Trial · NCT03072043

Phase 1b/2 Safety and Efficacy of APR-246 w/Azacitidine for tx of TP53 Mutant Myeloid Neoplasms

NCT03072043, Phase 1, Phase 2, Completed, H. Lee Moffitt Cancer Center and Research Institute; results first posted 2021-02-25

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Phase 1b/2 Safety and Efficacy of APR-246 w/Azacitidine for tx of TP53 Mutant Myeloid Neoplasms. Trials, record NCT03072043. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03072043

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Phase 1b/2 Safety and Efficacy of APR-246 w/Azacitidine for tx of TP53 Mutant Myeloid Neoplasms
NCT number: NCT03072043
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 55 participants, actual
Lead sponsor: H. Lee Moffitt Cancer Center and Research Institute
Conditions: Myelodysplastic Syndrome, Acute Myeloid Leukemia, Myeloproliferative Neoplasm, Chronic Myelomonocytic Leukemia
Interventions: APR-246, Azacitidine
First posted: 2017-03-07
Last updated: 2022-01-24
Results first posted: 2021-02-25

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Phase 1b: Maximum Tolerated Dose (MTD)	Up to 12 months	Maximum Tolerated Dose, defined as the dose level below which dose limiting toxicity (DLT) is manifested in ≥33% of the patients or at dose level 3 if DLT is manifested in \<33% of the patients.
Primary	Phase 2: Complete Response (CR) Rate	Up to 12 months	Complete Response Rate as defined by the 2006 International Working Group (IWG) criteria.
Secondary	Phase 2: Duration of Response	Up to 24 months	Duration of response defined as the time between achieving response and progression of disease.
Secondary	Overall Survival (OS)	Up to 24 months	OS:The length of time from the start of treatment until death by any cause.
Secondary	Phase 2: Overall Response Rate	Up to 24 months	Proportion of participants achieving hematological improvement (HI), partial response (PR), complete response (CR), and/or marrow CR (mCR) by the IWG 2006 criteria.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Phase 1b: Maximum Tolerated Dose (MTD)	Up to 12 months		mg/day	NUMBER
PRIMARY	Phase 2: Complete Response (CR) Rate	Up to 12 months	45 participants were evaluable for response	percentage of patients	NUMBER
SECONDARY	Phase 2: Duration of Response	Up to 24 months	45 participants were evaluable for response.	months	MEDIAN
SECONDARY	Overall Survival (OS)	Up to 24 months	All participants who received at least one dose of study drug	months	MEDIAN
SECONDARY	Phase 2: Overall Response Rate	Up to 24 months	45 participants were evaluable for response	percent of participants	NUMBER

Source ClinicalTrials.gov

Results by group7

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Phase 1b: Maximum Tolerated Dose (MTD)	Phase 1b Dose Escalation	4500		mg/day
Phase 2: Complete Response (CR) Rate	Phase 2: Participants in Dose Level 3/MTD	53	95% Confidence Interval 38 to 68	percentage of patients
Phase 2: Duration of Response	Evaluable Participants	8	Full Range 6.5 to 11.2	months
Overall Survival (OS)	Phase 1B Dose Level 1	14.6	Full Range 2.4 to 16.0	months
Overall Survival (OS)	Phase 1B Dose Level 2	11.6	Full Range 6.3 to 13.7	months
Overall Survival (OS)	Phase 1B: Dose Level 3 / Phase 2 MTD	10.4	Full Range 0.7 to 17.9	months
Phase 2: Overall Response Rate	All Participants	87	95% Confidence Interval 73 to 95	percent of participants

Source ClinicalTrials.gov

Participant flow9

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Phase 1b Dose Level 1	3
Overall Study	STARTED	Phase 1B Dose Level 2	3
Overall Study	STARTED	Phase 1B Dose Level 3 / Phase 2 MTD	49
Overall Study	COMPLETED	Phase 1b Dose Level 1	3
Overall Study	COMPLETED	Phase 1B Dose Level 2	3
Overall Study	COMPLETED	Phase 1B Dose Level 3 / Phase 2 MTD	40
Overall Study	NOT COMPLETED	Phase 1b Dose Level 1	0
Overall Study	NOT COMPLETED	Phase 1B Dose Level 2	0
Overall Study	NOT COMPLETED	Phase 1B Dose Level 3 / Phase 2 MTD	9

Source ClinicalTrials.gov

Adverse events696

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Nausea	Gastrointestinal disorders	Phase 1b Dose Level 1	1	3	2
other	Nausea	Gastrointestinal disorders	Phase 1B Dose Level 2	2	3	8
other	Nausea	Gastrointestinal disorders	Phase 1B Dose Level 3/Phase 2 MTD	32	49	57
other	Blurry vision	Eye disorders	Phase 1b Dose Level 1	0	3	0
other	Blurry vision	Eye disorders	Phase 1B Dose Level 2	1	3	1
other	Blurry vision	Eye disorders	Phase 1B Dose Level 3/Phase 2 MTD	0	49	0
other	Fever	General disorders	Phase 1b Dose Level 1	0	3	0
other	Fever	General disorders	Phase 1B Dose Level 2	0	3	0
other	Fever	General disorders	Phase 1B Dose Level 3/Phase 2 MTD	12	49	16
other	Flu like symptoms	General disorders	Phase 1b Dose Level 1	0	3	0

1 to 10 of 696Source ClinicalTrials.gov

See all 696 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Phase 1b Dose Level 1	1	3
Total, all-cause mortality	Phase 1B Dose Level 2	1	3
Total, all-cause mortality	Phase 1B Dose Level 3/Phase 2 MTD	36	49
Total, other adverse events	Phase 1b Dose Level 1	3	3
Total, other adverse events	Phase 1B Dose Level 2	3	3
Total, other adverse events	Phase 1B Dose Level 3/Phase 2 MTD	47	49
Total, serious adverse events	Phase 1b Dose Level 1	3	3
Total, serious adverse events	Phase 1B Dose Level 2	1	3
Total, serious adverse events	Phase 1B Dose Level 3/Phase 2 MTD	30	49

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33449813	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03072043

GET https://conductscience.org/api/trials/v1/NCT03072043/events?page=2

API reference For AI assistants