Actively Recruiting
Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
Led by Roberto Chiarle · Updated on 2025-12-26
42
Participants Needed
2
Research Sites
256 weeks
Total Duration
On this page
Sponsors
R
Roberto Chiarle
Lead Sponsor
B
Boston Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 12 months or older and less than 30 years at consent; first patient at each dose level must be at least 6 years old
- Histologic confirmation of neuroblastoma at diagnosis or relapse
- High-risk neuroblastoma by Children's Oncology Group risk classification at enrollment
- Persistent, refractory, or relapsed disease not responsive to standard curative treatments
- Evaluable or measurable disease per revised International Neuroblastoma Response Criteria
- Adequate washout from prior treatments
- Adequate organ function
- Lansky or Karnofsky performance score of 50% or higher
- Agreement to use acceptable birth control if of reproductive potential
- Signed informed consent
You will not qualify if you...
- Pregnant or nursing (lactating) women
- Uncontrolled active infection
- Concurrent treatment with other investigational agents
- Prior CAR T-cell or gene-modified immune-effector cell therapy within 8 weeks or with ongoing toxicities or persistence
- Known additional malignancy except non-melanomatous skin cancer or carcinoma in situ unless stable/disease-free for at least 3 years
- Uncontrolled central nervous system metastasis
- CNS disorders impairing neurotoxicity evaluation (e.g., cerebrovascular ischemia, dementia, cerebellar disease, autoimmune CNS disease)
- History of severe hypersensitivity to study compounds
- HIV, HBV, or HCV infection
- Receiving systemic steroid therapy (except physiologic replacement, inhaled steroids, or premedication for blood products)
- Primary immunodeficiency or systemic autoimmune disease requiring immunosuppression within last 2 years
- Uncontrolled intercurrent illness
- Inability to comply with study requirements
AI-Screening
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Trial Site Locations
Total: 2 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
2
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Actively Recruiting
Research Team
S
Susanne Baumeister, MD
CONTACT
A
Audra Caine
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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