CAR T-cell Therapy for Neuroblastoma
Recruiting in Palo Alto (17 mi)
Age: < 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1
Waitlist Available
Sponsor: Seattle Children's Hospital
No Placebo Group
Trial Summary
What is the purpose of this trial?Patients with recurrent or refractory neuroblastoma are resistance to conventional chemotherapy. For this reason, the investigators are attempting to use T cells obtained directly from the patient, which can be genetically modified to express a chimeric antigen receptor (CAR). The CAR enables the T cell to recognize and kill the neuroblastoma cell through the recognition of CD171, a protein expressed of the surface of the neuroblastoma cell in patients with neuroblastoma. This is a phase 1 study designed to determine the maximum tolerated dose of the CAR+ T cells.
Eligibility Criteria
This trial is for children and young adults up to 26 years old with high-risk neuroblastoma or ganglioneuroblastoma that's resistant to standard treatments. They must have a life expectancy of at least 8 weeks, be in relatively stable health, and not have received certain recent therapies like allogeneic stem cell transplants.Inclusion Criteria
My organs are functioning well.
My cancer can be measured or seen on tests.
I was diagnosed with high-risk neuroblastoma or it spread after I turned 18 months.
+13 more
Exclusion Criteria
I have a condition that affects my immune system or bone marrow.
I am currently suffering from a severe infection.
I have a history of or currently have significant brain-related health issues.
+7 more
Participant Groups
The ENCIT-01 study tests different versions of genetically engineered T cells designed to target CD171 on neuroblastoma cells. It aims to find the highest dose patients can tolerate without severe side effects. These T cells are taken from the patient, modified in the lab, then given back to attack the cancer.
3Treatment groups
Experimental Treatment
Group I: C: Long Spacer 2nd Generation CE7R CAR T CellsExperimental Treatment1 Intervention
Autologous CD4 and CD8 cells are lentivirally transduced to generate patient-derived CD171 specific CAR T cells also expressing an EGFRt.
Patients will receive lymphodepletion chemotherapy prior to T cell infusion. CD171 specific CAR T cells will be administered approximately 2-3 days after lymphodepletion chemotherapy.
Cells will be administered approximately 1:1 CD4 and CD8 cells with planned dose level evaluations of total T cell dose of 1x10\^6 cells/kg, 5x10\^6 cells/kg, 1x10\^7 cells/kg, 5x10\^7 cells/kg, and 1x10\^8 cells/kg will be evaluated.
Group II: B: 3rd Generation CE7R CAR T CellsExperimental Treatment1 Intervention
Autologous CD4 and CD8 cells are lentivirally transduced to generate patient-derived CD171 specific CAR T cells also expressing an EGFRt.
Patients will receive lymphodepletion chemotherapy prior to T cell infusion. CD171 specific CAR T cells will be administered approximately 2-3 days after lymphodepletion chemotherapy.
Cells will be administered approximately 1:1 CD4 and CD8 cells with planned dose level evaluations of total T cell dose of 1x10\^6 cells/kg, 5x10\^6 cells/kg, 1x10\^7 cells/kg, 5x10\^7 cells/kg, and 1x10\^8 cells/kg will be evaluated.
Group III: A: 2nd Generation CE7R CAR T CellsExperimental Treatment1 Intervention
Autologous CD4 and CD8 cells are lentivirally transduced to generate patient-derived CD171 specific CAR T cells also expressing an EGFRt.
Patients will receive lymphodepletion chemotherapy prior to T cell infusion. CD171 specific CAR T cells will be administered approximately 2-3 days after lymphodepletion chemotherapy.
Cells will be administered approximately 1:1 CD4 and CD8 cells with planned dose level evaluations of total T cell dose of 1x10\^6 cells/kg, 5x10\^6 cells/kg, 1x10\^7 cells/kg, 5x10\^7 cells/kg, and 1x10\^8 cells/kg will be evaluated.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Seattle Children's HospitalSeattle, WA
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Who Is Running the Clinical Trial?
Seattle Children's HospitalLead Sponsor
Ben Towne Center for Childhood Cancer ResearchCollaborator
The Evan FoundationCollaborator