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Decitabine Followed by a Cancer Antigen Vaccine for Patients With Neuroblastoma and Sarcoma

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: NCT01241162
Recruitment Status : Completed
First Posted : November 16, 2010
Last Update Posted : May 9, 2017
Solving Kids’ Cancer
Information provided by (Responsible Party):
University of Louisville

Brief Summary:
This treatment study for relapsed high-risk neuroblastoma, Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma or synovial sarcoma involves an autologous cancer testis (CT) antigen specific dendritic cell (DC) vaccine preceded by decitabine as a demethylating chemotherapy.

Condition or disease Intervention/treatment Phase
Neuroblastoma Ewings Sarcoma Osteogenic Sarcoma Rhabdomyosarcoma Synovial Sarcoma Biological: Autologous dendritic cell vaccine with adjuvant Phase 1

Detailed Description:
For vaccine production, mature DC will be pulsed with overlapping peptides mixes derived from full-length NY-ESO-1, MAGE-A1, and MAGE-A3.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Trial Combining Decitabine and Vaccine Therapy for Patients With Relapsed Neuroblastoma and Sarcoma.
Study Start Date : August 2010
Primary Completion Date : October 2016
Study Completion Date : October 2016

Arm Intervention/treatment
Experimental: Single arm study
Biological/Vaccine: Autologous dendritic cell vaccine with adjuvant
Biological: Autologous dendritic cell vaccine with adjuvant

Week 1:

Decitabine (DAC): 10 mg/m2/day IV (Mon-Fri)

Weeks 2 and 3:

Vaccine: 3-5 E6 peptide pulsed DC (Mon)

Hiltonol applied topically to vaccine site before and after vaccination

Other Names:
  • Decitabine (trade name Dacogen or 5-aza-2'-deoxycytidine/DAC
  • Hiltonol

Primary Outcome Measures :
  1. Tolerance of study treatment [ Time Frame: 2 years ]
    Tolerance to DAC, at at least 50% dosing, and 3 of the 4 planned vaccinations during the first two cycles.

Secondary Outcome Measures :
  1. Tumor Response [ Time Frame: 2 years ]
    Assessment of tumor responses--complete or partial remission, stable disease, and disease progression--by CT/PET/MIBG or MRI after cycles 2 and 4.

  2. Immune Response [ Time Frame: 2 years ]
    Assessment of post-vaccination T cell responses to MAGE-A1, MAGE-A3, and NY-ESO-1 by immunoassays.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 24 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed diagnosis of relapsed high-risk neuroblastoma,Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma, synovial sarcoma
  • Patient may have gross tumor that has been treated with multi-agent chemotherapy prior study entry, but does not need to have gross tumor prior to study entry.
  • Patients must have had a diagnosis of neuroblastoma or sarcoma either by histological verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
  • Have received standard chemotherapy and/or SCT, and are at least 6 months post-transplant.
  • Age: Patients must be 1 - < 25 years of age when registered on study.
  • Organ Function Requirements: All patients must have adequate organ function defined as:
  • Hematological Function: ANC ≥ 500; Platelet count ≥ 75.
  • Renal Function: Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 OR A maximum serum creatinine (mg/dL) based on age/gender as follows: 1YO M&F = 0.6, 2-5YO M&F = 0.8, 6-9YO M&F = 1, 10-12YO M&F = 1.2, 13-15YO M = 1.5, 13-15YO F = 1.4, 16+ M = 1.7, 16+ F = 1.4
  • Cardiac Function: Patient must have normal cardiac function documented by Ejection fraction (> 55%) documented by echocardiogram or radionuclide MUGA evaluation OR Fractional shortening (≥ 28%) documented by echocardiogram
  • Liver Function: Total bilirubin ≤ 1.5 x normal for age, AND SGPT (ALT) and SGOT (AST) ≤ 3 x normal for age.
  • Room air pulse oximetry >94%.
  • Male and female sexually active patients of reproductive age who wish to participate must agree to use acceptable contraception.
  • Lansky performance scale > 70, ECOG < 2 (Appendix I).

Exclusion Criteria:

  • Patient is pregnant.
  • Patients with a positive result for any of the following diagnostic tests: Hep B Ag, Hep B Core Ab, Hep C Ab, HIV-1 Ab, HIV-2 Ab, HTLV-1 Ab, HTLV-2 Ab, RPR.
  • Patient has a history of autoimmune disease, specifically inflammatory bowel disease, systemic lupus erythematosis, or rheumatoid arthritis.
  • Patient is receiving concurrent systemic steroid therapy.
  • Patient has a known systemic hypersensitivity to DAC, Hiltonol, or any vaccine component.

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): NCT01241162

United States, Kentucky
University of Louisville, Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, United States, 40202
Sponsors and Collaborators
University of Louisville
Solving Kids’ Cancer
Principal Investigator: Kenneth G Lucas, MD University of Louisville, Kosairs Charities Pediatric Clinical Research Unit

Responsible Party: University of Louisville Identifier: NCT01241162     History of Changes
Other Study ID Numbers: 34183
First Posted: November 16, 2010    Key Record Dates
Last Update Posted: May 9, 2017
Last Verified: May 2017

Keywords provided by University of Louisville:
Ewings sarcoma
osteogenic sarcoma
synovial sarcoma

Additional relevant MeSH terms:
Sarcoma, Ewing
Sarcoma, Synovial
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Muscle Tissue
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
Immunologic Factors
Physiological Effects of Drugs
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents