Trial Outcomes & Findings for APR-246 in Combination With Azacitidine for TP53 Mutated AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) Following Allogeneic Stem Cell Transplant (NCT NCT03931291)
NCT ID: NCT03931291
Last Updated: 2025-03-17
Results Overview
Relapse-free survival (RFS) at 12 months or longer if data permits. RFS was defined as the time from HCT to relapse after HCT or death, whichever occurred first.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
33 participants
Primary outcome timeframe
Through study completion, an average of 1 year
Results posted on
2025-03-17
Participant Flow
Participant milestones
| Measure |
Experimental Arm: APR-246 + Azacitidine
APR-246 and azacitidine maintenance therapy will continue for a maximum of 12 cycles
APR-246: APR-246 will be administered on Days 1-4, with azacitidine on Days 1-5, of every 28 day cycle. Patients may receive a maximum of 12 cycles.
|
|---|---|
|
Overall Study
STARTED
|
33
|
|
Overall Study
COMPLETED
|
33
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
APR-246 in Combination With Azacitidine for TP53 Mutated AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) Following Allogeneic Stem Cell Transplant
Baseline characteristics by cohort
| Measure |
Experimental Arm: APR-246 + Azacitidine
n=33 Participants
APR-246 and azacitidine maintenance therapy will continue for a maximum of 12 cycles
APR-246: APR-246 will be administered on Days 1-4, with azacitidine on Days 1-5, of every 28 day cycle. Patients may receive a maximum of 12 cycles.
|
|---|---|
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Age, Customized
|
65 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
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33 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Through study completion, an average of 1 yearPopulation: Allogenic Transplant Patients with TP53 AML or MDS
Relapse-free survival (RFS) at 12 months or longer if data permits. RFS was defined as the time from HCT to relapse after HCT or death, whichever occurred first.
Outcome measures
| Measure |
Experimental Arm: APR-246 + Azacitidine
n=33 Participants
APR-246 and azacitidine maintenance therapy will continue for a maximum of 12 cycles
APR-246: APR-246 will be administered on Days 1-4, with azacitidine on Days 1-5, of every 28 day cycle. Patients may receive a maximum of 12 cycles.
|
|---|---|
|
To Assess Relapse-free Survival (RFS) in Patients With TP53 Mutated AML or MDS After Undergoing Allogeneic Hematopoietic Stem Cell Transplant (HSCT).
|
12.5 months
Interval 9.6 to 14.5
|
Adverse Events
Experimental Arm: APR-246 + Azacitidine
Serious events: 13 serious events
Other events: 33 other events
Deaths: 13 deaths
Serious adverse events
| Measure |
Experimental Arm: APR-246 + Azacitidine
n=33 participants at risk
APR-246 and azacitidine maintenance therapy will continue for a maximum of 12 cycles
APR-246: APR-246 will be administered on Days 1-4, with azacitidine on Days 1-5, of every 28 day cycle. Patients may receive a maximum of 12 cycles.
|
|---|---|
|
General disorders
Pyrexia
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
Disease progression
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
General Oedema
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
Malise
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
Pain
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Injury, poisoning and procedural complications
Hip Fracture
|
9.1%
3/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Injury, poisoning and procedural complications
Seroma
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Injury, poisoning and procedural complications
Spinal Fracture
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
6.1%
2/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Infections and infestations
Device related infection
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Infections and infestations
Urinary tract infection
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Infections and infestations
Viremia
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Blood Bilirubin Increased
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Blood Creatinine Increased
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Dysponea
|
6.1%
2/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Cardiac disorders
Pericardial effusion
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain Lower
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Psychiatric disorders
Confusional state
|
3.0%
1/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
Other adverse events
| Measure |
Experimental Arm: APR-246 + Azacitidine
n=33 participants at risk
APR-246 and azacitidine maintenance therapy will continue for a maximum of 12 cycles
APR-246: APR-246 will be administered on Days 1-4, with azacitidine on Days 1-5, of every 28 day cycle. Patients may receive a maximum of 12 cycles.
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
60.6%
20/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Platelet Count Decreased
|
48.5%
16/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Vomitting
|
45.5%
15/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Blood and lymphatic system disorders
Anemia
|
39.4%
13/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Nervous system disorders
Dizziness
|
39.4%
13/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
White blood cell count decreased
|
39.4%
13/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
Fatigue
|
36.4%
12/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
11/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Nervous system disorders
Tremor
|
33.3%
11/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Neutrophil Count decreased
|
27.3%
9/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
24.2%
8/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Skin and subcutaneous tissue disorders
pruritus
|
24.2%
8/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
Pyrexia
|
24.2%
8/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Abdominal Pain
|
21.2%
7/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Consitpation
|
21.2%
7/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Decreased Appetite
|
21.2%
7/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Nervous system disorders
Headache
|
21.2%
7/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
21.2%
7/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Dry Mouth
|
18.2%
6/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Nervous system disorders
Dyskinesia
|
18.2%
6/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
18.2%
6/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
18.2%
6/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Vascular disorders
Hypertension
|
18.2%
6/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
18.2%
6/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Blood cholesterol Increased
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Blood Creatinine Increased
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Eye disorders
Dry Eye
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Renal and urinary disorders
Dysuria
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Psychiatric disorders
Insomnia
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
General disorders
Edema Peripheral
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.2%
5/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Nervous system disorders
Ataxia
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Blood Alkaline Phosphate Increased
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Blood Lactate Dehydrogenase Increased
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Investigations
Serum ferritin increased
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
12.1%
4/33 • 13 months
The AE reporting period starts from the day of signing the post-transplant informed consent form. AEs will be collected for 30 days after the last dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60