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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for children and young adults with neuroblastoma, a cancer that often affects nerve tissue. The treatment uses specially modified cells, called hALK.CAR T cells (a type of immunotherapy), designed to target and attack cancer cells. The first phase tests different dose levels to find the safest option, while the second phase evaluates the treatment's effectiveness. This trial targets those with high-risk neuroblastoma that has returned or hasn't responded to other treatments. Participants must have a diagnosis of neuroblastoma and symptoms that other treatments can't resolve. As a Phase 1 trial, this research focuses on understanding the treatment's effects in people, offering participants the opportunity to be among the first to receive this new therapy.

Will I have to stop taking my current medications?

The trial requires a washout period (time without taking certain medications) from prior treatments, so you may need to stop some medications. However, the protocol does not specify all medications that must be stopped, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that hALK.CAR T cells are likely to be safe for humans?

Research has shown that CAR T cell therapy, such as hALK.CAR T cells, has been safe and feasible in early studies for treating neuroblastoma. Although challenges exist in optimizing its effectiveness, the safety results are encouraging. These trials are in the early stages, focusing primarily on ensuring the treatment's safety.

In these studies, researchers are testing hALK.CAR T cells to ensure patients can tolerate the treatment without severe side effects. So far, evidence suggests the treatment could be safe for children with recurrent or hard-to-treat neuroblastoma. However, since this trial remains in the early stages, researchers are closely monitoring for any side effects as they continue testing. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatment options for neuroblastoma, which generally involve chemotherapy, surgery, radiation therapy, and sometimes stem cell transplants, hALK.CAR T cells present a novel approach by utilizing the body's own immune system to fight cancer. These engineered T cells are designed to specifically target and attack neuroblastoma cells that express the ALK protein, offering a more precise and potentially less toxic treatment option. Researchers are excited because this approach could lead to more effective and durable responses in patients who have limited options with conventional treatments.

What evidence suggests that hALK.CAR T cells might be an effective treatment for neuroblastoma?

Research has shown that a new treatment using special T cells, called hALK.CAR T cells, may help fight neuroblastoma, a type of cancer. These T cells target a specific part of the cancer cells. In lab tests, they demonstrated some ability to fight cancer, but their success was limited in certain animal studies. Early results suggest this treatment could be safe and might assist patients with challenging cases of neuroblastoma. While there is hope for its potential, more research is needed to determine its effectiveness in people. Participants in this trial will join a Phase 1 Dose Escalation arm to establish the maximum tolerated dose and recommended phase 2 dose of hALK.CAR T cells. ^{1 3 4 6 7}

Who Is on the Research Team?

Susanne Baumeister, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

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

My organs are working well.

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

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06803875?cond=NCT06803875&rank=1

NCT06803875 | Study of hALK.CAR T Cells for Patients ... This Phase 1/2 trial aims to determine the safety and feasibility of administration of autologous chimeric antigen receptor (CAR) T cells targeting the ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6232778/

CAR T Cell Therapy for Neuroblastoma - PMC - PubMed Central ALK CAR T cells demonstrated in vitro activity but had limited efficacy in vivo in xenograft models of neuroblastoma (114).

3. trials.braintumor.org trials.braintumor.org/trials/NCT06803875

Study of hALK.CAR T Cells for Patients With Relapsed ... 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 ...

4. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2025-00930

Autologous hALK. Chimeric Antigen Receptor T Cells ... Giving hALK. CAR T cells may be safe, tolerable, and/or effective in treating patients with relapsed or refractory high-risk neuroblastoma.

5. nature.com nature.com/articles/s41392-024-01827-y

rational dual strategy efficiently fights neuroblastoma This important approach demonstrates in a preclinical model that chimeric antigen receptor (CAR)-T cell treatment can be rendered more efficient.

6. dana-farber.org dana-farber.org/clinical-trials/24-392

A Phase 1/2 Study of hALK.CAR T cells for patients with ... This Phase 1/2 trial aims to determine the safety and feasibility of administration of autologous chimeric antigen receptor (CAR) T cells targeting the ...

7. trial.medpath.com trial.medpath.com/clinical-trial/f7bd16689f218e5e/nct06803875-halk-car-t-cells-neuroblastoma

Study of HALK.CAR T Cells for Patients with Relapsed ... This Phase 1/2 trial aims to determine the safety and feasibility of administration of autologous chimeric antigen receptor (CAR) T cells targeting the ...