JONAS: Vismodegib on Locally Advanced Basal Cell Carcinoma Under Real World Conditions
Study Details
Study Description
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.
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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:
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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)
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Disease control rate (complete response (CR), partial response (PR) or stable disease (SD))
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Recurrence rate: rate of patients who responded (CR or PR) and later progressed
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Time to progression: time from first treatment to progression
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Time to death: time from first treatment to death
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Time to response: time from first treatment to response
The explorative objectives of this study are to evaluate the following endpoints:
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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)
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Treatment decision for therapy with Vismodegib (tumor board OR decision by otorhinolaryngologist, surgeon, plastic surgeon, dermato-oncologist, radiotherapist, ophthalmologist)
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Utilization
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Previous treatment for the BCC lesion, which led to therapy with Vismodegib
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BCC therapy after Vismodegib therapy (if applicable)
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Treatment duration of Vismodegib (start and stop dates for therapy with Vismodegib)
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Assessment of duration and time point of treatment interruptions of Vismodegib
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Reason for treatment discontinuation
Study Design
Outcome Measures
Primary Outcome Measures
- 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
- 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 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: - disease control rate [up to 3 years from first dose Vismodegib]
number of complete response, partial response, stable disease
- 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: - time to progression [up to 3 years from first dose Vismodegib]
time from first dose Vismodegib to progression 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
Other Outcome Measures
- Explorative outcome measures: tumor response [up to 3 years from first dose Vismodegib]
- type of tumor response as determined by the treating physician by clinical assessment and/or histological assessment and/or imaging assessment
- Explorative outcome measures: treatment decision for therapy with Vismodegib [up to 3 years from first dose Vismodegib]
- treatment decision for therapy with Vismodegib by tumor board OR by otorhinolaryngologist, surgeon, plastic surgeon, dermato-oncologist, radiotherapist, ophthalmologist
- Explorative outcome measures: previous treatment [up to 3 years from first dose Vismodegib]
- previous treatment for the BCC lesion, which led to therapy with Vismodegib
- Explorative outcome measures: therapy after Vismodegib [up to 3 years from first dose Vismodegib]
- BCC therapy after Vismodegib therapy
- Explorative outcome measures: duration of therapy with Vismodegib [up to 3 years from first dose Vismodegib]
- time of treatment with Vismodegib
- Explorative outcome measures: Vismodegib therapy interruptions [up to 3 years from first dose Vismodegib]
- time to treatment interruptions of Vismodegib
- Explorative outcome measures: Vismodegib treatment discontinuation [up to 3 years from first dose Vismodegib]
- reason for treatment discontinuation of Vismodegib
Eligibility Criteria
Criteria
Inclusion Criteria:
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Willing and able to provide informed consent
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Age ≥ 18 years
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laBCC (inappropriate for surgery or radiotherapy)
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Patient is not included in any other trial
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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:
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Hypersensitivity to the active substance or to any of the excipients listed in section 6.1. of the latest SmPC
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Women who are pregnant or breast-feeding
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Women of childbearing potential who do not comply with the Vismodegib (Erivedge) Pregnancy Prevention Programme
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Coadministration of St John's wort (Hypericum perforatum)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie (Universitätsklinikum Essen) | Essen | Germany | 45122 |
Sponsors and Collaborators
- University Hospital, Essen
- OnkoDataMed GmbH
Investigators
- Principal Investigator: Dirk Schadendorf, Professor, Universitätsklinikum Essen
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ML29828