Giselle Sholler
The purpose of this study is to evaluate the investigational drug, tipifarnib (a pill taken by mouth), in combination with the Food and Drug Administration (FDA) approved drug, naxitimab, administered intravenously (IV; a liquid that continuously goes into your body through a tube that has been placed during a surgery into one of your veins). Naxitamab is FDA approved for pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy, it may not be approved in the type of disease used in this study. The goals of this part of the study are: * Test the safety and tolerability of tipifarnib in combination with naxitimab in participants with cancer * To determine the activity of study treatments chosen based on: * How each participant responds to the study treatment * How long a participant lives without their disease returning/progressing
Inclusion Criteria: 1. Age: Participants must be age ≤ 21 years at initial diagnosis. Participants must be \>12 months of age at enrollment. Safety Run-In (first 6 participants) must be age 6 years or older. As of 09Jun2026 the safety run-in is complete. 2. Pathology: All participants must have a pathologically confirmed diagnosis of neuroblastoma at any point in their treatment. 3. Tumor assessment: Disease staging must be performed. This disease assessment is required for eligibility and must be done within a maximum of 4 weeks before first dose of study drug. 4. Disease Status: Relapse…
Tablet
IV