NCT02085148

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

A Multi-center, Open-label, Non-randomized, Phase I Dose Escalation Study of Regorafenib (BAY 73-4506) in Pediatric Subjects With Solid Malignant Tumors That Are Recurrent or Refractory to Standard Therapy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Bayer · Industry
• Sex: All
• Age: 6 Months – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Expansion Phase of the study: Subjects must have relapsed/refractory RMS or a solid malignant tumor (Ewing sarcoma, hepatoblastoma, neuroblastoma and Wilms tumor).

Conditions

• Pediatric Oncology

Interventions

Type	Name	Description
DRUG	Regorafenib (BAY73-4506)	Regorafenib will be given orally once a day, across cycles of 21 days each. During each cycle regorafenib is taken for 2 weeks followed by one week off the drug. Doses of the study drug used in this study are age-dependent and the children's dose will been adjusted based on the age and the body surface area and given either as tablets or granulate.
DRUG	Vincristine (Cellcristin®)	Vincristine will be given at a dose of 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg, maximum 2.0 mg) on Day 1 and Day 8 in 21-day cycles.
DRUG	Irinotecan (Irinotecan Cell pharm®)	Irinotecan will be administered at a starting dose of 50 mg/m2/day from Day 1 to Day 5, in 21 day cycles.

Timeline

• Start date: 2014-04-11
• Primary completion: 2019-05-05
• Completion: 2024-03-13
• First posted: 2014-03-12
• Last updated: 2024-04-22

Locations

12 sites across 4 countries: France, Italy, Spain, United Kingdom