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Neuroblastoma Vaccine for Treatment of High-Risk Neuroblastoma After Chemotherapy (CYCHE2)

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.
 
ClinicalTrials.gov Identifier: NCT00048386
Recruitment Status : Completed
First Posted : November 1, 2002
Last Update Posted : July 27, 2012
Sponsor:
Collaborators:
Baylor College of Medicine
Center for Cell and Gene Therapy, Baylor College of Medicine
Information provided by (Responsible Party):
Malcolm Brenner, Baylor College of Medicine

Tracking Information
First Submitted Date  ICMJE October 30, 2002
First Posted Date  ICMJE November 1, 2002
Last Update Posted Date July 27, 2012
Study Start Date  ICMJE November 1999
Actual Primary Completion Date December 2004   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 20, 2007)
Patients who demonstrate immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete IL-2 [ Time Frame: 12 months post injections ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 20, 2007)
  • To determine the toxicity of the autologous neuroblastoma vaccine given according to this schedule [ Time Frame: 15 years ]
  • To obtain preliminary data on progression-free survival from high-risk neuroblastoma following vaccine administration [ Time Frame: 15 years ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Neuroblastoma Vaccine for Treatment of High-Risk Neuroblastoma After Chemotherapy
Official Title  ICMJE A Pilot Study of Gene Modified Autologous Neuroblastoma Vaccine for the Post-Chemotherapy Treatment of High-Risk Neuroblastoma
Brief Summary

PRIMARY OBJECTIVE To determine the percentage of patients with high risk neuroblastoma in first or subsequent partial response or better, or with microscopic residual bone marrow disease, who demonstrate an immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with subcutaneous injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete interleukin-2 (IL-2) (autologous neuroblastoma vaccine)

SECONDARY OBJECTIVES 1. To determine the toxicity of the autologous neuroblastoma vaccine given according to this schedule 2. To obtain preliminary data on the effect of vaccine administration on progression-free survival from high-risk neuroblastoma

Detailed Description

Tumor cells from subjects with high-risk neuroblastoma will be obtained at the time of presentation and initial diagnosis, during routine surveillance of bone marrow disease status, or at the time of surgical resection of disease during primary chemotherapy. The tumor cells will be irradiated to prevent the possibility of tumor growth. Autologous neuroblastoma tumor cells will be used to treat neuroblastoma following the completion of primary or salvage chemotherapy when peripheral blood lymphocyte counts have recovered to > 500 CD3+ cells/mm3. This preceding chemotherapy may or may not have included bone marrow or peripheral stem cell rescue. Therefore, following the achievement of best response with chemotherapy, vaccine will be administered for 6 months. Vaccine modified for secretion of IL-2 will be used.

Vaccine therapy will commence after complete recovery from the side effects of primary or salvage chemotherapy, as long as the subject has an absolute CD3+ lymphocyte count and an absolute neutrophil count greater than 500/mm3. A vaccine consisting of 0.3 x 10 8 cells/patient will be given per injection, based on data from the Phase I studies. Injections will be given twice monthly for two months, then monthly for four months, for a total of eight vaccine injections over six months. The immune effects of the vaccine, toxicity of treatment, and anti-tumor effects will be periodically assessed. Further evaluation will be done annually.

The first and second vaccine injection sites will be "punch biopsied" one week after the injection. Several x-rays and various types of scans will also be taken to assist with monitoring the status of the neuroblastoma. Complete details of the evaluations required by this study are included in.

Subjects may receive supportive care for any acute or chronic toxicity from the injections, including blood product support, antibiotics, and other appropriate intervention. Pneumocystis carinii prophylaxis initiated during chemotherapy may be continued during vaccine therapy for as long as deemed appropriate by the investigator. As well, subjects may receive concurrent therapy with cis-retinoic acid at the discretion of the treating physician.

An adenoviral vector is being used to transduce the cells ex-vivo. Subjects will not be treated with the viral vector.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Neuroblastoma
Intervention  ICMJE Biological: autologous neuroblastoma vaccine
A vaccine dose of 0.3 x 108 IL-2 secreting cells/patient/injection will be used. Injections will be given twice monthly for two months, then monthly for four months, for a total of eight vaccine injections over six months.
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 20, 2007)
13
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
15
Actual Study Completion Date  ICMJE October 2009
Actual Primary Completion Date December 2004   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

INCLUSION CRITERIA:

  • Patients with high risk neuroblastoma defined below, who, following completion of front-line or salvage chemotherapy that may or may not have included high-dose chemotherapy followed by peripheral blood stem cell or bone marrow rescue with or without cis-retinoic acid, have achieved partial response or better, or who have microscopic residual bone marrow:

    1. INSS Stage 4 neuroblastoma, diagnosed between 1 and 21 years of age (inclusive)
    2. INSS Stage 3, N-myc amplified neuroblastoma, diagnosed between 1 and 21 years of age (inclusive)
    3. INSS Stage 3, N-myc non-amplified, Shimada unfavorable histology neuroblastoma, diagnosed between 1 and 21 years of age (inclusive)
    4. INSS Stages 2A or 2B, N-myc amplified, Shimada unfavorable histology, diagnosed between 1 and 21 years of age (inclusive)
    5. INSS Stage 4 neuroblastoma, with Shimada unfavorable histology, diagnosed under 365 days of age
  • Patients with intermediate or low risk neuroblastoma, who have achieved a second or subsequent partial response or better following chemotherapy treatment of first or subsequent relapse
  • Patients must have recovered from the toxic effects of prior chemotherapy prior to treatment on this study, and must have both an absolute lymphocyte count and an absolute neutrophil count greater than 500/mm3.
  • Patients with disease status outlined in the first bullet who have been treated with allogeneic tumor vaccine are eligible for treatment with autologous tumor vaccine when that product becomes available
  • Patients may not concurrently receive any investigational agents or other tumor vaccines.
  • Patients must be HIV-negative.
  • Female patients must not be pregnant or lactating.
  • Patients must have autologous transduced neuroblastoma cells available that are demonstrably producing > 150 pg IL-2/10e6 cells/24 hours.
  • Patients or legal guardians must sign an informed consent according to institutional guidelines.
  • Patients who are sexually active 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 64 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00048386
Other Study ID Numbers  ICMJE H8354
HIGH RISK NEUROBLASTOMA
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Malcolm Brenner, Baylor College of Medicine
Study Sponsor  ICMJE Malcolm Brenner
Collaborators  ICMJE
  • Baylor College of Medicine
  • Center for Cell and Gene Therapy, Baylor College of Medicine
Investigators  ICMJE
Principal Investigator: Heidi V Russell, MD Baylor College of Medicine
PRS Account Baylor College of Medicine
Verification Date July 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP