A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
An open-label, global, multi-center study to evaluate the safety and
pharmacokinetics of venetoclax monotherapy, to determine the dose
limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to
assess the preliminary efficacy of venetoclax in pediatric and young
adult participants with relapsed or refractory malignancies.
Venetoclax ist a small molecule binding to the protein BCL-2. By attaching to Bcl-2 and blocking its actions, venetoclax causes the death of cancer cells and thereby slows down progression of the disease. Venetoclax is administered orally once daily with or without chemotherapy.