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New treatment option for Refractory Neuroblastoma
Official title GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma
ClinicalTrials.gov ID: NCT05650749
What this study is testing
What is GPC2 CAR T cells?
GPC2 CAR T cells is an investigational medicine, being studied as a potential treatment for refractory neuroblastoma.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 and older
You may be able to join if
- Neuroblastoma
- Patients must be ≥ 1 year of age
- Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially...
- Patients must have a previously histologically confirmed diagnosis of neuroblastoma:
- That is recurrent/relapsed or refractory/persistent according to INRC AND
You likely can't join if
- Patients with active hepatitis B or active hepatitis C.
- Patients with HIV infection.
- Patients with uncontrolled active infection.
- Patients with primary or acquired immunodeficiency disorder.
- Patients with a known hypersensitivity to DMSO.
- Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's...
See the full eligibility criteria
Who can join
- Neuroblastoma
- Patients must be ≥ 1 year of age
- Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially considered low- or intermediate-risk, but then reclassified as high-risk are...
- Patients must have a previously histologically confirmed diagnosis of neuroblastoma:
- That is recurrent/relapsed or refractory/persistent according to INRC AND
- For which standard curative measures do not exist or are no longer effective.
- patients at first relapse are eligible as no known curative therapies exist for relapsed high-risk neuroblastoma.
- Patients must have evaluable or measurable disease at enrollment.
- In addition, patient must have experienced at least one of the following: a. New disease site documented on at least one of the following: i. 123I-meta-iodobenzylguanidine (MIBG) or 18F-mFBG (meta-fluorobenzylguanidine)...
- Patients must have a Lansky (≤ 16 years) or Karnofsky (\> 16 years) score of ≥ 60
- Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age.
- Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)
- Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).
- Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).
- Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.
- Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist. Neuroblastoma
What rules you out
- Patients with active hepatitis B or active hepatitis C.
- Patients with HIV infection.
- Patients with uncontrolled active infection.
- Patients with primary or acquired immunodeficiency disorder.
- Patients with a known hypersensitivity to DMSO.
- Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or...
- Patients with actively progressing CNS metastases, including parenchymal or leptomeningeal involvement. (Note: CNS imaging at screening is only required if the there is a clinical indication of suspected CNS metastasis)
- Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and/or neurotoxicity.
- Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months...
- Patients who have received any live vaccines within 30 days prior to enrollment.
- Patients who are pregnant or nursing (lactating).
- Patients who have a life expectancy \< 6 months at time of consent. Retinoblastoma Inclusion Criteria:
- Patient age ≥ 6 months.
- Patients must have metastatic retinoblastoma according to International Retinoblastoma Staging System (IRSS) risk classification (4) at the time of study enrollment: a. Retinoblastoma Cohort 1 (Extra-CNS metastasis) i...
- Patients must have a Lansky (≤ 16 years) or Karnofsky (\> 16 years) score of ≥ 60
- Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN .
- Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)
- Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).
- Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).
- Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.
- Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist. Retinoblastoma Exclusion Criteria:
- Patients with active hepatitis B or active hepatitis C.
- Patients with HIV infection.
- Patients with uncontrolled active infection.
- Patients with primary or acquired immunodeficiency disorder.
- Patients with a known hypersensitivity to DMSO.
- Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or...
- Active medical disorder that, in the opinion of the investigator, would substantially increase the risk to the subject.
- Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months...
- Patients who have received any live vaccines within 30 days prior to enrollment.
- Patients who are pregnant or nursing (lactating).
- Patients who have a life expectancy \< 6 months at time of consent.
- Retinoblastoma Cohort 1 (Extra-CNS disease):
- Concurrent CNS disease (they may be eligible for Retinoblastoma Cohort 2)
- Stage III disease (orbital or lymph node regional extension without other hematogenous metastases).
- Retinoblastoma Cohort 2 (CNS disease):
- "Bulky" disease (\>5 cm in diameter) within or compressing the brainstem or thalamus. Note: Tumors touching the brainstem/thalamus without evidence of compression and/or tumors in other CNS locations do not have a...
- If evidence of clinically significant increased intracranial pressure at time of relapse, patient must demonstrate improvement by time of enrollment.
The study team makes the final eligibility decision.
Where it's taking place
- Philadelphia, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.