Accelerating drug development in pediatric cancer: a novel Phase I study design of venetoclax in relapsed/refractory malignancies

Future Oncol. 2018 Sep;14(21):2115-2129. doi: 10.2217/fon-2018-0121. Epub 2018 Mar 29.


Venetoclax is a highly selective, potent BCL-2 inhibitor that is approved for some patients previously treated for chronic lymphocytic leukemia, and has shown promising activity in adult studies across several hematologic malignancies. Preclinical studies have demonstrated venetoclax activity in pediatric patient-derived xenograft models and cell lines; however, clinical studies in pediatric patients have yet to be conducted. The prognosis is poor for children with most relapsed/refractory malignancies, and limited treatment options result in unmet clinical need. Herein, we describe the rationale and design of the first study of venetoclax in pediatric patients with relapsed/refractory malignancies: a Phase I trial investigating the safety and pharmacokinetics of venetoclax monotherapy followed by the addition of chemotherapy (Trial registration: EudraCT 2017-000439-14; NCT03236857).

Keywords: ALL; AML; BCL-2; NHL; neuroblastoma; pediatric cancer; relapsed/refractory; venetoclax.

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Age Factors
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Biomarkers
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use*
  • Child, Preschool
  • Clinical Protocols*
  • Drug Development*
  • Drug Resistance, Neoplasm
  • Humans
  • Neoplasms / drug therapy*
  • Recurrence
  • Research Design
  • Sulfonamides / pharmacology
  • Sulfonamides / therapeutic use*
  • Treatment Outcome


  • Antineoplastic Agents
  • Biomarkers
  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • venetoclax

Associated data