Phase II Trial of Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma

Summary and Objectives

Neuroblastoma, the most common solid tumor outside the skull in children, accounts for 7–10% of all childhood cancers—approximately 800 new cases each year in the U.S. Children diagnosed with high-risk neuroblastoma require intensive treatment to achieve cure. While cure rates have improved with current treatment approaches, outcomes are poor for patients who have refractory disease after intensive upfront treatment and for those whose disease returns after completing therapy. Salvage treatments have shown only modest success in the relapsed/refractory setting. Monoclonal antibodies have shown promise in improving outcomes for patients with high-risk neuroblastoma. Early studies also suggest that tipifarnib may enhance the tumor-fighting effects of monoclonal antibodies like naxitamab.

This study initially aims to evaluate the safety and tolerability of tipifarnib when combined with naxitamab in pediatric and young adult patients. Once the safety and tolerability has been evaluated in the first 6 enrolled participants, the study will begin the next phase, which then aims to evaluate the activity and efficacy of combining tipifarnib with naxitamab in children with relapsed or refractory high-risk neuroblastoma.

Eligibility criteria

In addition to some criteria that could prevent participation, there are criteria participants must meet including but not limited to the following:

• Age 0-21 years at initial diagnosis
• Must be > 12 months old at enrollment (the first 6 subjects – those enrolled in the safety and tolerability phase – must be ≥ 6 years old)
• Must have a pathologically confirmed diagnosis of neuroblastoma
• Must have active relapsed or refractory disease
• Tumor assessment and disease staging must be performed
• Must have protocol-defined adequate organ function
• Must not be pregnant or breastfeeding
• Must not be receiving another investigational drug or anti-cancer agent at the time of enrollment
• Must not have an active, uncontrolled infection

Clinicaltrials.gov Study Details >