Refractory Neuroblastoma Clinical Trial
— GPC2Official title:
Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma
This is a first in human dose escalation trial to determine the safety of administering GPC2 CAR T cells in patients with advanced neuroblastoma.
Status | Recruiting |
Enrollment | 30 |
Est. completion date | January 30, 2026 |
Est. primary completion date | January 30, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year and older |
Eligibility | Inclusion Criteria: 1. Signed Informed Consent Form 2. = 1 year of age 3. Disease status 1. Patients must have high-risk neuroblastoma according to Children's Oncology Group risk classification at the time of study enrollment. 2. Histologically confirmed diagnosis of neuroblastoma that is recurrent/relapsed/persistent according to International Neuroblastoma Response Criteria 3. Patients must have evaluable or measurable disease at enrollment 4. Adequate organ function 5. Adequate performance status defined as Lanksy or Karnofsky performance score =60. 6. Subjects of reproductive potential must agree to use acceptable birth control methods. Exclusion Criteria: 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. 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 Central Nervous System metastases, including parenchymal or leptomeningeal involvement. 7. Active medical disorder that, in the opinion of the investigator, would substantially increase. the risk of uncontrollable Cytokine Release Syndrome and/or neurotoxicity. 8. 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. 9. Patients who have received any live vaccines within 30 days prior to enrollment. 10. Pregnant or nursing (lactating) women. |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Stephan Grupp MD PhD | Children's Hospital of Philadelphia, Tmunity Therapeutics, University of Pennsylvania |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Determine the Maximum Tolerated Dose of GPC2 CAR T cells | The Maximum Tolerated Dose of GPC2 CAR T cells will be determined by measuring the incidence of dose limiting toxicities following administration of the product. | 5 years | |
Primary | Frequency of Adverse Events Following GPC2 CAR T cell administration | Assess the frequency and severity of treatment related adverse events following administration of GPC2 CAR T cells. | 5 years | |
Secondary | Manufacturing Feasibility of GPC2 CAR T cells | Manufacturing Feasibility will be evaluated as the Percentage of patients with GPC2 CAR T cell products that meet release criteria | 5 years | |
Secondary | Persistence of GPC2 CAR T cells | Persistence of GPC2 CAR T cells will be measured by Polymerase Chain Reaction (or flow cytometry) analysis of whole blood to detect and quantify survival of GPC2 CAR T cells over time. | 5 years | |
Secondary | Preliminarily define the clinical activity of GPC2 CAR T in patients with relapsed or refractory neuroblastoma | Overall Response Rate will be determined based on international Neuroblastoma Response Criteria | 5 years | |
Secondary | Severity of Adverse Events Following GPC2 CAR T cell administration. | The safety of GPC2 CAR T cell therapy reinfusions will be measured by the monitoring the frequency and severity of adverse events after multipleGPC2 CAR T cells infusions. | 5 years |
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