Clinical trial NCT01241162: Decitabine Followed by a Cancer Antigen Vaccine for Patients With Neuroblastoma and Sarcoma
Official title: A Phase I Trial Combining Decitabine and Vaccine Therapy for Patients With Relapsed Neuroblastoma and Sarcoma.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Neuroblastoma
Ewings Sarcoma
Osteogenic Sarcoma
Rhabdomyosarcoma
Synovial Sarcoma
Keywords: Neuroblastoma
Ewings sarcoma
osteogenic sarcoma
rhabdomyosarcoma
synovial sarcoma
high-risk
relapsed
children
Lead sponsor: University of Louisville
Sponsor class: OTHER
Collaborators: name: Solving Kids' Cancer
First posted: 2010-11-16
Enrollment: 19
Sex: ALL
Minimum age (years): 1
Maximum age (years): 24
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Tolerance of study treatment
timeFrame: 2 years
Secondary outcomes:
measure: Tumor Response
timeFrame: 2 years
measure: Immune Response
timeFrame: 2 years
Other outcomes:
Locations:
city: Louisville
country: United States
facility: University of Louisville, Kosair Charities Pediatric Clinical Research Unit
state: Kentucky
Study officials:
affiliation: University of Louisville, Kosairs Charities Pediatric Clinical Research Unit
name: Kenneth G Lucas, MD
role: PRINCIPAL_INVESTIGATOR
References: