HALK.CAR T Cells for Neuroblastoma

Recruiting at 1 trial location

Overseen By Susanne Baumeister, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Roberto Chiarle

Must not be taking: Investigational agents, Systemic steroids

Disqualifiers: Pregnancy, Uncontrolled infection, CNS metastasis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

42 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Susanne Baumeister, MD

Principal Investigator

Dana-Farber Cancer Institute

Eligibility Criteria

This trial is for pediatric patients with neuroblastoma that has come back or hasn't responded to treatment. Specific details about who can join are not provided, but typically participants need to meet certain health standards and may have restrictions based on previous treatments.

Inclusion Criteria

I have waited the required time after my last cancer treatment.

My organs are working well.

I can do most activities but may need help.

Exclusion Criteria

Pregnant or nursing (lactating) women

Patients who are concurrently receiving other investigational agents

I do not have any uncontrolled infections.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1: Dose Escalation

Determine the maximum tolerated dose (MTD) of hALK.CAR T cells using a 3+3 dose escalation design

5 years

Phase 2: Expansion

Estimate the complete response (CR) and partial response (PR) rates of participants treated with hALK.CAR T cells

Up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

Treatment Details

Interventions

• hALK.CAR T cells

Trial Overview The study is testing a new therapy using the patient's own immune cells, modified to target cancer cells. The first phase finds the highest dose kids can take without serious side effects (MTD), and the second phase sees how well it works at that dose.

Participant Groups

Group I: Phase 1 Dose Escalation Experimental Treatment 1 Intervention

The dose escalation arm will determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of hALK.CAR T cells using a standard 3+3 dose escalation design.

Who Is Running the Clinical Trial?

Roberto Chiarle

Lead Sponsor

Trials

1

Recruited

40+

Boston Children's Hospital

Collaborator

Trials

801

Recruited

5,584,000+

Dana-Farber Cancer Institute

Collaborator

Trials

1,128

Recruited

382,000+