A Study of Vemurafenib (RO5185426) in Participants With Metastatic or Unresectable Papillary Thyroid Cancer Positive for the BRAF V600 Mutation

Roche

Status and phase: Completed; Phase 2

Conditions: Neoplasms

Treatments: Drug: Vemurafenib

Study type: Interventional

Funder types: Industry

Identifiers

NCT01286753

NO25530

2010-024133-23

Details and patient eligibility

About

This open-label, multi-center study will evaluate the safety and efficacy of Vemurafenib (RO5185426) in participants with metastatic or unresectable papillary thyroid cancer (PTC) positive for the BRAF V600 mutation and resistant to radioactive iodine therapy. Participants will receive vemurafenib 960 milligrams (mg) orally twice daily until progressive disease or unacceptable toxicity occurs.

Enrollment: 51 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Adult participants. >/= 18 years of age
• Histologically confirmed metastatic or unresectable papillary thyroid cancer for which standard curative or palliative measures do not exist or are no longer effective; participants whose tumors exhibit areas of "other histology" may be enrolled, provided the tumor histology remains predominantly papillary
• Positive for BRAF V600 mutation (Roche Cobas 4800 BRAF V600 Mutation Test)
• Radioactive Iodine resistant disease
• Prior therapy excluding (Cohort 1, TKI Naive) or including (Cohort 2, TKI Experienced) TKI
• Clinically relevant disease progression according to RECIST criteria within the prior 14 months
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
• Adequate hematological, renal and liver function

Exclusion criteria

• Histological diagnosis other than papillary PTC, including squamous cell variants of PTC or PTC with areas of squamous metaplasia
• Active or untreated central nervous system metastases
• History of or known carcinomatous meningitis
• Anticipated or ongoing administration of any anti-cancer therapies other than those administered in the study
• Active squamous cell skin cancer that has not been excised or adequately healed post excision
• Previous treatment with any agent that specifically and selectively targets the MEK or BRAF pathway
• Prior radiotherapy to the only measurable lesion
• Clinically relevant cardio-vascular disease or event within the prior 6 months

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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51 participants in 2 patient groups

Tyrosine Kinase Inhibitor (TKI) Naive

Experimental group

Description:

Vemurafenib in participants naive to any prior systemic TKI therapy.

Treatment:

Drug: Vemurafenib

TKI Experienced

Experimental group

Description:

Vemurafenib in participants previously treated with TKI therapy active against vascular endothelial growth factor receptor 2 (VEGFR).

Treatment:

Drug: Vemurafenib