A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy

Summary

Dose escalation phase of the study : To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in repeating cycles of 28 days in pediatric subjects with solid malignant tumors recurrent or refractory to standard therapy. To characterize the pharmacokinetics (PK) of regorafenib The dose escalation phase of the study has been completed. Expansion phase: To define the safety profile, MTD and the RP2D of regorafenib administered orally in combination with backbone chemotherapy (vincristine and irinotecan) at relapse in pediatric subjects with rhabdomyosarcoma (RMS) and other solid malignant tumors recurrent or refractory to standard therapy.

Timeline

Start

2014-04-11

Primary completion

2019-05-05

Completion

2024-03-13

Publications

• Casanova M, Bautista F, Campbell-Hewson Q, Makin G, Marshall LV, Verschuur AC, Canete Nieto A, Corradini N, Ploeger BA, Brennan BJ, Mueller U, Zebger-Gong H, Chung JW, Geoerger B. Regorafenib plus Vincristine and Irinotecan in Pediatric Patients with Recurrent/Refractory Solid Tumors: An Innovative Therapy for Children with Cancer Study. Clin Cancer Res. 2023 Nov 1;29(21):4341-4351. doi: 10.1158/1078-0432.CCR-23-0257.
• Geoerger B, Morland B, Jimenez I, Frappaz D, Pearson ADJ, Vassal G, Maeda P, Kincaide J, Mueller U, Schlief S, Teufel M, Ploeger BA, Cleton A, Agostinho AC, Marshall LV. Phase 1 dose-escalation and pharmacokinetic study of regorafenib in paediatric patients with recurrent or refractory solid malignancies. Eur J Cancer. 2021 Aug;153:142-152. doi: 10.1016/j.ejca.2021.05.023. Epub 2021 Jun 20.

Drugs

Indications