title: A Case-Control Study Brings to Light the Causes of Screen Failures in Phase 1 Cancer Clinical Trials.
abstract: Enrolling cancer patients in phase I clinical trials (P1s) requires that they fulfill specific criteria. Between the time they sign the consent form and the 1st administration of the experimental drug, some patients may be excluded and considered as screen failures (SFs). Our objective was to assess SF patients profiles and the reasons and risk factors for SFs.
  
  
  
  All patients included in P1s at Gustave Roussy from 2008 to 2013 were reviewed retrospectively. SFs were matched with control P1 patients who were successfully enrolled. Patient and tumor characteristics, P1 types and the reasons for SF were analyzed.
  
  
  
  Among 1,293 patients, 192 (15%) were SF cases; 182 SF cases were matched with 182 controls: median age was 57 (48-64) and 55 (47-63), median home-cancer center distance was 69 vs 55 km, 45% vs 34% had more than 2 metastatic sites, median screening period was 14 vs 11 days, median progression-free survival during the previous line was 12 vs 14 weeks, 37% vs 29% of LDH values were above the upper limit of normal, 42% vs 36% of albumin values were < 35 g/L, respectively. Reasons for SFs were cancer progression (44%), sponsor decision unrelated to a clinical reason (25%), patient retrieval (13.5%), relevant comorbidity (13.5%). Multivariate analysis revealed that a high Royal Marsden Hospital (RMH) prognostic score was potentially associated with higher risk of SFs (OR = 2.3; 95% CI [1.0-5.7], p = 0.06).
  
  
  
  Cancer progression led to half of the SFs in P1s. Physicians should pay attention to the RMH score at the time of patient inclusion to avoid further SFs.
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Kempf
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Lemoine
authors.affiliation: Gustave-Roussy Cancer Campus, Biostatistics and Epidemiology unit, Villejuif, France.
authors.forename: Gabrielle
authors.initials: G
authors.lastname: Tergemina-Clain
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.
authors.forename: Anthony
authors.initials: A
authors.lastname: Turpin
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.|Université Paris Saclay, Université Paris-Sud, Faculté de médecine, Le Kremlin Bicêtre, France.|Inserm Unit U981, Gustave Roussy, Villejuif, France.
authors.forename: Sophie
authors.initials: S
authors.lastname: Postel-Vinay
authors.affiliation: Gustave-Roussy Cancer Campus, Biostatistics and Epidemiology unit, Villejuif, France.
authors.forename: Emilie
authors.initials: E
authors.lastname: Lanoy
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.|Université Paris Saclay, Université Paris-Sud, Faculté de médecine, Le Kremlin Bicêtre, France.|Inserm Unit U981, Gustave Roussy, Villejuif, France.
authors.forename: Jean-Charles
authors.initials: JC
authors.lastname: Soria
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.
authors.forename: Christophe
authors.initials: C
authors.lastname: Massard
authors.affiliation: Gustave-Roussy Cancer Campus, Drug Development Department, Villejuif, France.
authors.forename: Antoine
authors.initials: A
authors.lastname: Hollebecque
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D016022:Case-Control Studies; D017321:Clinical Trials, Phase I as Topic / Q000379:methods*; D055088:Early Detection of Cancer / Q000379:methods*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009369:Neoplasms / Q000175:diagnosis / Q000188:drug therapy; D012189:Retrospective Studies
chemical_list: D000970:Antineoplastic Agents