A Phase II Trial of Vinorelbine and Cyclophosphamide with Bevacizumab or Temsirolimus in Patients with Recurrent/Refractory Rhabdomyosarcoma

Rhabdomyosarcoma that has returned or does not respond to standard therapies is challenging to treat. The purpose of this Children’s Oncology Group study is to compare two different drug combinations to treat children, adolescents, and young adults with recurrent or refractory rhabdomyosarcoma. Patients will be randomly assigned to receive intravenous vinorelbine and cyclophosphamide with either bevacizumab or temsirolimus.

These drug combinations are considered investigational for recurrent/refractory rhabdomyosarcoma. However, vinorelbine, cyclophosphamide, and temsirolimus have all been used, by themselves or with other drugs, to treat children with newly diagnosed or recurrent/refractory rhabdomyosarcoma. Bevacizumab has not been used to treat rhabdomyosarcoma.

Vinorelbine and cyclophosphamide are commonly used chemotherapy drugs. Bevacizumab is approved for treating several advanced solid tumors in adults, and works by inhibiting the formation of blood vessels that tumors need to grow and spread. Temsirolimus, a drug which is approved for the treatment of kidney cancer, targets a protein called mTOR, and may also help some patients with sarcomas.