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Poster Display session

341 - AcSé immunotherapy trials: Anti-PD-1 therapy for adult patients with selected rare cancer types


14 Dec 2018


Poster Display session


Aurelien Marabelle


Annals of Oncology (2018) 29 (suppl_10): x24-x38. 10.1093/annonc/mdy487


A. Marabelle1, S. Chevret2, F. Janot3, B. Escudier4, D. Pouessel5, C. Tournigand6, K. Hoang-Xuan7, L. Mortier8, B.J. Rousseau9, M.J. Schlumberger10, I.L. Ray-Coquard11, J. Blay12, P. Niccoli13, A. Jaccard14, D. Couch15, N. Hoog-Labouret16, I. Pauporte17, C. Massard18

Author affiliations

  • 1 Drug Development Department, Gustave Roussy, 94805 - Villejuif/FR
  • 2 Service De Biostatistique Et Information Médicale, Hôpital Saint Louis, 75475 - Paris/FR
  • 3 Département De Cancérologie Cervico-faciale, Gustave Roussy, 94805 - Villejuif/FR
  • 4 Département De Médecine Oncologique, Gustave Roussy - Cancer Campus, 94805 - Villejuif/FR
  • 5 Oncologie Médiclae, IUCT, 31059 - Toulouse/FR
  • 6 Oncologie Médicale, Centre hospitalier universitaire Henri-Mondor, 94010 - Créteil/FR
  • 7 Service De Neurologie, Groupe Hospitalier Pitié Salpetriere, 75651 - Paris/FR
  • 8 Dermatology Clinic, Hopital Claude Huriez, 59037 - Lille/FR
  • 9 Service D'oncologie Médicale, Centre hospitalier universitaire Henri-Mondor, 94010 - Créteil/FR
  • 10 Service De Médecine Nucléaire Et Cancérologie Endocrinienne, Gustave Roussy Institut de Cancérologie, 94805 - Villejuif/FR
  • 11 Medical Oncology, Centre Léon Bérard, 69008 - Lyon/FR
  • 12 Medicine, Centre Léon Bérard, 69008 - Lyon/FR
  • 13 Département D'oncologie Médicale, Institut Paoli Calmettes, 13273 - Marseille/FR
  • 14 Service D'hématologie Clinique Et Thérapie Cellulaire, CHU de Limoges, 87042 - Limoges/FR
  • 15 R&d, UNICANCER, 75654 - Paris/FR
  • 16 Pôle Recherche Et Innovation, Institut National du Cancer, 92513 - Boulogne-Billancourt/FR
  • 17 Recherche, La Ligue Contre le Cancer, 75013 - Paris/FR
  • 18 Ditep, Gustave Roussy - Cancer Campus, 94805 - Villejuif/FR


Abstract 341


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

AcSé nivolumabAcSé pembrolizumab
Cohort 1: Non-clear cell RCCCohort 1: Rare sarcoma
Cohort 2: Rare head and neck cancerCohort 2: Rare ovarian cancer
Cohort 3: Rare skin cancerCohort 3: Primary CNS lymphoma
Cohort 4: MSI-H cancer (other than CRC)Cohort 4: Rare thyroid cancer
Cohort 5: Penile cancerCohort 5: Rare malignant neuroendocrine cancer
Cohourt 6: POLE exonuclease domain mutated cancerCohort 6: Germ-cell cancer
Cohort 7: NK/T-cell lymphoma

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.

Editorial acknowledgement

Clinical trial identification

Legal entity responsible for the study



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


All authors have declared no conflicts of interest.

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