Phase 12 Study of Autologous hALK.CAR T Cells for Treating Relapsed or Refractory High-risk Neuroblastoma in Children and Young Adults

What this Trial Is About

Researchers are evaluating the safety and feasibility of using autologous chimeric antigen receptor CAR T cells that target the human Anaplastic Lymphoma Kinase ALK receptor to treat children and young adults with relapsed or refractory high-risk neuroblastoma. This Phase 12 trial aims to find the best dose and assess the response rates to these specialized immune cells. The study is led by Roberto Chiarle and involves careful monitoring of treatment effects and patient outcomes. The trial has two phases Phase 1 uses a dose escalation design to determine the maximum tolerated dose MTD and recommended dose for Phase 2. The treatment involves giving patients their own modified T cells designed to attack cancer cells. Phase 2 then expands to evaluate how well patients respond to this therapy in terms of complete and partial tumor responses. The study focuses on children and young adults up to 29 years old with specific disease criteria. Participants will undergo thorough evaluations including disease status assessments based on the International Neuroblastoma Response Criteria, organ function tests, and performance status checks. Researchers will track the feasibility of manufacturing the CAR T cells, measure response rates, progression-free survival, overall survival, and patient-reported symptoms over up to five years. Blood tests will monitor the persistence of CAR T cells and cytokine levels. The total participation time includes long-term follow-up to assess treatment effects and safety.

Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma