Actively Recruiting
Phase 1 Trial of PHOX2B Peptide-Centric Chimeric Antigen Receptor Autologous T Cells for Relapsed Neuroblastoma
Led by Stephan Grupp MD PhD · Updated on 2025-12-26
38
Participants Needed
1
Research Sites
156 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 research aims to evaluate the safety of PHOX2B PC-CAR T cells in patients with advanced, high-risk neuroblastoma, a serious childhood tumor arising from nerve cells. Neuroblastoma varies widely, but about half of patients have a high-risk form that often returns after treatment and currently has no cure. The trial is a first-in-human, phase 1 dose escalation and expansion study to assess safety, tolerability, and manufacturing feasibility of this new therapy targeting the PHOX2B protein, which is highly specific to neuroblastoma. Participants will receive genetically modified autologous T cells designed to target PHOX2B. The trial has two parts: a dose escalation phase to find the highest safe dose using a standard 3+3 design, and a dose expansion phase to further evaluate safety and preliminary response at the safe dose. This open-label study is conducted at a single institution and will monitor patients over several years. Throughout the study, participants will undergo clinical assessments including monitoring for adverse events and evaluation of the therapy's persistence and clinical effects on neuroblastoma. Safety will be closely followed for up to five years, including repeated dosing effects. Researchers will also assess the feasibility of producing the PHOX2B PC-CAR T cells. The total participation time may extend over several years to fully capture long-term safety and activity.
CONDITIONS
Brief Title
PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 1 year or older
- Must have one of the specified HLA alleles confirmed by genotyping
- Diagnosed with high-risk neuroblastoma by COG risk classification
- Histologically confirmed diagnosis of neuroblastoma
- Have recurrent, progressive, refractory, or persistent neuroblastoma
- No effective standard curative treatments available
- Must have evaluable or measurable disease as defined by bone, bone marrow, or soft tissue involvement criteria
- Lansky or Karnofsky performance score of 60 or higher
- Adequate kidney function with age-adjusted serum creatinine 1.5 times upper limit normal
- Liver function within specified limits, with exceptions for Gilbert's Disease or liver metastases
- Pulmonary function with pulse oximetry at least 92% on room air and DLCO 60% if applicable
- Cardiac function with LVSF 28% or LVEF 50% or adequate ventricular function
- Negative pregnancy test for patients of child-bearing potential
- Use of medically acceptable contraception for at least 1 year after last infusion
You will not qualify if you...
- Active hepatitis B or C infection
- Active HIV infection (except controlled with undetectable viral load)
- Uncontrolled active infections
- Primary or acquired immunodeficiency disorders
- Use of systemic steroids or immunosuppression at cell infusion or collection times
- Progressing CNS metastases including brain or leptomeningeal involvement
- Active medical disorders increasing risk of uncontrollable cytokine release syndrome or neurotoxicity
- Receipt of live vaccines within 30 days before enrollment
- Pregnant or nursing (lactating) patients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Duration varies based on dosing and response
Participants receive the PHOX2B PC-CAR T cell therapy, which involves infusion of genetically modified autologous T cells targeting neuroblastoma.
1 to multiple visits depending on dose escalation and treatment schedule
Duration - Up to 5 years
Participants are monitored for safety, tolerability, and persistence of the therapy, as well as clinical response for up to 5 years after treatment.
Periodic visits over 5 years for monitoring
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.
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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