ZeSS: A Prospective Observational Safety Study of Patients with BRAFV600 Mutation-positive Unresectable or Metastatic Melanoma Treated with Vemurafenib (Zelboraf®)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Safety registry

Study drug and medical condition

• Name of medicine:
  ZELBORAF
• Medical condition to be studied:
  Malignant melanoma

Population studied

• Short description of the study population:
  Consenting patients identified within one month of initiating treatment with vemurafenib were enrolled from 85 clinical practice sites.
• 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:
  Malignant melanoma patients
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  This study will be a real-world evaluation of the effectiveness of the Summary of ProductCharacteristics (SmPC) monitoring recommendations for the safety of vemurafenib.
• Outcomes:
  Incidence of cutaneous squamous cell carcinomaIncidence of non-cutaneous squamous cell carcinomaIncidence of QT prolongation (defined as QTc >500 ms or an increase in QTc >60 ms)Incidence of abnormal liver function, Incidence of a second (or subsequent) primary melanoma Incidence of gastrointestinal polyps
• Data analysis plan:
  Since the purpose of this Study is largely descriptive, there are no formal sample sizecalculations based on formal comparative hypothesis testing.Most statistical analyses will be descriptive. Descriptive statistics include number of subjects,means, standard deviations, medians, minima, and maxima for continuous variables (e.g.age and duration of treatment) and frequencies and percentages for categorical variables (e.g. gender andevent types). Two sided 95% confidence intervals will be estimated as appropriate. Exposition to the study drug will be summarised and listed with respect to treatment duration, average daily dose, total dose, frequency and reason for dose reductions, time to first dose reduction and reasons for discontinuation from the study drug.