NCT06803875 Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
| NCT ID | NCT06803875 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Roberto Chiarle |
| Condition | Relapsed Neuroblastoma |
| Study Type | INTERVENTIONAL |
| Enrollment | 42 participants |
| Start Date | 2025-01-31 |
| Primary Completion | 2027-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 42 participants in total. It began in 2025-01-31 with a primary completion date of 2027-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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
Contact & Investigator
Susanne Baumeister, MD
PRINCIPAL INVESTIGATOR
Dana-Farber Cancer Institute
Frequently Asked Questions
Who can join the NCT06803875 clinical trial?
This trial is open to participants of all sexes, aged 12 Months or older, up to 29 Years, studying Relapsed Neuroblastoma. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06803875 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06803875 currently recruiting?
Yes, NCT06803875 is actively recruiting participants. Contact the research team at dfbchpedicelltherapy@dfci.harvard.edu for enrollment information.
Where is the NCT06803875 trial being conducted?
This trial is being conducted at Boston, United States, Boston, United States.
Who is sponsoring the NCT06803875 clinical trial?
NCT06803875 is sponsored by Roberto Chiarle. The principal investigator is Susanne Baumeister, MD at Dana-Farber Cancer Institute. The trial plans to enroll 42 participants.