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Relapsed NeuroblastomaPhase 1/2NCT06803875

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

A Phase 1/2 study testing Autologous hALK.CAR T cells in people with Relapsed Neuroblastoma and Refractory Neuroblastoma, run by Roberto Chiarle at 2 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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). Read more about this study

Who is it for?
Ages 12 months to 29 years
What is being tested?
Autologous hALK.CAR T cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
42 participants
Where
2 sitesUnited States
Study dates
31 Jan 2025to 31 Dec 2029
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 months to 29 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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)
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  • 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

Who cannot join

  • Pregnant or nursing (lactating) women
  • Patients with uncontrolled active infection
  • Patients who are concurrently receiving other investigational agents
  • Patients who have received prior CART-cell or other gene-modified immune-effector cell therapy, are not eligible unless they are >8 weeks from time of infusion, have fully recovered from any associated toxicities and have documented lack of persistence of the product
  • Patients with a known additional malignancy other than non-melanomatous skin cancer or carcinoma in situ, unless not requiring active treatment and stable or disease-free for at least 3 years
  • Uncontrolled CNS metastasis
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  • CNS disorder such as cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement which may impair the ability to evaluate neurotoxicity
  • History of severe hypersensitivity reaction to compounds used in the study
  • HIV/HBV/HCV infection
  • Patients receiving systemic steroid therapy (physiologic replacement, inhaled steroids and premedication for blood products are allowed)
  • Primary immunodeficiency or history of systemic autoimmune disease requiring systemic immunosuppression/disease modifying agents within the last 2 years
  • Uncontrolled intercurrent illness
  • Inability to comply with the study requirements

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Susanne Baumeister, MD contact

617-632-3796

dfbchpedicelltherapy@dfci.harvard.edu

Audra Caine contact

617-632-3796

audra_caine@dfci.harvard.edu

United States2 sites, 2 recruiting

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

    Susanne Baumeister, MD, 617-632-3796, dfbchpedicelltherapy@dfci.harvard.edu

    Susanne Baumeister, MD

    Recruiting

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115

    Susanne Baumeister, MD, 617-632-3796, dfbchpedicelltherapy@dfci.harvard.edu

    Susanne Baumeister, MD

    Recruiting

Other trials in United States

About this study

Official title: 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.

Read the 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.

Being tested
Autologous hALK.CAR T cells
Keywords
Relapsed Neuroblastoma, Refractory Neuroblastoma, High-Risk Neuroblastoma
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autologous hALK.CAR T cellsBiological

    Autologous chimeric antigen receptor T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor

Study groups in NCT06803875
Study groupRoleWhat they receive
Phase 1 Dose EscalationExperimentalThe 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.
Receives: Biological: Autologous hALK.CAR T cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1: Determine the Maximum Tolerated Dose (MTD) of hALK.CAR T cells

    The Maximum Tolerated Dose (MTD) of hALK.CAR T cells will be determined by measuring the incidence of dose limiting toxicities (DLT) following administration of the hALK.CAR T cell product using a 3+3 dose escalation design.

    Measured: 5 years

  2. Phase 1: Evaluate Manufacturing Feasibility of hALK.CAR T cells

    Manufacturing feasibility will be evaluated as the proportion of patients undergoing leukapheresis who achieve manufacturing of a hALK.CAR T cell product that meets release criteria.

    Measured: 5 years

  3. Phase 2: Estimate Response Rates

    The complete response (CR) and partial response (PR) rates per revised International Neuroblastoma Response Criteria (INRC) of subjects with relapsed or refractory high-risk neuroblastoma who are treated with hALK.CAR T cells will be estimated.

    Measured: Up to 5 years

Secondary outcomes (5)

  1. Phase 1: Estimate Response Rates

    The complete response (CR) and partial response (PR) rates per revised International Neuroblastoma Response Criteria (INRC) of subjects with relapsed or refractory high-risk neuroblastoma who are treated with hALK.CAR T cells in the Phase 1 cohort will be estimated.

    Measured: 5 years

  2. Estimate Progression Free Survival and Overall Survival

    To estimate the progression free survival (PFS) and overall survival (OS) rates of subjects with relapsed or refractory high-risk neuroblastoma who are treated with hALK.CAR T cells.

    Measured: 5 years

  3. Evaluate Patient-Reported Symptoms

    Patient-reported symptoms of interest (including mood, gastrointestinal and pain symptoms) will be measured in subjects treated with hALK.CAR T cells using the Ped-PRO-CTCAE v.1 inventory prior to and until 2 years after hALK.CAR T cell infusion

    Measured: Up to 5 years

  4. Persistence of hALK.CAR T cells

    Persistence of hALK.CAR T cells will be measured by Polymerase Chain Reaction (or flow cytometry) analysis to detect and quantify survival of hALK.CAR T cells in the peripheral blood over time.

    Measured: Up to 5 years

  5. Cytokine levels in the peripheral blood

    Activity of hALK.CAR T cells will be assessed by measuring cytokine levels in the peripheral blood over time

    Measured: 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Jan 2025
  2. Study start31 Jan 2025
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion31 Dec 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
42 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06803875
Sponsor's protocol ID
24-392
Status last verified
Dec 2025
First submitted
16 Sep 2024
Last updated
26 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: roberto.chiarle@childrens.harvard.edu. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Publications

  • Bergaggio E, Tai WT, Aroldi A, Mecca C, Landoni E, Nuesch M, Mota I, Metovic J, Molinaro L, Ma L, Alvarado D, Ambrogio C, Voena C, Blasco RB, Li T, Klein D, Irvine DJ, Papotti M, Savoldo B, Dotti G, Chiarle R. ALK inhibitors increase ALK expression and sensitize neuroblastoma cells to ALK.CAR-T cells. Cancer Cell. 2023 Dec 11;41(12):2100-2116.e10. doi: 10.1016/j.ccell.2023.11.004. Epub 2023 Nov 30.

    PubMed 38039964

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Autologous hALK.CAR T cells in people with Relapsed Neuroblastoma and Refractory Neuroblastoma, run by Roberto Chiarle at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 26 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Susanne Baumeister, MD (617-632-3796, dfbchpedicelltherapy@dfci.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1: Determine the Maximum Tolerated Dose (MTD) of hALK.CAR T cells (time frame: 5 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06803875, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Dec 2025.

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