PARPAML: A Phase I Protocol for Relapsed Pediatric AML to Determine the Safety and Efficacy of the PARP Inhibitor Talazoparib in Combination with Chemotherapy

Pediatric Blood Cancer
Jennifer Kamens, MD
Stanford University


Currently, the survival of children diagnosed with AML sits at less than 70%. Despite 80 to 90% of patients achieving first complete remission, 30 to 40% will relapse and outcomes remain dismal as the overall survival rate is less than 40%. Outcomes are even worse for patients with high-risk features (subtype, genetic alterations, etc). Preclinical data suggests that AML harboring high risk cytogenetic features are sensitive to PARP inhibitors. Talazoparib, a PARP inhibitor, prevents cancer cells from repairing themselves such as after exposure to chemotherapy, causing them to die. Normally PARP, a cellular protein, would repair any DNA damage a cell has endured. Thus, this phase I study will assess the safety and tolerability of talazoparib in combination with conventional chemotherapy in pediatric patients with relapsed or refractory (resistant) AML and ALAL.

Trial Registration: Identifier: NCT05101551