Study of Patritumab Deruxtecan to Treat Children With Relapsed or Refractory Hepatoblastoma or Rhabdomyosarcoma

What this Trial Is About

Researchers are evaluating a new treatment option for children with certain types of cancer, specifically hepatoblastoma and rhabdomyosarcoma RMS that have either come back after treatment or did not respond to prior therapies. Hepatoblastoma is a common liver cancer in very young children, while RMS begins in muscle cells and often appears in areas such as the head, neck, bladder, arms, or legs. This study is a phase 12 trial aiming to assess the safety and effectiveness of a drug called patritumab deruxtecan HER3-DXd in these pediatric patients. Participants will receive patritumab deruxtecan through an intravenous infusion on the first day of each three-week cycle. The study includes two parts first, a safety lead-in phase to determine a tolerable dose and assess safety, followed by a phase to evaluate how well the treatment works in shrinking or eliminating cancer. Participants will continue treatment until disease progression or other reasons for stopping occur. Throughout the study, children will be closely monitored for side effects and how the drug behaves in their bodies over time, with various blood tests and assessments conducted at designated times up to approximately five years. Researchers will measure outcomes such as the percentage of patients experiencing toxicities or adverse events, treatment response rates, and survival metrics. This long-term follow-up aims to gather comprehensive data on safety and effect.

A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)