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

Andrew E. Place, Kelly Goldsmith, Jean Pierre Bourquin, Mignon L. Loh, Lia Gore, Daniel A. Morgenstern, Yeshwant Sanzgiri, David Hoffman, Ying Zhou, Jeremy A. Ross, Betty Prine, Mohamad Shebley, Megan McNamee, Thalia Farazi, Su Young Kim, Maria Verdugo, Leanne Lash-Fleming, C. Michel Zwaan, Josef Vormoor

Onderzoeksoutput: Bijdrage aan tijdschriftArtikelpeer review

31 Citaten (Scopus)

Samenvatting

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).

Originele taal-2Engels
Pagina's (van-tot)2115-2129
Aantal pagina's15
TijdschriftFuture oncology (London, England)
Volume14
Nummer van het tijdschrift21
DOI's
StatusGepubliceerd - sep. 2018
Extern gepubliceerdJa

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