Study of hALK.CAR T Cells for Patients With Relapsed/Refractory High-risk Neuroblastoma
NCT ID: NCT06803875
Last Updated: 2025-12-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
42 participants
INTERVENTIONAL
2025-01-31
2029-12-31
Brief Summary
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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.
Detailed Description
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Phase 1:
* To identify the maximum tolerated dose (MTD) of autologous hALK.CAR T cells, and the recommended phase 2 dose (RP2D) in participants with relapsed/refractory high-risk neuroblastoma.
* To evaluate the feasibility of manufacturing autologous hALK.CAR T cells.
Phase 2:
To estimate the complete response (CR) and partial response (PR) rates per revised International Neuroblastoma Response Criteria (INRC) of participants with relapsed or refractory high-risk neuroblastoma who are treated with hALK.CAR T cells.
Conditions
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Keywords
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Phase 1 Dose Escalation
The dose escalation arm will determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of hALK.CAR T cells using a standard 3+3 dose escalation design.
Autologous hALK.CAR T cells
Autologous chimeric antigen receptor T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor
Interventions
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Autologous hALK.CAR T cells
Autologous chimeric antigen receptor T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor
Eligibility Criteria
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Inclusion Criteria
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
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
12 Months
29 Years
ALL
No
Sponsors
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Boston Children's Hospital
OTHER
Dana-Farber Cancer Institute
OTHER
Roberto Chiarle
OTHER
Responsible Party
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Roberto Chiarle
Professor in Pathology
Principal Investigators
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Susanne Baumeister, MD
Role: PRINCIPAL_INVESTIGATOR
Dana-Farber Cancer Institute
Locations
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Boston Children's Hospital
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Countries
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Central Contacts
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Facility Contacts
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Susanne Baumeister, MD
Role: primary
Susanne Baumeister, MD
Role: primary
References
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Bergaggio E, Tai WT, Aroldi A, Mecca C, Landoni E, Nuesch M, Mota I, Metovic J, Molinaro L, Ma L, Alvarado D, Ambrogio C, Voena C, Blasco RB, Li T, Klein D, Irvine DJ, Papotti M, Savoldo B, Dotti G, Chiarle R. ALK inhibitors increase ALK expression and sensitize neuroblastoma cells to ALK.CAR-T cells. Cancer Cell. 2023 Dec 11;41(12):2100-2116.e10. doi: 10.1016/j.ccell.2023.11.004. Epub 2023 Nov 30.
Other Identifiers
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24-392
Identifier Type: -
Identifier Source: org_study_id