Actively Recruiting
GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma
Led by Stephan Grupp MD PhD · Updated on 2025-12-29
45
Participants Needed
1
Research Sites
349 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 GPC2 CAR T cells in patients with advanced neuroblastoma or retinoblastoma.
CONDITIONS
Official Title
GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be at least 1 year old for neuroblastoma or at least 6 months old for retinoblastoma
- Patients must have high-risk neuroblastoma or metastatic retinoblastoma confirmed by standard classifications
- Patients must have relapsed, refractory, or persistent disease for which standard curative treatments do not exist or are no longer effective
- Patients must have measurable or evaluable disease at enrollment
- Patients must meet specific disease progression or response criteria including new or increasing lesions, biopsy confirmation, or partial response with residual disease
- Patients must have a Lansky score (for 16 years or younger) or Karnofsky score (for older than 16 years) of at least 60
- Patients must have adequate kidney, liver, lung, and heart function as defined by laboratory and clinical assessments
- Patients must have a baseline pulse oximetry of at least 92% on room air
- For neuroblastoma, patients must have a previously confirmed histological diagnosis
- For retinoblastoma cohort 1, extra-CNS metastasis must be histologically confirmed and measurable disease must be present
- For retinoblastoma cohort 2, CNS disease must meet defined measurable or non-measurable criteria
- Prior treatments vary by cohort but include intensive chemotherapy or equivalent regimens for recurrent or refractory disease
You will not qualify if you...
- Patients with active hepatitis B or hepatitis C infection
- Patients with HIV infection
- Patients with uncontrolled active infection
- Patients with primary or acquired immunodeficiency disorders
- Patients with known hypersensitivity to DMSO
- Patients using systemic steroids or immunosuppression during cell infusion or collection, except for physiologic replacement or inhaled steroids
- Patients with actively progressing CNS metastases (for neuroblastoma) or bulky CNS disease compressing brainstem or thalamus (for retinoblastoma cohort 2)
- Patients with active medical disorders increasing risk of severe cytokine release syndrome or neurotoxicity
- Patients with congestive heart failure (NYHA class III or IV), unstable angina, serious arrhythmias, recent myocardial infarction, or myocarditis
- Patients who received live vaccines within 30 days prior to enrollment
- Patients who are pregnant or nursing
- Patients with life expectancy less than 6 months
- Retinoblastoma cohort 1 patients with concurrent CNS disease or stage III disease
- Retinoblastoma cohort 2 patients with clinically significant increased intracranial pressure at enrollment if not improved
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
C
CART Nurse Navigator
CONTACT
M
Melissa Varghese, M.S.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here