E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Locally advanced or metastatic basal cell carcinoma (BCC) |
|
E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10004146 |
E.1.2 | Term | Basal cell carcinoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety of vismodegib (GDC-0449) in patients with locally advanced or metastatic BCC |
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E.2.2 | Secondary objectives of the trial |
- To assess the overall response rate (according to RECIST Version 1.1) in those patients with measurable disease as permitted by local regulatory requirement.
- To assess other efficacy parameters such as time to response, duration of response, progression-free survival and overall survival.
- To assess patient quality of life.
- To assess the impact of vismodegib treatment on disease symptoms in patients with metastatic BCC who enrolled after approval of the Study Protocol, version 4.0, using the M.D. Anderson Symptom Inventory.
- To assess the status of the Hedgehog pathway and/or other modifiers of vismodegib activity in tumor tissue obtained from patients with metastatic BCC who have disease progression on vismodegib therapy.
- Adverse event-related pharmacokinetic testing will be performed.
- Post-treatment pharmacokinetic testing will be performed. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Adult patients, >/=18 years of age
- Metastatic or locally advanced basal cell carcinoma considered inoperable or that surgery is contraindicated and radiotherapy is contraindicated or inappropriate
- Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2 |
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E.4 | Principal exclusion criteria |
- Concurrent anti-tumor therapy
- Completion of the most recent anti-tumor therapy less than 21 days prior to the initiation of treatment
- Uncontrolled medical illness
- Patients with one of the following rare hereditary conditions: galactose intolerance, primary hypolactasia, or glucose-galactose malabsorption |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary objective of this trial is to assess the safety of vismodegib (GDC-0449) in patients with locally advanced or metastatic BCC. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Until disease progression or unacceptable toxicity (approximately 2 years) |
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E.5.2 | Secondary end point(s) |
- Tumor response rate according to Response Evaluation Criteria in Solid Tumors (RECIST)
- Time to response
- Duration of response
- Progression-free survival
- Overall survival |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Until disease progression or unacceptable toxicity (approximately 2 years) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | Yes |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 137 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
Bulgaria |
Canada |
Denmark |
France |
Greece |
Ireland |
Italy |
Austria |
Croatia |
Netherlands |
New Zealand |
Norway |
Portugal |
Romania |
Slovakia |
Sweden |
Bosnia and Herzegovina |
Albania |
Argentina |
Australia |
Brazil |
Colombia |
Czech Republic |
Estonia |
Finland |
Germany |
Hungary |
Lithuania |
Spain |
Israel |
Mexico |
Peru |
Poland |
Russian Federation |
Serbia |
Slovenia |
South Africa |
Switzerland |
Turkey |
Ukraine |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
The study will end when the last patient on treatment develops progressive disease (as determined by the investigator) or unacceptable toxicity, withdraws consent, or dies; the treating physician deems the patient is no longer benefitting from treatment; or the study is terminated by the Sponsor; or 12 months after the last dose of vismodegib in the last enrolled patient still on study, whichever occurs first. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |