Clinical trial NCT02781389: Vismodegib on Locally Advanced Basal Cell Carcinoma Under Real World Conditions
Official title: Non-interventional Study to Investigate the Effectiveness, Safety and Utilization of Vismodegib on Locally Advanced Basal Cell Carcinoma Under Real World Conditions
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Basal Cell Carcinoma
Keywords: Basal cell carcinoma (BCC)
advanced BCC (aBCC)
Lead sponsor: University Hospital, Essen
Sponsor class: OTHER
Collaborators: name: OnkoDataMed GmbH
First posted: 2016-05-24
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 53 Patients with locally advanced BCC who are being treated with Vismodegib will be followed prospectively by centers across Germany and clinical course will be documented

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 under real world clinical conditions.

Detailed description: This is a multi-center non-interventional study with 53 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).

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 treatment to progression
* Time to death: time from first treatment to death
* Time to response: time from first treatment to response

The explorative objectives of this study are to evaluate the following endpoints:

* Type of tumor response evaluation (as determined by the treating physician, which might include and will be categorized in the following groups: clinical assessment , histological assessment, imaging assessment)
* Treatment decision for therapy with Vismodegib (tumor board OR decision by otorhinolaryngologist, surgeon, plastic surgeon, dermato-oncologist, radiotherapist, ophthalmologist)
* Utilization

  * Previous treatment for the BCC lesion, which led to therapy with Vismodegib
  * BCC therapy after Vismodegib therapy (if applicable)
  * Treatment duration of Vismodegib (start and stop dates for therapy with Vismodegib)
  * Assessment of duration and time point of treatment interruptions of Vismodegib
  * Reason for treatment discontinuation

Eligibility criteria: Inclusion Criteria:

* Willing and able to provide informed consent
* Age ≥ 18 years
* laBCC (inappropriate for surgery or radiotherapy)
* Patient is not included in any other trial
* Male or female patient is included in the pregnancy prevention program, as determined by the German authority (BfArM)

Exclusion Criteria:

Patients, for whom treatment with Vismodegib is contraindicated according to the Summary of Product Characteristics (SmPC), which has been in effect at the time of treatment with Vismodegib, including:

* Hypersensitivity to the active substance or to any of the excipients listed in section 6.1. of the latest SmPC
* Women who are pregnant or breast-feeding
* Women of childbearing potential who do not comply with the Vismodegib (Erivedge) Pregnancy Prevention Programme
* Coadministration of St John's wort (Hypericum perforatum)

Primary outcomes:
measure: Primary effectiveness measure: duration of response (partial or complete) until progression, death or up to 3 years from first dose Vismodegib (whichever occurs first)
timeFrame: up to 3 years from first dose Vismodegib
Secondary outcomes:
measure: Secondary effectiveness measure: - objective response rate
timeFrame: up to 3 years from first dose Vismodegib
measure: Secondary effectiveness measure: - time to response
timeFrame: up to 3 years from first dose Vismodegib
measure: Secondary effectiveness measure: - disease control rate
timeFrame: up to 3 years from first dose Vismodegib
measure: Secondary effectiveness measure: - recurrence rate
timeFrame: up to 3 years from first dose Vismodegib
measure: Secondary effectiveness measure: - time to progression
timeFrame: up to 3 years from first dose Vismodegib
measure: Secondary effectiveness measure: - survival
timeFrame: up to 3 years from first dose Vismodegib
Other outcomes:
measure: Explorative outcome measures: tumor response
timeFrame: up to 3 years from first dose Vismodegib
measure: Explorative outcome measures: treatment decision for therapy with Vismodegib
timeFrame: up to 3 years from first dose Vismodegib
measure: Explorative outcome measures: previous treatment
timeFrame: up to 3 years from first dose Vismodegib
measure: Explorative outcome measures: therapy after Vismodegib
timeFrame: up to 3 years from first dose Vismodegib
measure: Explorative outcome measures: duration of therapy with Vismodegib
timeFrame: up to 3 years from first dose Vismodegib
measure: Explorative outcome measures: Vismodegib therapy interruptions
timeFrame: up to 3 years from first dose Vismodegib
measure: Explorative outcome measures: Vismodegib treatment discontinuation
timeFrame: up to 3 years from first dose Vismodegib
Locations:
city: Essen
country: Germany
facility: Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie (Universitätsklinikum Essen)
Study officials:
affiliation: Universitätsklinikum Essen
name: Dirk Schadendorf, Professor
role: PRINCIPAL_INVESTIGATOR
References: