ALK inhibitors increase ALK expression and sensitize neuroblastoma cells to ALK.CAR-T cells.
Elisa Bergaggio, Wei-Tien Tai, Andrea Aroldi...
https://pubmed.ncbi.nlm.nih.gov/38039964Actively Recruiting
Phase 1
Phase 2
Age: 12Months - 29Years
All Genders
ID06803875
A Phase 1/2 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
104 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
Researchers are evaluating the safety and feasibility of using autologous chimeric antigen receptor (CAR) T cells that target the human Anaplastic Lymphoma Kinase (ALK) receptor to treat children and young adults with relapsed or refractory high-risk neuroblastoma. This Phase 1/2 trial aims to find the best dose and assess the response rates to these specialized immune cells. The study is led by Roberto Chiarle and involves careful monitoring of treatment effects and patient outcomes. The trial has two phases: Phase 1 uses a dose escalation design to determine the maximum tolerated dose (MTD) and recommended dose for Phase 2. The treatment involves giving patients their own modified T cells designed to attack cancer cells. Phase 2 then expands to evaluate how well patients respond to this therapy in terms of complete and partial tumor responses. The study focuses on children and young adults up to 29 years old with specific disease criteria. Participants will undergo thorough evaluations including disease status assessments based on the International Neuroblastoma Response Criteria, organ function tests, and performance status checks. Researchers will track the feasibility of manufacturing the CAR T cells, measure response rates, progression-free survival, overall survival, and patient-reported symptoms over up to five years. Blood tests will monitor the persistence of CAR T cells and cytokine levels. The total participation time includes long-term follow-up to assess treatment effects and safety.
CONDITIONS
Brief Title
Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
Who Can Participate
Age: 12Months - 29Years
All Genders
Eligibility Criteria
You may qualify if you...
- Age 1 month or older and under 30 years at time of consent; first patient on each dose level must be 6 years or older
- Histologic verification of neuroblastoma at diagnosis or relapse
- High-risk neuroblastoma as defined by Children's Oncology Group criteria at enrollment
- Persistent, refractory, or relapsed disease not responsive to standard curative treatments
- Evaluable or measurable disease by revised International Neuroblastoma Response Criteria
- Adequate washout period from prior treatments
- Adequate organ function
- Performance status of 50% or higher by Lansky or Karnofsky score
- Agreement to use acceptable birth control methods if of reproductive potential
- Signed informed consent
You will not qualify if you...
- Pregnant or nursing women
- Uncontrolled active infections
- Concurrent use of other investigational agents
- Prior CAR T-cell or gene-modified immune therapy within 8 weeks unless fully recovered and no persistence of prior product
- Known additional malignancy except certain skin cancers unless stable or disease-free for 3 years
- Uncontrolled central nervous system metastasis
- CNS disorders impairing neurotoxicity evaluation
- History of severe allergic reactions to study compounds
- HIV, HBV, or HCV infection
- Systemic steroid therapy (except physiologic replacement, inhaled steroids, or premedication for blood products)
- Primary immunodeficiency or recent systemic autoimmune disease needing immunosuppression
- Uncontrolled intercurrent illness
- Inability to comply with study requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 5 years depending on participant response and dosing schedule
Participants receive autologous hALK.CAR T cells as an investigational therapy for relapsed or refractory high-risk neuroblastoma.
Visits occur according to dose escalation and monitoring schedules during treatment
Follow-up
Duration - Up to 5 years
Participants are monitored for response to treatment, side effects, and long-term outcomes including survival and persistence of hALK.CAR T cells.
Regular follow-up visits for assessments and monitoring
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
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Research Team
S
Susanne Baumeister, MD
A
Audra Caine
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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