Decitabine Followed by a Cancer Antigen Vaccine for Patients With Neuroblastoma and Sarcoma
- Conditions
- Osteogenic SarcomaNeuroblastomaEwings SarcomaRhabdomyosarcomaSynovial Sarcoma
- Interventions
- Biological: Autologous dendritic cell vaccine with adjuvant
- Registration Number
- NCT01241162
- Lead Sponsor
- 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.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 19
- 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).
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Single arm study Autologous dendritic cell vaccine with adjuvant Biological/Vaccine: Autologous dendritic cell vaccine with adjuvant
- Primary Outcome Measures
Name Time Method Tolerance of study treatment 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
Name Time Method Tumor Response 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.
Immune Response 2 years Assessment of post-vaccination T cell responses to MAGE-A1, MAGE-A3, and NY-ESO-1 by immunoassays.
Trial Locations
- Locations (1)
University of Louisville, Kosair Charities Pediatric Clinical Research Unit
🇺🇸Louisville, Kentucky, United States