NCT01479244

Efficacy and Safety Study of NeuVax™ (Nelipepimut-S or E75) Vaccine to Prevent Breast Cancer Recurrence

PRESENT: Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer With Low to Intermediate HER2 Expressions With NeuVax™Treatment

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 758 (actual)
• Sponsor: Galena Biopharma, Inc. · Industry
• Sex: Female
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Purpose of this trial: 1. To assess the efficacy and safety of NeuVax™ administered with adjuvant Leukine® (sargramostim, GM-CSF). 2. To evaluate and compare the disease free survival (DFS) in the vaccinated and control subjects.

Detailed description

This is a multicenter, multinational, prospective, randomized, double-blind, controlled Phase 3 study. The subjects eligible for this trial have an early stage node-positive breast cancer. Their tumors express low or intermediate levels of the HER2 protein. NeuVax™ will be administered after completion of front-line, standard of care therapy (surgery, radiation therapy, and chemotherapy) and can be given concomitantly with physician prescribed endocrine treatment. NeuVax™ is the immmunodominant nonapeptide derived from the extracellular domain of the HER2 protein, a well-established target for therapeutic intervention in breast carcinoma. The nelipepimut sequence stimulates specific CD8+ cytotoxic T lymphocytes (CTL) following binding to HLA-A2/A3 molecules on antigen presenting cells (APC). These activated specific CTLs recognize, neutralize and destroy through cell lysis HER2 expressing cancer cells, including occult cancer cells and micrometastatic foci. The nelipepimut immune response can also generate CTLs to other immunogenic peptides through inter- and intra-antigenic epitope spreading. Based on a successful Phase 2 trial, which achieved its primary endpoint of DFS, the Food and Drug Administration (FDA) granted NeuVax a Special Protocol Assessment (SPA) for its Phase 3 PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment) study. The active portion of the study will last three years (36 months). The follow-up will last from 5 to 10 years. Endpoints: 1. Primary efficacy endpoint: * 3-year DFS 2. Secondary efficacy endpoints: * 5- and 10-year DFS * 3-year OS * 5- and 10-year OS * Safety profile, and adverse events (AEs) * Patterns of recurrence to include Time to recurrence (TTR), time to local recurrence (TTLR), time to distant recurrence (TTDR), time to bone metastases (TTBM) Safety Assessments: Subjects will be assessed at every study visit for the safety endpoints, AEs,vital signs, physical examinations and laboratory data; yearly follow-up of survival will include imaging studies, ECGs, MUGA or ECHO scans and concomitant medications.

Conditions

• Breast Cancer With Low to Intermediate HER2 Expression

Interventions

Type	Name	Description
BIOLOGICAL	NeuVax™ vaccine	E75 peptide acetate (HER2/neu p366-379) in WFI (1.5 mg/mL) mixed with 250 micrograms Leukine® (sargramostim, GM-CSF), will be administered in intradermal injections, in four divided doses (4 sites on upper thigh) once a month, for six consecutive months. At the end of the six months, NeuVax™ vaccine boosters will be given the same way once every six months for the next five times (totaling 36 months).
BIOLOGICAL	Leukine® (sargramostim, GM-CSF) and water for injection	Leukine® 250 micrograms in water for injection, will be administered in intradermal injections, in four divided doses (4 sites on upper thigh) once a month, for six consecutive months. At the end of the six months, boosters inoculations will be given the same way once every six months for the next five times (totaling 36 months).

Timeline

• Start date: 2011-11-01
• Primary completion: 2016-09-21
• Completion: 2016-09-21
• First posted: 2011-11-24
• Last updated: 2017-02-27

Locations

197 sites across 13 countries: United States, Bulgaria, Canada, Czechia, France, Germany, Hungary, Israel, Poland, Romania, Russia, Ukraine, United Kingdom