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This Phase 1/2 trial assesses the safety and feasibility of autologous CAR T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor in pediatric patients with relapsed or refractory neuroblastoma. Phase 1 determines the maximum tolerated dose (MTD) using a 3+3 dose escalation design, and Phase 2 is an expansion phase measuring response rates. Participants must be at least 12 months and under 30 years of age at consent with histologic verification of high-risk neuroblastoma, and the first patient at each dose level must be 6 years or older.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. 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 2. Disease Status: 1. Patients must have histologic verification of neuroblastoma at diagnosis or at relapse 2. Patients must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at time of study enrollment 3. Patients must have persistent/refractory or relapsed disease for which standard curative measures are no longer effective, as defined in the protocol 4. Patients must have evaluable or measurable disease per the revised International Neuroblastoma Response Criteria (INRC) 3. Adequate washout from prior treatment regimens 4. Adequate organ function 5. Adequate performance status defined as Lansky or Karnofsky performance score ≥50% 6. Subjects of reproductive potential must agree to use acceptable birth control methods 7. Signed informed consent Exclusion Criteria: 1. Pregnant or nursing (lactating) women 2. Patients with uncontrolled active infection 3. Patients who are concurrently receiving other investigational agents 4. 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 5. 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 6. Uncontrolled CNS metastasis 7. CNS disorder such as cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement which may impair the ability to evaluate neurotoxicity 8. History of severe hypersensitivity reaction to compounds used in the study 9. HIV/HBV/HCV infection 10. Patients receiving systemic steroid therapy (physiologic replacement, inhaled steroids and premedication for blood products are allowed) 11. Primary immunodeficiency or history of systemic autoimmune disease requiring systemic immunosuppression/disease modifying agents within the last 2 years 12. Uncontrolled intercurrent illness 13. Inability to comply with the study requirements