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

Launched by ROBERTO CHIARLE · Jan 28, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for children and young adults with high-risk neuroblastoma, a type of cancer that can come back after treatment or does not respond to standard therapies. The treatment involves using specially modified immune cells, called CAR T cells, that are designed to target a specific marker (the ALK receptor) found on neuroblastoma cells. The goal of the trial is to find out how safe this treatment is and to determine the best dose to use, as well as to see how well it works for patients.

To be eligible for this trial, participants must be between 12 months and 30 years old and have a confirmed diagnosis of neuroblastoma that is either relapsed or not responding to other treatments. They should have measurable disease and good overall health, meaning they can carry out daily activities reasonably well. However, some individuals, such as pregnant women or those with certain health issues, may not qualify. If they choose to participate, patients can expect close monitoring throughout the trial, including assessments of their health and how their cancer responds to the treatment. This study is currently recruiting participants, and it represents an important step in exploring new options for children facing challenging cancer cases.

Gender

ALL

Eligibility criteria

• 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