For clinicians

About the OPTiM trial (OncoVEXGM-CSF Pivotal Trial in Melanoma)

As of March 1, 2009, the OncoVEXGM-CSF protocol is the most recently added Phase 3 study for patients with advanced melanoma on ClinicalTrials.gov web site (Study identifier: NCT00769704). As far as we are aware, there are no other actively recruiting Phase 3 studies for these patients. The study will begin actively recruiting patients in the US in April 2009, and in the United Kingdom in subsequent months. A total of 360 patients will be enrolled (240 to the OncoVEXGM-CSF arm and 120 to the control arm).  The US Food and Drug Administration (FDA) gave the study a Special Protocol Assessment (SPA), which is a designation from the FDA that the trial's design, clinical endpoints, and statistical analyses are acceptable for FDA approval.

The trial is a multi-national, open-label, randomized Phase 3 study to assess OncoVEXGM-CSF monotherapy or control in patients with unresectable, stage IIIb, IIIc or stage IV melanoma (Figure 1). Patients who have participated in prior investigational studies are qualified for enrollment.

Figure 1: Study Design

OncoVEXGM-CSF will be administered by intra-tumoral injection into cutaneous, sub-cutaneous or nodal masses, including through the use of ultrasound guidance for non-palpable masses not located in vital organs (i.e. lung or liver). The control treatment is GM-CSF (administered subcutaneously for 14 consecutive days every month). The primary objective is achieving a statistically greater proportion of durable objective responses (maintained for 6 months) than the control therapy.

The design of the OncoVEX Pivotal Trial in Melanoma study is based on a 50-patient Phase 2 study in which treatment with OncoVEXGM-CSF monotherapy resulted in a 26% objective response rate (CRs and PRs) with impressive duration of response and survival data.