Phase II Trial of Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma BCC022
Objective
Safety Run-in: To evaluate the safety and tolerability of Tipifarnib in combination with Naxitamab in pediatric and young adult patients in the first 6 DLT (dose limiting toxicity) evaluable subjects. The first 6 subjects enrolled will be age 6 years or older.
Primary Objective: To evaluate the activity of Tipifarnib in combination with Naxitamab in relapsed/refractory neuroblastoma based on overall response rate (ORR).
Secondary Objectives: To evaluate Duration of response (DOR), defined as the time from first documentation of response (CR or PR) to the date of first documented disease progression or death due to any cause, whichever occurs first. To evaluate the efficacy of Tipifarnib in combination with Naxitamab based upon: Event Free Survival (PFS), Overall survival (OS). To evaluate the safety and tolerability of Tipifarnib in combination with Naxitamab in pediatric and young adult patients. To evaluate response of subjects with only bone and bone marrow disease. To evaluate response of subjects with relapsed versus refractory disease. To evaluate disease control rate (e.g., CR, PR, MR + SD with duration ≥ 12 weeks).
