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CAR T-cell Therapy

CAR T-Cell Therapy for Neuroblastoma (GRAIN Trial)

Phase 1
Waitlist Available
Led By Andras A. Heczey, MD
Research Sponsored by Baylor College of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
PROCUREMENT
Karnofsky/Lansky score of 60% or greater
Must not have
Rapidly progressive disease
Evidence of tumor potentially causing airway obstruction
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 15 years
Awards & highlights
No Placebo-Only Group

Summary

This trial involves treating patients with relapsed or refractory neuroblastoma using specially modified immune cells that can better recognize and attack their cancer. These modified cells are supported by chemotherapy and an additional drug to help them work more effectively. The goal is to see if this approach can safely extend the time these cells stay active in the body and improve their ability to fight cancer.

Who is the study for?
This trial is for children with high-risk neuroblastoma that's come back or didn't respond to treatment. They should have normal liver and kidney function, be stable after previous treatments, not have certain allergies, and must not be on immunosuppressive drugs. A key requirement is having T-cells modified to fight cancer ready for infusion.
What is being tested?
The study tests a new gene therapy using T cells engineered to target neuroblastoma more effectively by living longer in the body. It includes lymphodepletion chemotherapy (cyclophosphamide and fludarabine) followed by these special T cells plus pembrolizumab to counteract substances released by tumors that weaken immune responses.
What are the potential side effects?
Possible side effects include reactions from the genetically modified T cells or chemotherapy like fatigue, nausea, low blood counts leading to infection risk; pembrolizumab can cause immune-related issues such as inflammation of organs but varies among patients.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can care for myself but may need occasional help.
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My kidney function tests are within acceptable limits.
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Pembrolizumab is available for my treatment.
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My neuroblastoma is high risk and has either come back or not gone away.
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My T-cells are modified to target my cancer effectively.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My cancer is growing quickly.
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My tumor might block my airways.
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I am currently taking drugs that suppress my immune system.
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I had a severe reaction to a PD-1 inhibitor like pembrolizumab.
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I had severe side effects from cyclophosphamide or fludarabine.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~15 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 15 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Dose limiting toxicities at 6 weeks post T cell infusion
Secondary study objectives
Change in serum cytokine and chemokine levels
Time to progression of disease
To evaluate the expansion and persistence of 3rd generation iC9-GD2 T cells

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: iC9-GD2 T cells,Cytoxan,Fludara,KeytrudaExperimental Treatment4 Interventions
Fresh T cells will be given IV over 5-10 mins. There is a possibility for additional doses of iC9-GD2 T cells.
Group II: iC9-GD2 T Cells - frozen - CLOSEDExperimental Treatment1 Intervention
The cells will be given IV over 5-10 minutes. There is a possibility for additional doses of iC9-GD2 T cells.
Group III: iC9-GD2 T Cells - fresh - CLOSEDExperimental Treatment1 Intervention
The cells will be given IV over 5-10 minutes. There is a possibility for additional doses of iC9-GD2 T cells.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cytoxan
2007
Completed Phase 3
~1460
Fludara
2017
Completed Phase 2
~30
Keytruda
2016
Completed Phase 1
~110

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Genetically modified T cells targeting GD2, such as iC9-GD2-CD28-OX40 T cells, are designed to recognize and kill Neuroblastoma cells by expressing chimeric antigen receptors (CARs) specific to the GD2 protein. Enhancements like CD28 and OX40 are incorporated to increase the T cells' longevity and efficacy. Lymphodepletion with chemotherapy and immune checkpoint inhibitors like pembrolizumab are also used to improve the persistence and effectiveness of these CAR T cells. This targeted approach is crucial for Neuroblastoma patients as it aims to more effectively eliminate cancer cells, potentially prolonging the time to disease progression and improving overall treatment outcomes.
The State of Cellular Adoptive Immunotherapy for Neuroblastoma and Other Pediatric Solid Tumors.Neuroblastoma: molecular pathogenesis and therapy.Surgical excision combined with autologous whole tumor cell vaccination is an effective therapy for murine neuroblastoma.

Find a Location

Who is running the clinical trial?

Baylor College of MedicineLead Sponsor
1,025 Previous Clinical Trials
6,029,771 Total Patients Enrolled
13 Trials studying Neuroblastoma
312 Patients Enrolled for Neuroblastoma
Center for Cell and Gene Therapy, Baylor College of MedicineOTHER
112 Previous Clinical Trials
2,847 Total Patients Enrolled
11 Trials studying Neuroblastoma
270 Patients Enrolled for Neuroblastoma
The Methodist Hospital Research InstituteOTHER
286 Previous Clinical Trials
81,755 Total Patients Enrolled
3 Trials studying Neuroblastoma
152 Patients Enrolled for Neuroblastoma
Solving Kids' CancerOTHER
9 Previous Clinical Trials
122 Total Patients Enrolled
3 Trials studying Neuroblastoma
25 Patients Enrolled for Neuroblastoma
The Evan FoundationOTHER
4 Previous Clinical Trials
227 Total Patients Enrolled
4 Trials studying Neuroblastoma
227 Patients Enrolled for Neuroblastoma
National Cancer Institute (NCI)NIH
13,938 Previous Clinical Trials
41,023,159 Total Patients Enrolled
206 Trials studying Neuroblastoma
53,556 Patients Enrolled for Neuroblastoma
Kids Cancer Research FoundationOTHER
Andras A. Heczey, MDPrincipal InvestigatorBaylor College of Medicine
2 Previous Clinical Trials
18 Total Patients Enrolled
2 Trials studying Neuroblastoma
18 Patients Enrolled for Neuroblastoma

Media Library

iC9-GD2-CD28-OX40 T Cells (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01822652 — Phase 1
Neuroblastoma Research Study Groups: iC9-GD2 T Cells - fresh - CLOSED, iC9-GD2 T Cells - frozen - CLOSED, iC9-GD2 T cells,Cytoxan,Fludara,Keytruda
Neuroblastoma Clinical Trial 2023: iC9-GD2-CD28-OX40 T Cells Highlights & Side Effects. Trial Name: NCT01822652 — Phase 1
iC9-GD2-CD28-OX40 T Cells (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01822652 — Phase 1
~1 spots leftby Dec 2025