Randomized phase II trial of bevacizumab and temsirolimus in combination with vinorelbine (V) and cyclophosphamide (C) for first relapse/disease progression of rhabdomyosarcoma (RMS): A report from the Children's Oncology Group (COG).

10003 Background: Patients with RMS have a poor prognosis at first relapse/disease progression. VC has activity in RMS and preclinical data supports the use of bevacizumab and temsirolimus in RMS. Methods: Patients with biopsy proven RMS, 8 weeks, performance status < 2, adequate organ function and written informed consent. Patients were randomized between two regimens administered every 3 weeks for a maximum of 12 cycles: Regimen A- V 25 mg/m2 intravenously (IV) days 1 & 8, C 1.2 gms/m2 IV day 1, bevacizumab 15 mg/Kg IV day 1; b) Regimen B- VC identical to regimen A, temsirolimus 15 mg/m2 IV days 1, 8 & 15. Primary endpoint was event free survival (EFS) at 6 months. Disease response at week 6 was assessed using RECIST. The study had a phase 2 screening design and was powered to detect a 15% difference in EFS between the two regimens (α=0.2, 1-β=0.8, 2-sided test). Interim analysi...