Tests CAR T cells against relapsed high-risk neuroblastoma in children
Tests patient's own ALK-targeting CAR T cells in children with relapsed neuroblastoma.
Key facts
- Who runs it
- Roberto Chiarle
- Where
- Boston, United StatesSee the 2 sites
- Trial code
- Registry title
- Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
- Status
- Recruiting
- Phase
- Phase 1/2
- People taking part
- 42 people, planned
- Started
- 31 Jan 2025
- Expected to end
- 31 Dec 2029
- Type of study
- Interventional
- Treatments tested
- Autologous hALK.CAR T cells
- Record updated
- 26 December 2025
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children and young adults with high-risk neuroblastoma that came back or did not respond to treatment. They would receive CAR T cells made from their own blood cells.
- Age
- From 12 months to under 30 years. The first patient at each dose level must be 6 years or older.
- Sex
- All sexes can join.
- Disease stage
- High-risk neuroblastoma (Children's Oncology Group classification) that came back or did not respond, with disease that can be seen or measured. Standard treatments meant to remove all of the disease no longer work.
- Earlier treatment
- Enough time must have passed since earlier treatments. Earlier CAR T or other gene-modified immune cell therapy is allowed only if more than 8 weeks have passed, side effects have fully gone, and the cells are no longer detectable.
Main reasons for not taking part
- Pregnant or breastfeeding
- Uncontrolled active infection
- HIV, hepatitis B or hepatitis C infection
- Uncontrolled spread of the cancer to the central nervous system
- Taking systemic steroids or other investigational drugs at the same time
The registry criteria, word for word
Show all 20 criteriaShow less
Who can take part
- 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)
- 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 take part
- 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 receiving systemic steroid therapy (physiologic replacement, inhaled steroids and premedication for blood products are allowed)
- Patients with uncontrolled active infection
- 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
- 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
- Primary immunodeficiency or history of systemic autoimmune disease requiring systemic immunosuppression/disease modifying agents within the last 2 years
- Uncontrolled intercurrent illness
- Pregnant or nursing (lactating) women
- Uncontrolled CNS metastasis
- Inability to comply with the study requirements
Where in United States
- Boston Children's HospitalBoston, United StatesContact: Susanne Baumeister, MDTaking people
- Dana-Farber Cancer InstituteBoston, United StatesContact: Susanne Baumeister, MDTaking people
About the trial
What the trial wants to find out
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 full original descriptionShow less
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.
The group in the trial
- Phase 1 Dose EscalationTreatment being tested
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.
Treatment: Autologous hALK.CAR T cells
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A Phase 1/2 Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
- Plain-language title
- Tests CAR T cells against relapsed high-risk neuroblastoma in childrenGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Record updated
- 26 December 2025