Table 1.

List of ongoing phase 1/2 and 3 trials in NPM1mut-AML (£: trials exploring pre-emptive treatment on molecular relapse) (date accessed-January 7, 2023) (ref. Clinicaltrials.gov).

Trial	Intervention	Current Status	Outcomes
1. NCT00893399 (Phase 3)	Chemotherapy with ATRA with or without gemtuzumab- ozogamicin	Completed, final data collection, September 2021	Primary—OS Secondary—EFS, safety, CR post-induction, QoL
2. NCT04689815 £ (Phase 2)	Phase 2, single arm study with oral arsenic trioxide, ascorbic acid and azacytidine for MRD positive for NPM1 AML post consolidation, transplant ineligible	Recruiting	Primary—rate of NPM1 MRD negativity Secondary—DOR, leukemia- free survival (LFS), safety
3. NCT04867928 £ (Phase 2)	Phase 2, single arm, open label- venetoclax and azacitidine on molecular relapse as bridge to allo-HSCT	Recruiting	Primary—efficacy of venetoclax and azacitidine as bridge to transplant
4. NCT05020665 (Phase 3)	Phase 3, randomized, double blind, placebo control oral entospletinib vs. placebo with combination with intensive induction and consolidation chemotherapy in newly diagnosed NMP1 mut-AML	Active, not recruiting	Primary—MRD negative CR Secondary—EFS, RFS, CR, adverse events
5. NCT01237808 (Phase 3)	Low-Dose cytarabine (20 mg/day, SQ BID day 1–7) and etoposide with or without ATRA older patients not eligible for intensive chemotherapy in NPM1mut-AML	Completed	Primary—overall survival Secondary—rates of CR, cumulative incidence of relapse, EFS, deaths, adverse events, QoL
6. NCT03769532 £ (Phase 2)	Single arm, pembrolizumab with azacitidine in morphological remission but MRD positive	Recruiting	Primary—EFS Secondary—OS, EFS, safety
7. NCT03031249 (Phase 1,2)	Post induction consolidation with combination of cytarabine, ATO and ATRA vs. cytarabine alone	Unknown	Primary—RFS (up to 5 years from randomization) Secondary—OS, cumulative incidence of relapse
8. NCT04988555 (Phase 1,2)	Open-label, single arm safety and efficacy of DSP-5336 in relapsed/ refractory AML/ALL	Recruiting	Primary—safety Secondary—efficacy
9. NCT04065399 (Phase 1,2)	Open label, single arm study of SNDX-5613 in relapsed/refractory leukemia (AUGMENT-001)	Recruiting	Primary—safety Secondary—CR, median RFS, OS, DOR

(OS—overall survival, DOR—duration of response, EFS—event free survival, MRD—measurable residual disease, CR—complete response, QoL—quality of life, ATRA—all-trans retinoic acid, ATO—arsenic trioxide, RFS—relapse free survival).