A Phase 1/2 Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
Roberto Chiarle
Summary
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.
Description
This study consists of two phases. The primary objectives of Phase 1 and Phase 2 are: Phase 1: * To identify the maximum tolerated dose (MTD) of autologous hALK.CAR T cells, and the recommended phase 2 dose (RP2D) in participants with relapsed/refractory high-risk neuroblastoma. * To evaluate the feasibility of manufacturing autologous hALK.CAR T cells. Phase 2: To estimate the complete response (CR) and partial response (PR) rates per revised International Neuroblastoma Response Criteria (INRC) of participants with relapsed or refractory high-risk neuroblastoma who are treated with hALK.C…
Eligibility
- Age range
- 1–29 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. 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 2. Disease Status: 1. Patients must have histologic verification of neuroblastoma at diagnosis or at relapse 2. Patients must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at time of study enrollment 3. Patients must have persistent/refractory or relapsed disease for which standard curative measures are no longer effective, as defined in the protocol 4. Patients must have evaluable or…
Interventions
- BiologicalAutologous hALK.CAR T cells
Autologous chimeric antigen receptor T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor
Locations (2)
- Boston Children's HospitalBoston, Massachusetts
- Dana-Farber Cancer InstituteBoston, Massachusetts