Vismodegib on Locally Advanced Basal Cell Carcinoma Under Real World Conditions

Completed

• Conditions: Basal Cell Carcinoma

• Registration Number: NCT02781389
• Lead Sponsor: University Hospital, Essen

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

Study Timeline

• Study First Submitted: 2016-04-24
• Study Start: 2016-04-29
• Study First Submitted QC: 2016-05-19
• Study First Posted: 2016-05-24
• Primary Completion: 2021-05-31
• Study Completion: 2021-05-31
• Status Verified: 2021-06
• Last Update Submitted: 2021-06-02
• Last Update Posted: 2021-06-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 53

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)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary effectiveness measure: duration of response (partial or complete) until progression, death or up to 3 years from first dose Vismodegib (whichever occurs first)	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.

Secondary Outcome Measures

Name	Time	Method
Secondary effectiveness measure: - time to response	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: - survival	up to 3 years from first dose Vismodegib	time from first dose Vismodegib to death of any cause
Secondary effectiveness measure: - recurrence rate	up to 3 years from first dose Vismodegib	number of patients who responded (partial or complete) and later progressed
Secondary effectiveness measure: - disease control rate	up to 3 years from first dose Vismodegib	number of complete response, partial response, stable disease
Secondary effectiveness measure: - objective response rate	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 progression	up to 3 years from first dose Vismodegib	time from first dose Vismodegib to progression up to 3 years

Trial Locations

Locations (1)

Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie (Universitätsklinikum Essen); 🇩🇪 Essen, Germany