Phase I First-in-Human Study of Venetoclax in Patients With Relapsed or Refractory Non-Hodgkin Lymphoma
This is a phase I adult study of a new inhibitor of BCL-2, an anti-apoptotic protein, called venetoclax. It is of relevance to paediatrics because this target is also thought to be important in paediatric leukaemia/lymphoma and potentially neuroblastoma.
Phase I dose escalation study in patients with non-Hodgkin lymphoma (part of a larger study that also included patients with CLL). Standard phase I inclusion criteria and toxicity grading according to CTCAE.
Maximum tolerated dose was not defined (no dose level exceeded 30% DLT rate). Manageable side effects. Impressive evidence of clinical benefit in context of phase I.
Interesting that despite this being a phase I study with max 6 dose levels, 106 patients were enrolled. Reflects a growing trend for significant expansion within the context of a phase I to demonstrate efficacy once dose has been determined.