Clinical Trial: Vismodegib on Locally Advanced and Metastatic Basal Cell Carcinoma Under Real World Conditions

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Non-interventional Study to Investigate the Effectiveness, Safety and Utilization of Vismodegib on Locally Advanced and Metastatic Basal Cell Carcinoma Under Real World Conditions

Brief Summary: The aim of this non-interventional study is to provide further data on the utilization, effectiveness, safety and clinical benefit including duration of response of Vismodegib for treatment of locally advanced basal cell carcinoma (laBCC), inappropriate for surgery or radiotherapy and metastatic basal cell carcinoma (mBCC) under real world clinical conditions.

Detailed Summary:

This is a multi-center non-interventional study with approximately 50 patients with locally advanced BCC who start Vismodegib (received at least one dose) in Q4 2015. Duration of recruitment will be one year. Patients will be followed prospectively until disease progression, death, or for 3 years from first dose Vismodegib (whichever occurs first).

In addition to the 50 laBCC patients approximately 3 patients with mBCC will be documented including the following symptoms (pain, fatigue, disturbed sleep, lack of appetite, nausea/vomiting, shortness of breath, disturbed remembering, dry mouth and coughing).

The primary effectiveness objective for this study is as follows:

• To evaluate the 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, which might include and will be categorized in the following groups: clinical assessment, histological assessment, imaging assessment) for laBCC patients.

The secondary effectiveness objectives for this study are as follows:

• Objective response rate (rate of patients with CR or PR) (as determined by the treating physician, which might include and will be categorized in the following groups: clinical assessment, histological assessment, imaging assessment)
• Disease control rate (complete response (CR), partial response (PR) or stable disease (SD))
• Recurrence rate: rate of patients who responded (CR or PR) and later progressed
• Time to progression: time from first treatm
  Sponsor: University Hospital, Essen
  

  Current Primary Outcome: Primary effectiveness measure: duration of response (partial or complete) until progression, death or up to 3 years from first dose Vismodegib (whichever occurs first) [ Time Frame: up to 3 years from first dose Vismodegib ]

  The primary effectiveness measure for this study is as follows:

  • To evaluate the duration of response, defined as duration from first documented complete response (CR) or first documented partial response (PR), whichever cam first, until disease progression (as determined by the treating physician), or death of any reason or up to 3 years from first dose Vismodegib, whichever occurs first, for laBCC patients.

  
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome:

  • Secondary effectiveness measure: - objective response rate [ Time Frame: up to 3 years from first dose Vismodegib ]
    number of patients with complete response or partial response as determined by the treating physician by clinical assessment, histological assessment, imaging assessment
  • Secondary effectiveness measure: - time to response [ Time Frame: up to 3 years from first dose Vismodegib ]
    time from first dose Vismodegib to complete response or partial response up to 3 years
  • Secondary effectiveness measure: - disease control rate [ Time Frame: up to 3 years from first dose Vismodegib ]
    number of complete response, partial response, stable disease
  • Secondary effectiveness measure: - recurrence rate [ Time Frame: up to 3 years from first dose Vismodegib ]
    number of patients who responded (partial or complete) and later progressed
  • Secondary effectiveness measure: - time to progression [ Time Frame: up to 3 years from first dose Vismodegib ]
    time from first dose Vismodegib to progression up to 3 years
  • Secondary effectiveness measure: - survival [ Time Frame: up to 3 years from first dose Vismodegib ]
    time from first dose Vismodegib to death of any cause
  
  
  

  Original Secondary Outcome: Same as current
  
  Information By: University Hospital, Essen
  

  Dates:
  Date Received: April 24, 2016
  Date Started: April 29, 2016
  Date Completion: April 2021
  Last Updated: April 1, 2017
  Last Verified: April 2017