drugset / Trial / NCT03236857
A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
NaSingle-groupOpen-labelTreatment
Summary
An open-label, global, multi-center study to evaluate the safety and pharmacokinetics of venetoclax monotherapy, to determine the dose limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to assess the preliminary efficacy of venetoclax in pediatric and young adult participants with relapsed or refractory malignancies.
Timeline
- Start
- 2017-11-08
- Primary completion
- 2023-04-19
- Completion
- 2023-04-19
Publications
- Karol SE, Khaw SL, Zwaan CM, Baruchel A, Bittencourt H, Cooper TM, Flotho C, Fraser C, Forlenza CJ, Goldsmith KC, Macy ME, Morgenstern DA, O'Brien MM, Petit A, Ziegler DS, Reinhardt D, Opferman JT, Rubnitz JE, Onishi M, Dunshee DR, Dunbar F, Vishwamitra D, Ross JA, Chen X, Unnebrink K, Kammerlander M, Salem AH, Palenski TL, Sunkersett G, Place AE. Venetoclax Alone or in Combination With Chemotherapy in Paediatric and Adolescent/Young Adult Patients With Relapsed/Refractory Acute Myeloid Leukaemia. Pediatr Blood Cancer. 2025 Jul;72(7):e31714. doi: 10.1002/pbc.31714. Epub 2025 Apr 23.
- Place AE, Karol SE, Forlenza CJ, Cooper TM, Fraser C, Cario G, O'Brien MM, Gerber NU, Bourquin JP, Reinhardt D, Rubnitz JE, Opferman JT, Sunkersett G, Onishi M, Dunshee DR, Chen X, Unnebrink K, Vishwamitra D, Dunbar F, Badawi M, Ross JA, Loh ML. Venetoclax Combined With Chemotherapy in Pediatric and Adolescent/Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia. Pediatr Blood Cancer. 2025 Jun;72(6):e31630. doi: 10.1002/pbc.31630. Epub 2025 Mar 10.
- Badawi M, Gopalakrishnan S, Engelhardt B, Palenski T, Karol SE, Rubnitz JE, Menon R, Salem AH. Dosing of Venetoclax in Pediatric Patients with Relapsed Acute Myeloid Leukemia: Analysis of Developmental Pharmacokinetics and Exposure-Response Relationships. Clin Ther. 2024 Oct;46(10):759-767. doi: 10.1016/j.clinthera.2024.09.008. Epub 2024 Oct 5.
- Dalton KM, Krytska K, Lochmann TL, Sano R, Casey C, D'Aulerio A, Khan QA, Crowther GS, Coon C, Cai J, Jacob S, Kurupi R, Hu B, Dozmorov M, Greninger P, Souers AJ, Benes CH, Mosse YP, Faber AC. Venetoclax-based Rational Combinations are Effective in Models of MYCN-amplified Neuroblastoma. Mol Cancer Ther. 2021 Aug;20(8):1400-1411. doi: 10.1158/1535-7163.MCT-20-0710. Epub 2021 Jun 4.
- Place AE, Goldsmith K, Bourquin JP, Loh ML, Gore L, Morgenstern DA, Sanzgiri Y, Hoffman D, Zhou Y, Ross JA, Prine B, Shebley M, McNamee M, Farazi T, Kim SY, Verdugo M, Lash-Fleming L, Zwaan CM, Vormoor J. 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.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Venetoclax | Small molecule | — | Oral |
| Background | Azacitidine | Other / unclassified | — | — |
| Background | Cyclophosphamide | Other / unclassified | — | — |
| Background | Cytarabine | Small molecule | — | — |
| Background | Decitabine | Small molecule | — | — |
| Background | Dexamethasone | Small molecule | — | — |
| Background | Etoposide | Small molecule | — | — |
| Background | Pegaspargase | Protein / enzyme biologic | — | — |
| Background | Rituximab | Monoclonal antibody | — | — |
| Background | Topotecan | Small molecule | — | — |
| Background | Vincristine | Small molecule | — | — |