3RD GENERATION GD2 SPECIFIC CHIMERIC ANTIGEN RECEPTOR TRANSDUCED AUTOLOGOUS NATURAL KILLER T-CELLS FOR NEUROBLASTOMA

Summary

This research study is for patients that have a cancer called Neuroblastoma that has either come back after treatment or did not respond to the standard medicines used to treat it. This study combines two different ways of fighting cancer: antibodies and Natural Killer T cells. Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting 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. The 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, the investigators made a gene called a chimeric antigen receptor (CAR), from an antibody called 14g2a that recognizes GD2, a molecule found on almost all neuroblastoma cells (GD2-CAR). They put this gene into the patients' own T cells and gave them back to patients that had neuroblastoma. Nineteen patients were treated on that study and there were no long term side-effects seen after the GD2 T cell infusion. As the investigators have followed the patients over time, they noticed that for those patients with disease at the time of their infusion, the time to progression (the amount of time it takes before their neuroblastoma got worse) was longer in those whom they could find GD2 T cells in the blood for more than 6 weeks after the last T cell infusion. Because of this, the investigators think that if effector cells are able to last longer, they may have a better chance of killing neuroblastoma tumor cells.

Natural Killer T cells are a special subset of innate lymphocytes that can effectively go into tumor tissues of neuroblastoma. Inside the tumor, there are certain white blood cells which help the cancer cells to grow and recover from injury. Natural Killer T-cells can specifically kill these cells. In this study, Natural Killer T cells will be genetically engineered to express GD2-CAR to attack neuroblastoma cells and the white blood cells inside the tumor tissue.

Conditions

• Neuroblastoma

Interventions

DRUG

cyclophosphamide

500 mg/m2/day x 2 days; for patients \<12 kg the dose is 16.7 mg/kg/day x 2 days; Cyclophospamide will be given daily starting Day -4 prior to administration of GINAKIT cells.

DRUG

Fludarabine

30 mg/m2/day x 3 days; for patients \<12 kg the dose is 1 mg/kg/day x 3 days; Fludarabine will be given daily starting Day -4 prior to administration of GINAKIT cells

GENETIC

GINAKIT Cells

* Dose Level 1 = 3 x 10\^6 * Dose Level 2 = 1 x 10\^7 * Dose Level 3 = 3 x 10\^7 * Dose Level 4 = 1 x 10\^8 After the end of the 4 week evaluation period, if subjects have not had a severe side effects and if disease has not gotten worse, the subjects may be offered additional doses of the same cell dose in the future.

Sponsors & Collaborators

• Center for Cell and Gene Therapy, Baylor College of Medicine

  collaborator OTHER

• Alex's Lemonade Stand Foundation

  collaborator INDUSTRY

• Baylor College of Medicine

  lead OTHER

Principal Investigators

• Andras A Heczey, MD · Baylor College of Medicine - Texas Children's Hospital

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

1 Year

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-08-31

Primary Completion

2019-09-30

Completion

2030-10-31

FDA Drug

Yes

Countries

• United States

Study Locations