Using Gene Modified Neuroblastoma Cells for the Treatment of Relapsed/Refractory Neuroblastoma

NCT00062855 | Completed Phase 1 DMC

• 2 other identifiers: H6442Cyche
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This research study is designed to determine the safety and dosage of special cells that may make a patients own immune system fight the cancer. To do this we will put two special genes into cancer cells taken from the patients body. The genes we put in make the cancer cells produce lymphotactin, a natural substance that attracts immune system cells to the cancer, and IL-2 a natural substance that may help the immune system kill cancer cells. Some of these cells will then be put back in the patient's body. Studies of cancers in animals and in cancer cells that are grown in laboratories suggest that substances like lymphotactin and IL-2 help the body kill cancer cells. A treatment similar to this has been used in ten children previously and similar treatments are being used in adults with other cancers. The purpose of this study is to learn the side effects and safe 'dosage' of these special cells.

Conditions

Neuroblastoma

Keywords

GENE MODIFIED AUTOLOGOUS NEUROBLASTOMA CELLS; Gene Therapy

Outcome Measures

Primary Outcomes (2)

• To determine the safety of up to four subcutaneous (SC) injections of autologous neuroblastoma cells which have been genetically modified by adenoviral vectors to secrete lymphotactin (Lptn) and Interleukin-2 (IL-2).

  2 months

• To determine the safety of up to eight (total) of these injections in patients who have received the first four injections without unacceptable toxicity and have evidence of stable disease or better after receiving these injections.

  6 months

Secondary Outcomes (2)

• To determine whether MHC restricted or unrestricted antitumor immune responses are induced by SC injection of modified autologous neuroblasts and the cell doses required to produce these effects.

  3 weeks

• To obtain preliminary data on the antitumor effects of this treatment regimen

  8 weeks

Study Arms (1)

Gene Modified Neuroblastoma Cells

EXPERIMENTAL

Gene modified neuroblastoma cells given as 4 subcutaneous injections over 5 weeks

Procedure: Skin Biopsy; Genetic: Gene Modified Neuroblastoma Cells

Interventions

Skin Biopsy PROCEDURE

Gene Modified Neuroblastoma Cells

Gene Modified Neuroblastoma Cells GENETIC

The first two injections will be given at week 1 and week 2 (i.e., separated by one week). Patients will then have a two-week rest and the remaining two injections will be given (again separated by one week) at week 4 and week 5. A complete evaluation for evidence of toxicity and response will be performed at week 8 (after a 3 week rest). At the 8 week (month 2) evaluation, in the absence of progressive disease requiring therapy without excessive toxicity and if more transduced cells are available, the patient will have the option to receive four additional SC injections each separated by 1 month at the higher of the two dosage levels they originally received.

Gene Modified Neuroblastoma Cells

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

* All patients under 21 years of age at diagnosis with recurrent, advanced stage neuroblastoma. * Patients must have a life expectancy of at least 8 weeks. * Patients must have recovered from the toxic effects of all prior chemotherapy before entering this study, and have an absolute lymphocyte and neutrophil count of \>500/mm3 each. * Patients must not be currently receiving any investigational agents or have not received any tumor vaccines within the previous six weeks. * Patients must not be HIV-positive. * Patients must have bilirubin \<1.5 mg%. * Patients must have creatinine \<1.5 mg/dl. * Patients must have ECOG performance status of 0-2. * Patients must have autologous transduced neuroblastoma cells available that are demonstrably producing \>150 pg IL-2/106 cells/24 hr and are secreting Lptn. * Patients or legal guardians must sign an informed consent indicating that they are aware this is a research study and have been told of its possible benefits and toxic side effects. Patients or their guardians will be given a copy of the consent form. * Sexually active patients must be willing to utilize one of the more effective birth control methods during the study and for 3 months after the study is concluded. The male partner should use a condom.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

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

Study Sites (1)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Malcolm Brenner, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor, Director Center for Cell and Gene Therapy

Study Record Dates

• First Submitted: June 17, 2003
• First Posted: June 18, 2003
• Study Start: November 1, 1997
• Primary Completion: August 1, 2001
• Study Completion: March 1, 2006
• Last Updated: October 29, 2012

Record last verified: 2012-10

Locations

US (1)