Non-Interventional Study To Investigate The Effectiveness, Safety And Utilization Of Vismodegib On Locally Advanced Basal Cell Carcinoma Under Real World Conditions (NIELS)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Drug interaction study

Study drug and medical condition

• Medicinal product name, other:
  Erivadge
• Study drug International non-proprietary name (INN) or common name:
  VISMODEGIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XX43) vismodegib

  vismodegib
• Additional medical condition(s):
  LOCALLY ADVANCED BASAL CELL CARCINOMA

Population studied

• Short description of the study population:
  Locally advanced basal cell carcinoma
• Age groups:
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Basel cell carcinoma patients
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The aim of this multi-center, non-interventional study is to provide further data on effectiveness with a special focus on duration of response, safety and utilization of Vismodegib for treatment of laBCC in daily practice in Germany.
• Outcomes:
  Duration of response defined as duration from first documented response of complete response (CR) or partial response (PR) until disease progression (as determined by the treating physician) for laBCC patients, •Objective response rate (ORR) (CR + PR)•Disease control rate (CR + PR + SD)•Recurrence rate•Progression free survival time and overall survival•Time to response•All protocol-specified adverse events (AEs) •Incidence, risk factors, and outcomes of serious and non-serious AEs of interest
• Data analysis plan:
  No formal statistical hypothesis and no formal sample size calculation will be performed.All efficacy and safety variables documented in this study will be analyzed by means of descriptive analysis.Continuous data will be summarized using mean, standard deviation, median, minimum and maximum. Categorical variables will be expressed as absolute and relative number and 95% Cl. Kaplan-Meier estimates and Cox-regression will be used to model data on time to disease progression and duration of response.