E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Metastatic basal cell carcinoma or unresectable locally advanced basal cell carcinoma |
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E.1.1.1 | Medical condition in easily understood language |
advanced basal cell carcinoma, which is a type of skin cancer |
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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 | 20.0 |
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 estimate the overall response rate (ORR) for metastatic Basal Cell Carcinoma (BCC) (group 1) or for unresectable locally advanced BCC (group 2) when treated with cemiplimab as a monotherapy
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E.2.2 | Secondary objectives of the trial |
-Duration of response -Progression free survival -Complete response rate -Change in scores of patient-reported outcomes in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) -Change in scores of patient-reported outcomes in Skindex-16 -Overall Survival |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Histologically confirmed diagnosis of invasive BCC -At least 1 measurable lesion -Eastern Cooperative Oncology Group (ECOG) performance status ≤1 -At least 18 years old -Willing and able to comply with clinic visits and study-related procedures -Provide signed informed consent prior to any screening procedures
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E.4 | Principal exclusion criteria |
-Ongoing or recent (within 5 years) evidence of significant autoimmune disease that required treatment with systemic immunosuppressive treatments, which may suggest risk for immune-related adverse events -Prior treatment with an agent that blocks the PD-1/PD-L1 pathway -History of pneumonitis within the last 5 years -Allergic reactions or acute hypersensitivity reaction attributed to antibody treatments -Patients with a history of solid organ transplant -Prior treatment with other immune modulating agents within fewer than 28 days prior to the first dose of cemiplimab. Examples of immune modulating include therapeutic vaccines, cytokine treatments, or agents that target cytotoxic T-lymphocyte antigen 4 (CTLA-4), 4-1BB (CD137), or OX-40 -Untreated brain metastasis(es) that may be considered active
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint for this study is the ORR as determined by central review. The ORR will be assessed separately for patients with metastatic BCC (Group 1) or unresectable locally advanced BCC (Group 2) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
5 treatment cycles of 9 weeks and 4 treatment cycles of 12 weeks |
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E.5.2 | Secondary end point(s) |
· Duration of response · PFS · OS · CR rate · Change in scores of patient-reported outcomes in the EORTC QLQ-C30 and the Skindex-16 · AEs · Concentrations of cemiplimab in serum |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
5 treatment cycles of 9 weeks and 4 treatment cycles of 12 weeks |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 45 |
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 |
Australia |
Austria |
Belgium |
Canada |
France |
Germany |
Greece |
Italy |
Spain |
Switzerland |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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
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When the last patient to enter retreatment completes the retreatment plus safety follow up |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 22 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 22 |