Gene Modified Allogeneic Neuroblastoma Cells For Treatment of Relapsed/Refractory Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00186862
Recruitment Status : Completed
First Posted : September 16, 2005
Last Update Posted : June 3, 2008
Texas Children's Cancer Center
National Cancer Institute (NCI)
Information provided by:
St. Jude Children's Research Hospital

Brief Summary:

Neuroblastoma affects approximately 500 children a year in the United States. When the tumor occurs in infants, it is frequently localized and responds well to therapy. Even disseminated disease can be eradicated in about 75% of infants, and indeed may undergo spontaneous remission. In older children, the prognosis is far worse, and 80% or more of those with disseminated tumor can be expected to relapse within 3 years.

This study will utilize the concept of exploiting the immune system to eradicate neuroblastoma. In tumors in which there is consistent expression of tumor specific antigens as part of the malignant process, it may be possible to generate immune T-cells ex-vivo or in-vivo by using the specific protein or peptide(s) derived therefrom and eradicate the tumor. This study will evaluate the use of four to eight injections of IL-2 gene-transduced autologous neuroblastoma cells to induce a local, polyclonal T-cell infiltrate as well as an anti-tumor immune response.

Condition or disease Intervention/treatment Phase
Neuroblastoma Drug: Interleukin-2 Phase 1

Detailed Description:

Secondary objectives for this protocol included the following:

  • To determine whether major histocompatibility complex (MHC) restricted or unrestricted antitumor immune responses are induced by injection of modified allogeneic neuroblasts and the cell doses required to produce these effects.
  • To obtain preliminary data on the antitumor effects of this treatment regimen.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Chemokine and Cytokine Gene Modified Allogeneic Neuroblastoma Cells For Treatment of Relapsed/Refractory Neuroblastoma Using a Retroviral Vector
Study Start Date : August 1998
Actual Primary Completion Date : April 2000
Actual Study Completion Date : October 2007

Arm Intervention/treatment
1 Drug: Interleukin-2

A genetically modified (retroviral) allogeneic tumor vaccine coupled with the human interleukin-2. Patients were treated with 4 injections of these gene-modified tumor cells. The first two were given at weeks 1 and 2. Patients then had a 2 week rest and the remaining 2 injections were given at weeks 4 and 5. A complete evaluation for evidence of toxicity and response were performed at week 8.

At this week 8 evaluation, if there was no excessive toxicity, progressive disease requiring therapy, and if more transduced cells are available, patients had the option to receive 4 additional injections. These additional injections were separated by 1 month at the higher of the two dosage levels originally received.

Other Name: Immunotherapy; gene transfer

Primary Outcome Measures :
  1. • To determine the safety of up to eight subcutaneous injections of allogeneic neuroblastoma cells that have been genetically modified by retroviral vectors to secrete lymphotactin and Interleukin-2 [ Time Frame: 1 year ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Diagnosis of recurrent advanced stage neuroblastoma.
  • Must have a life expectancy of at least 8 weeks.
  • Must have recovered from the toxic effects of all prior chemotherapy before entering this study, and have an absolute neutrophil count of >500/mm3.
  • Not be currently receiving any investigational agents or have not received any tumor vaccines within the previous six months.
  • Bilirubin <1.5 mg/dl.
  • Creatinine <1.5 mg/dl.
  • ECOG performance status of 0-2 as below:
  • Does not have rapidly progressive disease.
  • Not pregnant or lactating.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00186862

United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Texas Children's Cancer Center
National Cancer Institute (NCI)
Principal Investigator: Gregory A Hale, MD St. Jude Children's Research Hospital

Additional Information:
Responsible Party: Gregory Hale, MD, St. Jude Children's Research Hospital Identifier: NCT00186862     History of Changes
Other Study ID Numbers: CYCHAL
First Posted: September 16, 2005    Key Record Dates
Last Update Posted: June 3, 2008
Last Verified: June 2008

Keywords provided by St. Jude Children's Research Hospital:
Gene therapy

Additional relevant MeSH terms:
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Antineoplastic Agents
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs