Venetoclax in Pediatric and Young Adult Patients With Relapsed/Refractory Solid Tumors: Results of a Phase 1 Study

Daniel A. Morgenstern; Giuseppe Barone; N. Corradini; Sara M. Federico; Christian Flotho; N J Gerber; Seong Lin Khaw; Margaret E. Macy; Andrew E. Place; Kieuhoa T. Vo; Mohamed Badawi; Xin Chen; Adam Luo; Mark A. Rocco; Jeremy A. Ross; Frank Scherer; Gauri Sunkersett; Maika Onishi; Kelly C. Goldsmith

doi:10.1002/1545-5017.70178

Key Points

To evaluate the safety and efficacy of venetoclax in children and young adults with relapsed or refractory solid tumors.
Conducted a Phase 1 study to determine the recommended dose of venetoclax.
Used a dosing regimen of 800 mg/day over 21-day cycles.
Combined venetoclax with Cy-Topo chemotherapy.
Recommended dose of venetoclax determined to be 800 mg/day.
The combination treatment showed a predictable safety profile.
Clinical activity was modest in the studied cohorts.

Implication

Phase 1 study finds venetoclax dosage of 800 mg/day safe with chemotherapy in pediatric and young adults, suggesting potential benefits for solid tumors.

Abstract

Recommended Phase 2 dose of venetoclax was 800 mg/day AED (Days 1-10 of 21-day cycles). Venetoclax combined with Cy-Topo chemotherapy had a predictable safety profile and showed modest clinical activity in these cohorts.

Discussion

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Cite This Study

Morgenstern et al. (2026) studied this question.

synapsesocial.com/papers/69b6069b83145bc643d1ca8a https://doi.org/10.1002/1545-5017.70178

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Venetoclax in Pediatric and Young Adult Patients With Relapsed/Refractory Solid Tumors: Results of a Phase 1 Study

Key Points

Implication

Abstract

Discussion

Cite This Study

Venetoclax in Pediatric and Young Adult Patients With Relapsed/Refractory Solid Tumors: Results of a Phase 1 Study

Key Points

Implication

Abstract

Discussion

Cite This Study

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