期刊
FUTURE ONCOLOGY
卷 14, 期 21, 页码 2115-2129出版社
FUTURE MEDICINE LTD
DOI: 10.2217/fon-2018-0121
关键词
ALL; AML; BCL-2; neuroblastoma; NHL; pediatric cancer; relapsed; refractory; venetoclax
类别
资金
- AbbVie
- Roche/Genentech
- Jazz Pharmaceuticals
- Genentech
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).
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