RECRUITING

PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma

Conditions

Refractory Neuroblastoma Relapsed Neuroblastoma High-Risk Neuroblastoma Neuroblastoma PHOX2B PC-CAR T Cells Cell Therapy

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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 PHOX2B PC-CAR T cells in patients with advanced, high-risk neuroblastoma.

Official Title

Phase 1 Trial of PHOX2B Peptide-Centric Chimeric Antigen Receptor Autologous T Cells (PHOX2B PC-CAR T) for Relapsed Neuroblastoma

Quick Facts

Study Start:2025-06-20

Study Completion:2035-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07007117

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 years of age * 2. Patients must demonstrate expression of at least one of the following HLA alleles by HLA genotyping (conducted at CHOP) to be eligible. 3. 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. 4. 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. 5. Patients must have evaluable or measurable disease at enrollment. 5. The 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. 7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age: Age Male Female 12 months to \< 2 years 0.6 0.6 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1.0 1.0 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4 * 16 years 1.7 1.4 8. Liver Function as follows: 1. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease or liver metastases). 2. Alanine aminotransferase (ALT) ≤ 3.0 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases). 3. Aspartate aminotransferase (AST) ≤ 3.0 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases). 9. Pulmonary Function as follows: 8. Cardiac Function as follows: 10. Patients of child-bearing potential ( patients who have reached menarche and have not experienced treatment-related premature ovarian failure) must have a negative serum pregnancy test performed at the time of screening It is recommended that all patients of reproductive potential use at least one medically acceptable form of contraception for at least 1 year after their last infusion of PHOX2B PC-CAR T cells. Investigators shall counsel patients on the importance of pregnancy prevention and the implications of an unexpected pregnancy.	* 1. Patients with active hepatitis B or active hepatitis C. 2. Patients with active HIV infection (patients undergoing anti-retroviral therapy with undetectable HIV viral load are eligible). 3. Patients with uncontrolled active infection 4. Patients with primary or acquired immunodeficiency disorder. 5. 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. 6. Patients with actively progressing CNS metastases, including parenchymal or leptomeningeal involvement. (Note: CNS imaging at screening is only required if there is a clinical indication of suspected CNS metastasis) 7. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and/or neurotoxicity. 8. Patients who have received any live vaccines within 30 days prior to enrollment. 9. Pregnant or nursing (lactating) patients.

Inclusion Criteria

Exclusion Criteria

* 1. Patients must be ≥ 1 years of age
* 2. Patients must demonstrate expression of at least one of the following HLA alleles by HLA genotyping (conducted at CHOP) to be eligible.
3. 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.
4. 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.
5. Patients must have evaluable or measurable disease at enrollment. 5. The 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.
7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age: Age Male Female 12 months to \< 2 years 0.6 0.6 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1.0 1.0 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4
* 16 years 1.7 1.4
8. Liver Function as follows:
1. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease or liver metastases).
2. Alanine aminotransferase (ALT) ≤ 3.0 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).
3. Aspartate aminotransferase (AST) ≤ 3.0 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).
9. Pulmonary Function as follows:
8. Cardiac Function as follows:
10. Patients of child-bearing potential ( patients who have reached menarche and have not experienced treatment-related premature ovarian failure) must have a negative serum pregnancy test performed at the time of screening It is recommended that all patients of reproductive potential use at least one medically acceptable form of contraception for at least 1 year after their last infusion of PHOX2B PC-CAR T cells. Investigators shall counsel patients on the importance of pregnancy prevention and the implications of an unexpected pregnancy.

* 1. Patients with active hepatitis B or active hepatitis C. 2. Patients with active HIV infection (patients undergoing anti-retroviral therapy with undetectable HIV viral load are eligible).
3. Patients with uncontrolled active infection 4. Patients with primary or acquired immunodeficiency disorder. 5. 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.
6. Patients with actively progressing CNS metastases, including parenchymal or leptomeningeal involvement. (Note: CNS imaging at screening is only required if there is a clinical indication of suspected CNS metastasis) 7. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and/or neurotoxicity.
8. Patients who have received any live vaccines within 30 days prior to enrollment.
9. Pregnant or nursing (lactating) patients.

Contacts and Locations

Study Contact

Melissa Varghese, B.A.

CONTACT

845-553-5358

varghesem@chop.edu

Principal Investigator

Jacquelyn Crane, MD

STUDY_DIRECTOR

Children's Hospital of Philadelphia

Stephan A. Grupp, MD, PhD

STUDY_DIRECTOR

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

Jacquelyn Crane, MD, STUDY_DIRECTOR, Children's Hospital of Philadelphia
Stephan A. Grupp, MD, PhD, STUDY_DIRECTOR, 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 Date2025-06-20

Study Completion Date2035-06-30

Study Record Updates

Study Start Date2025-06-20

Study Completion Date2035-06-30

Terms related to this study

Keywords Provided by Researchers

Neuroblastoma
PHOX2B PC-CAR T Cells
Cell Therapy

Additional Relevant MeSH Terms

Refractory Neuroblastoma
Relapsed Neuroblastoma
High-Risk Neuroblastoma