Entry Detail
| General information | |
| Database: | DB00367 |
| Objective: | Clinical benefit from cytotoxic chemotherapy for metastatic papillary thyroid carcinoma (PTC) is disappointing, and effective therapeutic approaches for these patients are urgently needed. Because kinaseactivating mutations in the BRAF protooncogene commonly occur in advanced PTC, and inhibition of BRAF(V600E) has shown promising clinical activity in melanoma, BRAF inhibitor therapy may be an effective strategy to treat metastatic PTC. |
| Authors: | Kim KB, et al |
| Title: | Clinical responses to vemurafenib in patients with metastatic papillary thyroid cancer harboring BRAF(V600E) mutation. |
| Journal: | Thyroid |
| Year: | 2013 |
| PMID: | 23489023 |
| Trial Design | |
| Clinical Trial Id: | NCT00215605 |
| Agent: | vemurafenib |
| Target: | BRaf protooncogene serine/threonineprotein kinase |
| Cancer Type: | thyroid cancer |
| Cancer Subtype: | advanced papillary thyroid cancer harboring BRAF(V600E) mutation |
| Therapy Type: | mono |
| Therapeutic Combination Type: | NA |
| Therapeutic Combination Content: | NA |
| Study Type: | phase I study |
| Key Patients Feature: | patients with histologically confirmed solid tumorsthat were refractory to standard therapy or for which standard or curative therapy did not exist, were eligible. They hadto be 18 years of age or older and had to have Eastern Cooperative Oncology Group performance status 0 to 1 withadequate hematologic, hepatic, and renal function. |
| Biomarker: | BRAF(V600E) mutation |
| Biomark Analysis: | this data suggest that the BRAF(V600E) mutant kinase is a relevant target for therapy in this patient population |
| Control Group Info: | single arm |
| Treatment Info: | Vemurafenib was initially dosed at 240360 mg twice a day, later escalated to 720 mg twice a day. Response evaluation was performed every 8 weeks per RECIST. |
| Primary End Point: | efficacy and safety |
| Secondary End Point: | NA |
| Patients Number: | 3 |
| Trial Results | |
| DLT_MTD: | NA |
| Objective Response Rate: | Among these three patients, one had a confirmed partial response, and the other two patients had stable disease as their best initial response to therapy. |
| Disease Control Rate: | NA |
| Median Time to Progression: | meidan TTP: 11.7 months; For the two pts who had stable disease, the TTP was 13.2 and 11.4 months, respectively |
| Median PFS A vs. C: | NA |
| Median OS A vs. C: | NA |
| Adverse Event(agent arm): | NA |
| Conclusions: | Vemurafenib appears to have a promising clinical activity in patients with metastatic PTC, and our data suggest that the BRAF(V600E) mutant kinase is a relevant target for therapy in this patient population. Further investigation of inhibitors of mutated BRAF kinase in patients with PTC in aphase II study is warranted. |