NCT06803875

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

A Phase 1/2 Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Roberto Chiarle · Academic / Other
• Sex: All
• Age: 12 Months – 29 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.CAR T cells.

Conditions

• Relapsed Neuroblastoma
• Refractory Neuroblastoma
• High-risk Neuroblastoma

Interventions

Type	Name	Description
BIOLOGICAL	Autologous hALK.CAR T cells	Autologous chimeric antigen receptor T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor

Timeline

• Start date: 2025-01-31
• Primary completion: 2027-12-31
• Completion: 2029-12-31
• First posted: 2025-01-31
• Last updated: 2025-12-26

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study