Pembrolizumab in patients with thymic carcinoma: a single-arm, single-centre, phase 2 study
Phase 2N = 41NCT02364076
- Population
- Adults with recurrent thymic carcinoma that had progressed after at least one line of chemotherapy, ECOG performance status 0-2, no history of autoimmune disease or other malignancy requiring treatment, and adequate organ function.
- Design
- Single-arm, single-centre phase 2 study at the Lombardi Comprehensive Cancer Center, Georgetown University, USA. Pembrolizumab 200 mg every 3 weeks for up to 2 years. Enrolment 12 March 2015 to 16 December 2016: 41 patients enrolled, 40 eligible and evaluable (one excluded for elevated liver enzymes at screening). Primary objective: proportion of patients with a response by RECIST 1.1, analysed per protocol in all eligible patients; final analysis. Funded by Merck & Co. A protocol amendment on 10 July 2017 added a separate pembrolizumab plus epacadostat cohort, reported separately; this record covers the pembrolizumab-alone cohort.
- Primary endpoint
- ORR
- Arms
- Pembrolizumab (experimental, n = 41): Pembrolizumab 200 mg every 3 weeks for up to 2 years
- People
- Giaccone G, Lombardi Comprehensive Cancer Center, Georgetown University
Show the numbers
| Endpoint | Analysis | Pembrolizumab | Control | Effect |
|---|---|---|---|---|
| ORREligible and evaluable patients (40 of 41 enrolled; per-protocol analysis) | finalmedian follow-up 20 monthsGiaccone 2018 | 22.5% (10.8–38.5%) | — | One patient (3%) had a complete response, eight (20%) partial responses and 21 (53%) stable disease. Median follow-up 20 months (IQR 14-26). The most common grade 3 or 4 adverse events were increased aspartate aminotransferase and alanine aminotransferase (5 patients [13%] each). 17 deaths at the time of analysis, none due to toxicity. |
| Severe autoimmune toxicityEligible and evaluable patients (40 of 41 enrolled; the full text gives 40 evaluable for safety and activity) | finalGiaccone 2018 | 15% | — | Six patients (15%) developed severe autoimmune toxicity, including two (5%) with myocarditis. The abstract's interpretation states that severe autoimmune disorders are more frequent in thymic carcinoma than in other tumour types and that careful monitoring is essential. |
| Endpoint | Analysis | Pembrolizumab | Control | Effect |
|---|---|---|---|---|
| ORREligible and evaluable patients (40 of 41 enrolled; per-protocol analysis) | finalmedian follow-up 20 monthsGiaccone 2018 | 22.5% (10.8–38.5%) | — | One patient (3%) had a complete response, eight (20%) partial responses and 21 (53%) stable disease. Median follow-up 20 months (IQR 14-26). The most common grade 3 or 4 adverse events were increased aspartate aminotransferase and alanine aminotransferase (5 patients [13%] each). 17 deaths at the time of analysis, none due to toxicity. |
| Severe autoimmune toxicityEligible and evaluable patients (40 of 41 enrolled; the full text gives 40 evaluable for safety and activity) | finalGiaccone 2018 | 15% | — | Six patients (15%) developed severe autoimmune toxicity, including two (5%) with myocarditis. The abstract's interpretation states that severe autoimmune disorders are more frequent in thymic carcinoma than in other tumour types and that careful monitoring is essential. |