A Phase 1/2 Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
This Phase 1/2 trial aims to determine the safety and feasibility of administration of autologous chimeric antigen receptor (CAR) T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor in pediatric subjects with relapsed or refractory neuroblastoma (NB). The trial will be conducted in two phases: Phase 1 will determine the maximum tolerated dose (MTD) of autologous hALK.CAR T cells using a 3+3 dose escalation design. Phase 2 will be an expansion phase to determine rates of response to hALK.CAR T cells.
• Age ≥ 12 months and \< 30 years at the time of consent. The first patient on each dose level will need to be age ≥ 6 years old
• Disease Status:
‣ Patients must have histologic verification of neuroblastoma at diagnosis or at relapse
⁃ Patients must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at time of study enrollment
⁃ Patients must have persistent/refractory or relapsed disease for which standard curative measures are no longer effective, as defined in the protocol
⁃ Patients must have evaluable or measurable disease per the revised International Neuroblastoma Response Criteria (INRC)
• Adequate washout from prior treatment regimens
• Adequate organ function
• Adequate performance status defined as Lansky or Karnofsky performance score ≥50%
• Subjects of reproductive potential must agree to use acceptable birth control methods
• Signed informed consent