Randomized, Double Blind, Placebo-controlled Topical Resiquimod Adjuvant for NY-ESO-1 Protein Vaccination

Phase 1

Completed

• Conditions: Tumors
• Interventions: Drug: NY-ESO-1 protein; Montanide ISA®-51 VG; Resiquimod; Drug: NY-ESO-1 protein; Montanide ISA®-51 VG; Resiquimod/placebo

• Registration Number: NCT00821652
• Lead Sponsor: Nina Bhardwaj

Brief Summary

The study is designed to see if a course of injections containing the NY-ESO-1 protein (a tumor antigen, marker expressed by tumors); in combination with an immune stimulant (adjuvant) Montanide, with or without resiquimod (another adjuvant) is well tolerated and safe in patients with surgically resected Stage IIB, IIC, Stage III or Stage IV (AJCC criteria) melanoma, a tumor that expresses NY-ESO-1. In addition, this study is designed to see if the patient's body's defense (immune) system can be boosted (strengthened) by this vaccine and if the addition of resiquimod to the vaccine makes this more likely.

Detailed Description

There is no published data on the application of topical resiquimod in combination with an antigen in Montanide, therefore, this study includes a 2-part design where Part I represents a dose-escalation part with topical resiquimod in an open-label fashion. Part II represents the randomized part.

In Part I, 2 cohorts are planned: If no dose-limiting toxicity (DLT) occurs by day 8 of the last vaccination cycle in the last patient enrolled the first cohort, 3 additional patients (cohort 2) will be enrolled. If no DLT occurs by day 8 of the last vaccination cycle in the last patient enrolled the second cohort in Part I, the trial will proceed to Part II where patients will be randomized.

Study Timeline

• Study First Submitted: 2009-01-12
• Study First Submitted QC: 2009-01-12
• Study First Posted: 2009-01-13
• Study Start: 2009-02
• Primary Completion: 2010-01
• Status Verified: 2014-12
• Study Completion: 2014-12
• Last Update Submitted: 2015-01-06
• Last Update Posted: 2015-01-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: FACTORIAL

Arm && Interventions

Group	Intervention	Description
Dose-esclation	NY-ESO-1 protein; Montanide ISA®-51 VG; Resiquimod	Part I represents a dose-escalation part with topical resiquimod in an open-label fashion.
2	NY-ESO-1 protein; Montanide ISA®-51 VG; Resiquimod/placebo	Part II: Eligible patients will be randomized to receive a subcutaneous vaccination of 100µg NY-ESO-1 protein emulsified in 1.25mL Montanide ISA®-51 VG (day 1) followed by topical placebo gel (Arm A) or topical resiquimod gel (Arm B) on days 1, 3 and 5; or resiquimod dosing regimen established in Part I.

Primary Outcome Measures

Name	Time	Method
The primary objectives of the study are to define the safety and immunogenicity of vaccination with NY-ESO-1 protein emulsified in Montanide® ISA-51 VG when given with or without the topical TLR 7/8 agonist resiquimod.	Blood samples are obtained at baseline, 1 week after each vaccination, and at follow-up 1visit.

Secondary Outcome Measures

Name	Time	Method
To document tumor response by RECIST criteria if applicable. Skin section analysis of resiquimod/placebo treated site for immune cell infiltration and gene expression analysis. Investigation of polymorphisms for TLR7/8 through germline SNP analysis	Skin biopsies will be obtained after the last vaccination cycle. Clinical hematology and biochemistry measurements will be taken at baseline, one week after the second vaccination and two to four weeks after the fourth vaccination

Trial Locations

Locations (1)

Icahn School of Medicine at Mount Sinai; 🇺🇸 New York, New York, United States