54 Participants Needed

GINAKIT Cells for Neuroblastoma

(GINAKIT2 Trial)

AH
JR
RS
Overseen ByRamy Sweidan
Age: < 65
Sex: Any
Trial Phase: Phase 1
Sponsor: Baylor College of Medicine
Must be taking: Etanercept
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

What is the purpose of this trial?

This research study combines two different ways of fighting cancer: antibodies and Natural Killer T cells (NKT). Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special white blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. Investigators have found from previous research that they can put a new gene into T cells that will make them recognize cancer cells and kill them. In a previous clinical trial, investigators made artificial genes called a chimeric antigen receptors (CAR), from an antibody called 14g2a that recognizes GD2, a molecule found on almost all neuroblastoma cells (GD2-CAR). Investigators put these genes into the patients' own T cells and gave them back to patients that had neuroblastoma. NKT cells are another special subgroup of white blood cells that can specifically go into tumor tissue of neuroblastoma. Inside the tumor, there are other white blood cells called macrophages which help the cancer cells to grow and recover from injury. NKT cells can specifically kill these macrophages and slow the tumor growth. We will expand NKT cells and add GD2-specific chimeric antigen receptors to the cells. We think these cells might be better able to attack NB since they also work by destroying the macrophages that allows the tumor to grow. The chimeric antigen receptor will also contain a gene segment to make the NKT cells last longer. This gene segment is called CD28. In addition, to further improve the antitumor activity of the GINAKIT cells we added another gene expressing a molecule called Interleukin -15 (IL-15). The combination of these 3 components showed the most antitumor activity by CAR expressing NKT cells and improved these cells' survival in animal models. We also found that a medicine called ETANercept can slow down neuroblastoma growth, which might enhance the effects of the modified cells. In this part of our study, we aim to treat children with hard-to-treat neuroblastoma using these modified NKT cells along with ETANercept. Though ETANercept has been used to treat other diseases, such as rheumatoid arthritis in children, there is limited information about the safety, efficacy, and risk of ETANercept treatment in combination with cellular therapies. GD2-CAR expressing NKTs have not been tested in patients so far. The purpose of this study is to find the largest effective and safe dose of GD2-CAR NKT cells (GINAKIT cells), to evaluate their effect on the tumor and how long they can be detected in the patient's blood and what affect they have on the patient's neuroblastoma.

Will I have to stop taking my current medications?

The trial requires that you are not currently taking immunosuppressive drugs like corticosteroids, tacrolimus, or cyclosporine, unless the corticosteroid dose is less than 0.5mg/kg/day of prednisone equivalent. If you are on these medications, you may need to stop or adjust them before participating.

What data supports the effectiveness of the GINAKIT Cells treatment for neuroblastoma?

Research shows that combining invariant natural killer T (iNKT) cells with anti-GD2 antibodies can enhance the body's immune response against neuroblastoma, potentially improving treatment outcomes. Additionally, similar therapies using GD2-targeted approaches have shown promise in treating neuroblastoma, suggesting that GINAKIT Cells could be effective.12345

Is GINAKIT Cells treatment safe for humans?

Research shows that GD2-CAR NKT cells, similar to GINAKIT Cells, have been tested in humans with neuroblastoma and are generally safe. In a study, no severe dose-limiting toxicities were observed, and only mild side effects like grade 2 cytokine release syndrome occurred, which were manageable.12467

How is the GINAKIT Cells treatment different from other neuroblastoma treatments?

GINAKIT Cells treatment is unique because it uses genetically engineered natural killer T cells that specifically target the GD2 marker on neuroblastoma cells, enhancing the immune system's ability to attack the cancer without the high toxicity seen in other treatments. This approach combines the benefits of targeted immunotherapy with the natural cancer-fighting abilities of the immune system.13489

Research Team

AH

Andras Heczey, MD

Principal Investigator

Baylor College of Medicine

Eligibility Criteria

This trial is for children and young adults (1-21 years old) with high-risk neuroblastoma that has relapsed or not responded to treatment. They must weigh over 12kg, have a life expectancy of at least 12 weeks, and be able to undergo leukocyte apheresis. Their kidney function, blood counts, liver enzymes, and heart/lung health should meet specific criteria. Those who are pregnant or breastfeeding, have HIV infection or severe disease progression cannot participate.

Inclusion Criteria

My white blood cell count is healthy without needing medication for at least 2 days.
My blood cell counts meet the required levels for treatment.
My treatment involves NKT cells modified to target my cancer more effectively.
See 14 more

Exclusion Criteria

You have had an allergic reaction to products containing murine protein in the past.
My tumor is blocking my airways.
I had severe side effects from cyclophosphamide or fludarabine.
See 6 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Lymphodepletion

Participants receive cyclophosphamide and fludarabine intravenously for 2 days and fludarabine alone for one more day before GINAKIT cell infusion.

3 days
In-patient stay for chemotherapy administration

Treatment

Participants receive GINAKIT cells infusion and ETANercept subcutaneously once per week for 5 doses.

5 weeks
Weekly visits for ETANercept administration

Initial Follow-up

Participants are monitored for side effects and tumor response with follow-up visits at weeks 1, 2, 3, 4, and 8.

8 weeks
Weekly visits for monitoring

Long-term Follow-up

Participants are monitored for long-term safety and effectiveness with visits every 3 months for the first year, every 6 months for the next 4 years, and then annually for the next 10 years.

15 years
Regular follow-up visits

Treatment Details

Interventions

  • GINAKIT Cells
Trial OverviewThe study tests GINAKIT cells in patients with neuroblastoma. These cells are the patient's own Natural Killer T (NKT) cells genetically modified to express GD2-CAR targeting cancer cells and IL-15 for improved survival. The goal is to find the highest safe dose that can effectively target the tumor.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: GINAKIT cellsExperimental Treatment1 Intervention
GINAKIT cells will be administer to patients with Neuroblastomas on Day 0.
Group II: Experimental: GINAKIT cells + Etanercept.Experimental Treatment1 Intervention
GINAKIT cells will be administer to patients with Neuroblastomas on Day 0 in combination of Etanercept.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Baylor College of Medicine

Lead Sponsor

Trials
1,044
Recruited
6,031,000+

Center for Cell and Gene Therapy, Baylor College of Medicine

Collaborator

Trials
114
Recruited
2,900+

Findings from Research

Combining activated invariant natural killer T (iNKT) cells with anti-GD2 antibodies enhances the effectiveness of the treatment for neuroblastoma by increasing the cytotoxicity of natural killer (NK) cells, which is crucial for fighting cancer.
This combination therapy works by upregulating important cytokines like granzyme A, granzyme B, and interferon gamma (IFNγ), suggesting that iNKT cells can improve the immune response against neuroblastoma while potentially minimizing toxicity.
Antibody-dependent cellular cytotoxicity toward neuroblastoma enhanced by activated invariant natural killer T cells.Mise, N., Takami, M., Suzuki, A., et al.[2021]
The combination of dinutuximab beta (DB) and subcutaneous interleukin-2 (scIL2) after haploidentical stem cell transplantation (haplo-SCT) in patients with relapsed high-risk neuroblastoma showed a 54.4% success rate, indicating it is a feasible treatment option.
Long-term outcomes were promising, with a 5-year event-free survival rate of 43% and overall survival rate of 53%, particularly among patients in complete or partial remission before immunotherapy, suggesting enhanced antineuroblastoma activity from donor-derived immune cells.
Anti-GD2 Antibody Dinutuximab Beta and Low-Dose Interleukin 2 After Haploidentical Stem-Cell Transplantation in Patients With Relapsed Neuroblastoma: A Multicenter, Phase I/II Trial.Flaadt, T., Ladenstein, RL., Ebinger, M., et al.[2023]
IL-2 significantly enhances the effectiveness of anti-GD2 antibodies in promoting antibody-dependent cell-mediated cytotoxicity (ADCC) against neuroblastoma cells, potentially allowing for lower doses of antibodies and reduced toxicity.
The study identified that licensed NK cells with specific receptor mismatches against tumor HLA showed the highest degranulation and cytotoxic activity, suggesting that individual variations in NK cell characteristics could influence treatment outcomes.
The role of interleukin-2, all-trans retinoic acid, and natural killer cells: surveillance mechanisms in anti-GD2 antibody therapy in neuroblastoma.Nguyen, R., Houston, J., Chan, WK., et al.[2022]

References

Antibody-dependent cellular cytotoxicity toward neuroblastoma enhanced by activated invariant natural killer T cells. [2021]
Anti-GD2 Antibody Dinutuximab Beta and Low-Dose Interleukin 2 After Haploidentical Stem-Cell Transplantation in Patients With Relapsed Neuroblastoma: A Multicenter, Phase I/II Trial. [2023]
The role of interleukin-2, all-trans retinoic acid, and natural killer cells: surveillance mechanisms in anti-GD2 antibody therapy in neuroblastoma. [2022]
Antitumor activity without on-target off-tumor toxicity of GD2-chimeric antigen receptor T cells in patients with neuroblastoma. [2021]
Murine anti-GD2 monoclonal antibody 3F8 combined with granulocyte-macrophage colony-stimulating factor and 13-cis-retinoic acid in high-risk patients with stage 4 neuroblastoma in first remission. [2021]
Choice of costimulatory domains and of cytokines determines CAR T-cell activity in neuroblastoma. [2021]
Anti-GD2 CAR-NKT cells in relapsed or refractory neuroblastoma: updated phase 1 trial interim results. [2023]
Disialoganglioside-specific human natural killer cells are effective against drug-resistant neuroblastoma. [2021]
TH-MYCN tumors, but not tumor-derived cell lines, are adrenergic lineage, GD2+, and responsive to anti-GD2 antibody therapy. [2023]