RECRUITING

GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma

Conditions

Refractory Neuroblastoma Relapsed Neuroblastoma High-risk Neuroblastoma Retinoblastoma Metastatic Retinoblastoma

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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 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.

Official Title

Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma

Quick Facts

Study Start:2023-05-23

Study Completion:2030-01-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05650749

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:1 Year

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Patients must be ≥ 1 year of age 2. 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 also eligible. 3. Patients must have a previously histologically confirmed diagnosis of neuroblastoma: 1. That is recurrent/relapsed or refractory/persistent according to INRC (see Section 21.1) AND 2. For which standard curative measures do not exist or are no longer effective. 3. patients at first relapse are eligible as no known curative therapies exist for relapsed high-risk neuroblastoma. 4. Patients must have evaluable or measurable disease at enrollment. 5. In addition, patient must have experienced at least one of the following: 6. Patients must have a Lansky (≤ 16 years) or Karnofsky (\> 16 years) score of ≥ 60 (See Table 13) 7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age. 8. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease) 9. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases). 10. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases). 11. 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. 12. 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.	1. Patients with active hepatitis B or active hepatitis C. 2. Patients with HIV infection. 3. Patients with uncontrolled active infection. 4. Patients with primary or acquired immunodeficiency disorder. 5. Patients with a known hypersensitivity to DMSO. 6. 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 immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well. 7. 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) 8. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and/or neurotoxicity. 9. 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 prior to study entry or a history of myocarditis. 10. Patients who have received any live vaccines within 30 days prior to enrollment. 11. Patients who are pregnant or nursing (lactating). 12. Patients who have a life expectancy \< 6 months at time of consent.

Inclusion Criteria

Exclusion Criteria

1. Patients must be ≥ 1 year of age
2. 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 also eligible.
3. Patients must have a previously histologically confirmed diagnosis of neuroblastoma:
1. That is recurrent/relapsed or refractory/persistent according to INRC (see Section 21.1) AND
2. For which standard curative measures do not exist or are no longer effective.
3. patients at first relapse are eligible as no known curative therapies exist for relapsed high-risk neuroblastoma.
4. Patients must have evaluable or measurable disease at enrollment.
5. In addition, patient must have experienced at least one of the following:
6. Patients must have a Lansky (≤ 16 years) or Karnofsky (\> 16 years) score of ≥ 60 (See Table 13)
7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age.
8. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)
9. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).
10. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).
11. 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.
12. 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.

1. Patients with active hepatitis B or active hepatitis C.
2. Patients with HIV infection.
3. Patients with uncontrolled active infection.
4. Patients with primary or acquired immunodeficiency disorder.
5. Patients with a known hypersensitivity to DMSO.
6. 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 immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.
7. 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)
8. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and/or neurotoxicity.
9. 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 prior to study entry or a history of myocarditis.
10. Patients who have received any live vaccines within 30 days prior to enrollment.
11. Patients who are pregnant or nursing (lactating).
12. Patients who have a life expectancy \< 6 months at time of consent.

Contacts and Locations

Study Contact

CART Nurse Navigator

CONTACT

445-942-5891

CARTNurseNavigator@chop.edu

Melissa Varghese, M.S.

CONTACT

8455535358

varghesem@chop.edu

Principal Investigator

Lisa Wray, MD

PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Study Locations (Sites)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: Stephan Grupp MD PhD

Lisa Wray, MD, PRINCIPAL_INVESTIGATOR, Children's Hospital of Philadelphia

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 Date2023-05-23

Study Completion Date2030-01-30

Study Record Updates

Study Start Date2023-05-23

Study Completion Date2030-01-30

Terms related to this study

Additional Relevant MeSH Terms

Refractory Neuroblastoma
Relapsed Neuroblastoma
High-risk Neuroblastoma
Retinoblastoma
Metastatic Retinoblastoma