RECRUITING

Study of HALK.CAR T Cells for Patients with Relapsed/refractory High-risk Neuroblastoma

Conditions

Relapsed Neuroblastoma Refractory Neuroblastoma High-risk Neuroblastoma

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase 1/2 Study of HALK.CAR T Cells for Patients with Relapsed/refractory High-risk Neuroblastoma

Quick Facts

Study Start:2025-03

Study Completion:2029-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06803875

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Months to 29 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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	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

Inclusion Criteria

Exclusion 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

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

Contacts and Locations

Study Contact

Susanne Baumeister, MD

CONTACT

617-632-3796

dfbchpedicelltherapy@dfci.harvard.edu

Audra Caine

CONTACT

617-632-3796

audra_caine@dfci.harvard.edu

Principal Investigator

Susanne Baumeister, MD

PRINCIPAL_INVESTIGATOR

Dana-Farber Cancer Institute

Study Locations (Sites)

Boston Children's Hospital

Boston, Massachusetts, 02115

United States

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115

United States

Collaborators and Investigators

Sponsor: Roberto Chiarle

Susanne Baumeister, MD, PRINCIPAL_INVESTIGATOR, Dana-Farber Cancer Institute

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-03

Study Completion Date2029-12-31

Study Record Updates

Study Start Date2025-03

Study Completion Date2029-12-31

Terms related to this study

Keywords Provided by Researchers

Relapsed Neuroblastoma
Refractory Neuroblastoma
High-Risk Neuroblastoma

Additional Relevant MeSH Terms

Relapsed Neuroblastoma
Refractory Neuroblastoma
High-risk Neuroblastoma