Phase 1 Study to Evaluate Safety and Efficacy of APR-548 in Combination with Azacitidine for the Treatment of TP53-Mutant Myelodysplastic Syndromes
Phase 1 study evaluating the safety and efficacy of APR-548 in combination with Azacitidine for the treatment of TP53-Mutant Myelodysplastic Syndromes.
1. To investigate the PK, safety and tolerability of APR-548 as monotherapy and in combination with azacitidine and determine the RP2D of APR-548 in subjects with TP53-mutant MDS.
1. To determine the complete remission (CR) rate, defined as the proportion of patients who achieve CR with APR-548 and azacitidine using modified IWG criteria (2006; Appendix V)
1. To assess preliminary clinical activity of APR-548 administered in combination with azacitidine -
a. Duration of CR
b. Overall response rate (ORR)
c. Duration of ORR
d. Rate of and time to development of acute myeloid leukemia (AML), as defined World Health Organization (WHO) criteria
e. Overall survival (OS)
f. Relapse-free survival (RFS)
g. Rate of transition to hematopoietic stem cell transplant (HSCT)
h. Rate of red blood cell and/or platelet transfusion independence for 8 weeks
2. To characterize PK of APR-548 after single and multiple dosing when given p.o.;
3. To evaluate the PK drug-drug interaction between APR-548 and azacitidine.
Cardiac abnormalities, which includes, but not limited to: Myocardial infarction within six months prior to enrollment, New York Heart Association Class III or IV heart failure or known LVEF > Concomitant malignancies or previous malignancies with less than a 1 year disease-free interval at the time of signing informed consent
Use of cytotoxic chemotherapeutic agents, or experimental agents for the treatment of MDS within 14 days or 5 half-lives of the product (whichever is shorter) of the first day of study drug treatment
Prior exposure to eprenetapopt (APR-246)
A female subject who is pregnant or breast-feeding
Known history of human immunodeficiency virus (HIV), active hepatitis B or active hepatitis C infection
Malabsorption syndrome or other condition likely to affect gastrointestinal absorption of APR-548
Known history or current evidence of ocular disease in either eye
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