NCT03745716

APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)

A Phase III Multicenter, Randomized, Open Label Study of APR-246 in Combination With Azacitidine Versus Azacitidine Alone for the Treatment of (Tumor Protein) TP53 Mutant Myelodysplastic Syndromes

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 154 (actual)
• Sponsor: Aprea Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

A Phase III, multicenter, randomized study to compare the rate of CR and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone. Treatment will be administered on an outpatient basis. No investigational or commercial agents or therapies other than those described below may be administered with the intent to treat the patient's disease. Patients will be randomized (1:1) to one of two arms: 1. Experimental arm: APR-246 + azacitidine; or 2. Control arm: Azacitidine

Conditions

• MDS

Interventions

Type	Name	Description
DRUG	APR-246 + azacitidine	Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): Experimental arm: APR-246 + azacitidine; or Control arm: Azacitidine
DRUG	Azacitidine	Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): Experimental arm: APR-246 + azacitidine; or Control arm: Azacitidine

Timeline

• Start date: 2019-01-11
• Primary completion: 2020-11-27
• Completion: 2022-01-14
• First posted: 2018-11-19
• Last updated: 2025-03-18
• Results posted: 2022-07-12

Locations

27 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study