Background

Immune checkpoint blockade represents a major breakthrough in cancer therapy with recent approvals of PD-1 or PD-L1 antagonist therapy in France for a range of cancer indications. To generate high evidenced-based knowledge and to prevent off-label use, the French National Cancer Institute (INCa) launched the AcSé Immuno-therapy Program: two exploratory phase II trials, to allow for a nationwide safe and controlled access to nivolumab or pembrolizumab outside of their current marketing approvals for selected rare cancer indications where the literature suggests a potential benefit for patients, but where the difficulties of development render individual experimental studies unattractive to the pharmaceutical industry.

Trial design

The two trials, AcSé Nivolumab and AcSé Pembrolizumab are Phase 2, single-arm, national, multicentre trials investigating the efficacy and safety of nivolumab and pembrolizumab, respectively, in adult patients with specific rare cancers who have unresectable locally advanced or metastatic disease which is resistant or refractory to standard therapy and for whom no alternative approved or experimental treatment options exist. Up to 650 patients will be enrolled across the two trials and assigned to one of 13 cohorts (max. 50 patients/cohort) according to their indication (see table).Table: 109TiP

The trials will use a two-stage Bayesian enrichment design to identify potentially sensitive indications and assess treatment efficacy per cohort. Toxicity will also be assessed per cohort and biological samples collected to explore the predictive factors of response and mechanisms of acute and acquired resistance to anti-PD-1 therapy in these populations.

Legal entity responsible for the study

UNICANCER.

Funding

Institut National du Cancer (INCa), La Ligue contre le Cancer, Bristol-Myers Squibb (BMS), MSD.

Disclosure

All authors have declared no conflicts of interest.