Trial Outcomes & Findings for APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (NCT NCT04638309)
NCT ID: NCT04638309
Last Updated: 2025-03-10
Results Overview
Occurrence of frequency of treatment emergent adverse events by reviewing safety data including AEs, vital signs, laboratory data, ECG, ophthalmologic assessment findings, and other physical exam findings.
Recruitment status
TERMINATED
Study phase
PHASE1
Target enrollment
4 participants
Primary outcome timeframe
Through study completion, approximately 28 days
Results posted on
2025-03-10
Participant Flow
Study was terminated prior to enrollment into cohort 2 and 3.
Participant milestones
| Measure |
Cohort 1
Dose level 1
APR-548 50 mg/d + Azacitidine 75 mg/d: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
Cohort 2
Dose level 2
APR-548 150 mg/d + Azacitidine 75 mg/d: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
Cohort 3
Dose level 3
APR-548 300 mg/d + Azacitidine 75 mg/d: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
0
|
0
|
|
Overall Study
COMPLETED
|
3
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS)
Baseline characteristics by cohort
| Measure |
Cohort 1
n=4 Participants
Dose level 1
APR-548 + Azacitidine: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
Cohort 2
Dose level 2
APR-548 + Azacitidine: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
Cohort 3
Dose level 3
APR-548 + Azacitidine: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
64 years
n=99 Participants
|
—
|
—
|
64 years
n=7 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
—
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
—
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
—
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
—
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=99 Participants
|
—
|
—
|
4 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
—
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
—
|
—
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=99 Participants
|
—
|
—
|
4 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Through study completion, approximately 28 daysPopulation: Study was terminated.
Occurrence of frequency of treatment emergent adverse events by reviewing safety data including AEs, vital signs, laboratory data, ECG, ophthalmologic assessment findings, and other physical exam findings.
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Dose level 1
APR-548 + Azacitidine: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
|---|---|
|
To Investigate the "Number of Participants With Treatment Emergent Adverse Events From Treatment With APR-548 as Monotherapy and in Combination With Azacitidine
|
3 Participants
|
Adverse Events
Cohort 1
Serious events: 2 serious events
Other events: 2 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Cohort 1
n=4 participants at risk
Dose level 1
APR-548 + Azacitidine: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
|---|---|
|
Injury, poisoning and procedural complications
Fall
|
25.0%
1/4 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
pneumonia
|
25.0%
1/4 • 28 days
|
|
Infections and infestations
Sinusitis
|
25.0%
1/4 • 28 days
|
|
Vascular disorders
Stroke
|
25.0%
1/4 • Number of events 2 • 28 days
|
|
Cardiac disorders
Myocardial Infarcation
|
25.0%
1/4 • 28 days
|
|
Cardiac disorders
Cardiogenic Shock
|
25.0%
1/4 • Number of events 2 • 28 days
|
Other adverse events
| Measure |
Cohort 1
n=4 participants at risk
Dose level 1
APR-548 + Azacitidine: APR-548 monotherapy period followed by APR-548 in combination with Azacitidine
|
|---|---|
|
Investigations
Alkaline phosphatase increase
|
25.0%
1/4 • 28 days
|
|
General disorders
Drug hypersensitivity
|
25.0%
1/4 • 28 days
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
25.0%
1/4 • 28 days
|
|
Infections and infestations
Candida Infection
|
25.0%
1/4 • 28 days
|
|
Psychiatric disorders
Confusional state
|
25.0%
1/4 • 28 days
|
|
Injury, poisoning and procedural complications
Contusion
|
50.0%
2/4 • 28 days
|
|
General disorders
Dehydration
|
25.0%
1/4 • 28 days
|
|
Psychiatric disorders
Depression
|
25.0%
1/4 • 28 days
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
2/4 • Number of events 4 • 28 days
|
|
General disorders
Dizziness
|
25.0%
1/4 • 28 days
|
|
Psychiatric disorders
Dysphemia
|
25.0%
1/4 • 28 days
|
|
Nervous system disorders
Extrapyramidal disorder
|
25.0%
1/4 • 28 days
|
|
General disorders
Fatigue
|
50.0%
2/4 • 28 days
|
|
Gastrointestinal disorders
Flatulence
|
25.0%
1/4 • 28 days
|
|
Skin and subcutaneous tissue disorders
Flushing
|
25.0%
1/4 • 28 days
|
|
General disorders
hemorrhoids hemmorage
|
25.0%
1/4 • 28 days
|
|
General disorders
Headache
|
25.0%
1/4 • Number of events 2 • 28 days
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
25.0%
1/4 • 28 days
|
|
Psychiatric disorders
Hypersomnia
|
25.0%
1/4 • 28 days
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
25.0%
1/4 • 28 days
|
|
Metabolism and nutrition disorders
Hypokalemia
|
25.0%
1/4 • 28 days
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
50.0%
2/4 • 28 days
|
|
Metabolism and nutrition disorders
Hyponatremia
|
25.0%
1/4 • 28 days
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
25.0%
1/4 • 28 days
|
|
Skin and subcutaneous tissue disorders
Infusion site pruritis
|
25.0%
1/4 • 28 days
|
|
Skin and subcutaneous tissue disorders
Ingrown Nail
|
25.0%
1/4 • 28 days
|
|
Metabolism and nutrition disorders
Lactic Acidosis
|
25.0%
1/4 • 28 days
|
|
Gastrointestinal disorders
Nausea
|
50.0%
2/4 • 28 days
|
|
Vascular disorders
Odema Peripheral
|
25.0%
1/4 • Number of events 3 • 28 days
|
|
General disorders
Pain in extremity
|
25.0%
1/4 • 28 days
|
|
Eye disorders
Periorbital Edema
|
25.0%
1/4 • 28 days
|
|
Vascular disorders
Phlebitis
|
25.0%
1/4 • 28 days
|
|
Nervous system disorders
Tremor
|
25.0%
1/4 • 28 days
|
|
Renal and urinary disorders
urinary hestiation
|
25.0%
1/4 • 28 days
|
|
Eye disorders
Vison Blurred
|
25.0%
1/4 • 28 days
|
|
Eye disorders
Vision Impairment
|
25.0%
1/4 • 28 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60