Actively Recruiting
PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma
Led by Stephan Grupp MD PhD · Updated on 2025-12-26
38
Participants Needed
1
Research Sites
523 weeks
Total Duration
On this page
Sponsors
S
Stephan Grupp MD PhD
Lead Sponsor
C
Children's Hospital of Philadelphia
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a first in human dose escalation trial to determine the safety of administering PHOX2B PC-CAR T cells in patients with advanced, high-risk neuroblastoma.
CONDITIONS
Official Title
PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be 1 year of age or older
- Patients must have one of the specified HLA alleles confirmed by genotyping
- Patients must have high-risk neuroblastoma according to COG risk classification
- Patients must have a previously confirmed diagnosis of neuroblastoma
- Patients must have recurrent, progressive, refractory, or persistent neuroblastoma
- Patients must have disease for which standard curative treatments do not exist or are ineffective
- Patients must have measurable or evaluable disease with at least one bone, bone marrow, or soft tissue lesion meeting specific criteria
- Patients must have a Lansky score (for those 16 years or younger) or Karnofsky score (for older than 16) of 60 or higher
- Patients must have adequate renal function with age-adjusted serum creatinine within 1.5 times the upper limit of normal
- Patients must have liver function with total bilirubin ≤ 1.5 times upper limit of normal (exceptions allowed), ALT ≤ 3 times upper limit (exceptions allowed), and AST ≤ 3 times upper limit (exceptions allowed)
- Patients must have pulmonary function with pulse oximetry ≥ 92% on room air and DLCO ≥ 60% if testing is appropriate
- Patients must have cardiac function with left ventricular shortening fraction ≥ 28% or ejection fraction ≥ 50%, or equivalent ventricular function
- Female patients of child-bearing potential must have a negative pregnancy test and use contraception for at least 1 year after treatment
You will not qualify if you...
- Patients with active hepatitis B or active hepatitis C
- Patients with active HIV infection (except those on anti-retroviral therapy with undetectable viral load)
- Patients with uncontrolled active infection
- Patients with primary or acquired immunodeficiency disorder
- Patients using systemic steroids or immunosuppression at the time of cell infusion or collection, or expected to require these during the process
- Patients with actively progressing central nervous system metastases
- Patients with medical conditions increasing risk of uncontrollable cytokine release syndrome or neurotoxicity
- Patients who received live vaccines within 30 days prior to enrollment
- Pregnant or breastfeeding patients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
M
Melissa Varghese, B.A.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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