Vismodegib on Locally Advanced Basal Cell Carcinoma Under Real World Conditions (JONAS)

• ClinicalTrials.gov Identifier: NCT02781389
• Recruitment Status: Active, not recruiting
• First Posted: May 24, 2016
• Last Update Posted: September 6, 2019
• Sponsor: University Hospital, Essen
• Collaborator: OnkoDataMed GmbH
• Information provided by (Responsible Party): Prof. Dr. med. Dirk Schadendorf, University Hospital, Essen

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.

Condition or disease
Basal Cell Carcinoma

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 Design

• Study Type: Observational
• Actual Enrollment: 53 participants
• Observational Model: Case-Control
• Time Perspective: Prospective
• Official Title: Non-interventional Study to Investigate the Effectiveness, Safety and Utilization of Vismodegib on Locally Advanced Basal Cell Carcinoma Under Real World Conditions
• Actual Study Start Date: April 29, 2016
• Estimated Primary Completion Date: April 2021
• Estimated Study Completion Date: April 2021

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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
2. 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
3. Secondary effectiveness measure: - disease control rate [ Time Frame: up to 3 years from first dose Vismodegib ]
   number of complete response, partial response, stable disease
4. 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
5. 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
6. Secondary effectiveness measure: - survival [ Time Frame: up to 3 years from first dose Vismodegib ]
   time from first dose Vismodegib to death of any cause

Other Outcome Measures:

1. Explorative outcome measures: tumor response [ Time Frame: 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
2. Explorative outcome measures: treatment decision for therapy with Vismodegib [ Time Frame: 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
3. Explorative outcome measures: previous treatment [ Time Frame: up to 3 years from first dose Vismodegib ]
   - previous treatment for the BCC lesion, which led to therapy with Vismodegib
4. Explorative outcome measures: therapy after Vismodegib [ Time Frame: up to 3 years from first dose Vismodegib ]
   - BCC therapy after Vismodegib therapy
5. Explorative outcome measures: duration of therapy with Vismodegib [ Time Frame: up to 3 years from first dose Vismodegib ]
   - time of treatment with Vismodegib
6. Explorative outcome measures: Vismodegib therapy interruptions [ Time Frame: up to 3 years from first dose Vismodegib ]
   - time to treatment interruptions of Vismodegib
7. Explorative outcome measures: Vismodegib treatment discontinuation [ Time Frame: up to 3 years from first dose Vismodegib ]
   - reason for treatment discontinuation of Vismodegib

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• 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

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)

Contacts and Locations

Locations

Germany

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

More Information

• Responsible Party: Prof. Dr. med. Dirk Schadendorf, Prof. Dr. med., University Hospital, Essen
• ClinicalTrials.gov Identifier: NCT02781389
• Other Study ID Numbers: ML29828
• First Posted: May 24, 2016
• Last Update Posted: September 6, 2019
• Last Verified: September 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

Keywords provided by Prof. Dr. med. Dirk Schadendorf, University Hospital, Essen:

Basal cell carcinoma (BCC); advanced BCC (aBCC)

Additional relevant MeSH terms:

Carcinoma; Carcinoma, Basal Cell; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Basal Cell