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

NCT01241162 | Completed Phase 1 DMC

• 1 other identifier: 34183
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

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.

Conditions

Neuroblastoma; Ewings Sarcoma; Osteogenic Sarcoma; Rhabdomyosarcoma; Synovial Sarcoma

Keywords

Neuroblastoma; Ewings sarcoma; osteogenic sarcoma; rhabdomyosarcoma; synovial sarcoma; high-risk; relapsed; children

Outcome Measures

Primary Outcomes (1)

• Tolerance of study treatment

  Tolerance to DAC, at at least 50% dosing, and 3 of the 4 planned vaccinations during the first two cycles.

  2 years

Secondary Outcomes (2)

• Tumor Response

  2 years

• Immune Response

  2 years

Study Arms (1)

Single arm study

EXPERIMENTAL

Biological/Vaccine: Autologous dendritic cell vaccine with adjuvant

Biological: Autologous dendritic cell vaccine with adjuvant

Interventions

Autologous dendritic cell vaccine with adjuvant BIOLOGICAL

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

Also known as: Decitabine (trade name Dacogen or 5-aza-2'-deoxycytidine/DAC, Hiltonol

Single arm study

Eligibility Criteria

• Age: 1 Year - 24 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Louisville: lead
• Solving Kids' Cancer: collaborator

Study Sites (1)

University of Louisville, Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, 40202, United States

Location

MeSH Terms

Conditions

Neuroblastoma; Sarcoma, Ewing; Osteosarcoma; Rhabdomyosarcoma; Sarcoma, Synovial; Recurrence

Interventions

Adjuvants, Pharmaceutic; Decitabine; poly ICLC

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; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Sarcoma; Myosarcoma; Neoplasms, Muscle Tissue; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pharmaceutic Aids; Pharmaceutical Preparations; Specialty Uses of Chemicals; Chemical Actions and Uses; Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Kenneth G Lucas, MD

  University of Louisville, Kosairs Charities Pediatric Clinical Research Unit

  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: SPONSOR

Study Record Dates

• First Submitted: November 15, 2010
• First Posted: November 16, 2010
• Study Start: August 1, 2010
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: May 9, 2017

Record last verified: 2017-05

Locations

US (1)