants With Incidence of Adverse EventsSecondary· For up to 3 years
Will be graded and reported according to Common Terminology Criteria for Adverse Events version 5. The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns.
Any grade
Group
Value
95% CI
Cohort I (Higher and Lower Dose Pinometostat)
3
Cohort II (Higher Dose Pinometostat)
3
Grade 4
Group
Value
95% CI
Cohort I (Higher and Lower Dose Pinometostat)
3
Cohort II (Higher Dose Pinometostat)
2
Progression Free SurvivalSecondary· From the start of study treatment until progression or death, whichever occurs earliest, assessed up to 3 years
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
Group
Value
95% CI
Cohort I (Higher and Lowers Dose Pinometostat)
100
95 – 133
Cohort II (Higher Dose Pinometostat)
400
400 – 400
Overall SurvivalSecondary· Up to 3 years
Estimated using the Kaplan-Meier method.
Group
Value
95% CI
Cohort I (Higher and Lowers Dose Pinometostat)
326
181 – 450
Cohort II (Higher Dose Pinometostat)
298
168 – 428
Adverse events — posted to ClinicalTrials.gov
Time frame: The Adverse event information was collected for study patients from baseline through study completion utilizing the CTCAE version 5.0 to determine the severity of the reaction for adverse event reporting from baseline to after study completion, up to 2 years, All-Cause Mortality monitored/assessed up to 3 years..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This phase Ib/II trial studies the side effects and best dose of pinometostat and how well it works with standard chemotherapy in treating patients with newly diagnosed acute myeloid leukemia and a type of genetic mutation called MLL gene rearrangement. Pinometostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in standard chemotherapy, such as daunorubicin hydrochloride and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving pinometostat with standard chemotherapy may work better at treating acute myeloid leukemia.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07053020 — A Phase 1b/2 Open-label, Dose-ranging Safety and Efficacy Study of Oral Cladribine in Patients With Acute Myeloid Leukem
· Phase 1, PHASE2
· not yet recruiting
NCT07498465 — A Study to Find the Highest Dose of SNDX-5613 (Revumenib) as a Treatment Option After Hematopoietic Stem Cell Transplant
· Phase 1
· withdrawn
NCT07444710 — Testing the Addition of an Anti-Cancer Drug, Glofitamab, to the Usual Chemotherapy Treatment (Alternating R-CHOP/R-DHAP)
· Phase 1
· not yet recruiting
NCT07428486 — A Phase 1 Study Of FLAG Chemotherapy In Combination With Lisaftoclax And Pelcitoclax In Patients With Relapsed/Refractor
· Phase 1
· not yet recruiting
NCT07022678 — Xylitol Dental Wipes for the Reduction of Bloodstream Infection Risk in Children With Acute Myeloid Leukemia
· Phase 3
· not yet recruiting
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 18 June 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03724084.