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APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)
Phase 3 trial testing APR-246 + azacitidine in MDS in 154 participants. Completed in 14 January 2022.
| Lead sponsor | Aprea Therapeutics |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 154 |
| Start date | 11 January 2019 |
| Primary completion | 27 November 2020 |
| Estimated completion | 14 January 2022 |
| Sites | 27 locations across France, United States |
Aprea Therapeutics — full company profile →
18 and older, any sex, with MDS. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
To compare the complete response rate, defined as the proportion of patients who achieve complete remission (CR) with APR 246 + azacitidine treatment vs. azacitidine only.
| Group | Value | 95% CI |
|---|---|---|
| Experimental Arm: APR-246 + Azacitidine | 27 | |
| Control Arm: Azacitidine | 17 |
Time frame: 12 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Experimental Arm: APR-246 … | Control Arm: Azacitidine |
|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Pneumonia | Infections and infestations | — | — |
| Pyrexia | General disorders | — | — |
| Sepsis | Infections and infestations | — | — |
| Muscular weakness | Musculoskeletal and connective tissue disorders | — | — |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | — | — |
| Septic shock | Infections and infestations | — | — |
| Confusional state | Psychiatric disorders | — | — |
| Cellulitis | Infections and infestations | — | — |
| Coronavirus infection | Infections and infestations | — | — |
| Urinary tract infection | Infections and infestations | — | — |
| Anemia | Blood and lymphatic system disorders | — | — |
| Thrombocytopenia | Blood and lymphatic system disorders | — | — |
| Asthenia | General disorders | — | — |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | — | — |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | — | — |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — | — |
| Encephalopathy | Nervous system disorders | — | — |
| Pericarditis | Cardiac disorders | — | — |
| Fall | Injury, poisoning and procedural complications | — | — |
| Acute febrile neutrophilic dermatosis | Skin and subcutaneous tissue disorders | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — |
| Skin infection | Infections and infestations | — | — |
| Abdominal infection | Infections and infestations | — | — |
| Acute sinusitis | Infections and infestations | — | — |
| Reaction | System | Experimental Arm: APR-246 … | Control Arm: Azacitidine |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Neutrophil count decreased | Investigations | — | — |
| Anemia | Blood and lymphatic system disorders | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| White blood cell count decreased | Investigations | — | — |
| Fatigue | General disorders | — | — |
| Platelet count decreased | Investigations | — | — |
| Headache | Nervous system disorders | — | — |
| Dizziness | Nervous system disorders | — | — |
| Diarrhea | Gastrointestinal disorders | — | — |
| Pyrexia | General disorders | — | — |
| Oedema peripheral | General disorders | — | — |
| Thrombocytopenia | Blood and lymphatic system disorders | — | — |
| Hypokalemia | Metabolism and nutrition disorders | — | — |
| Injection site reaction | General disorders | — | — |
| Neutropenia | Blood and lymphatic system disorders | — | — |
| Decreased appetite | Metabolism and nutrition disorders | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Hypoalbuminaemia | Metabolism and nutrition disorders | — | — |
| Lymphocyte count decreased | Investigations | — | — |
| Paresthesia | Nervous system disorders | — | — |
| Tremor | Nervous system disorders | — | — |
| Hypophosphatemia | Metabolism and nutrition disorders | — | — |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | — | — |
| Confusional state | Psychiatric disorders | — | — |
| Stomatitis | Gastrointestinal disorders | — | — |
| Chills | General disorders | — | — |
| Hyponatremia | Metabolism and nutrition disorders | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — |
| Pruritus | Skin and subcutaneous tissue disorders | — | — |
| Muscular weakness | Musculoskeletal and connective tissue disorders | — | — |
| Anxiety | Psychiatric disorders | — | — |
| Hypotension | Vascular disorders | — | — |
| Hypertension | Vascular disorders | — | — |
| Pneumonia | Infections and infestations | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — |
| Insomnia | Psychiatric disorders | — | — |
| Contusion | Injury, poisoning and procedural complications | — | — |
Most-reported serious reactions: Febrile neutropenia, Pneumonia, Pyrexia, Sepsis, Muscular weakness, Respiratory failure, Septic shock, Confusional state.
Data from ClinicalTrials.gov NCT03745716 adverse events section.
A Phase III, multicenter, randomized study to compare the rate of complete response (CR) and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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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/NCT03745716.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing