A Study of Vemurafenib in Pediatric Patients With Stage IIIC or Stage IV Melanoma Harboring BRAFV600 Mutations

Status: Recruiting
Diagnosis: Pediatric Solid Tumors
NCT ID: NCT01519323 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID: 11-388


This open-label, multicenter. single arm Phase I dose-escalation study with efficacy tail extension will evaluate the maximum tolerated dose/recommended dose, the safety and efficacy of vemurafenib (RO5185426) in pediatric patients (aged 12 through 17) with newly diagnosed or recurrent surgically incurable and unresectable Stage IIIC or Stage IV melanoma harboring BRAFV600 mutations. Patients will receive vemurafenib orally twice daily until disease progression or unacceptable toxicity occurs.


Conducting Institutions:
Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:
Carlos Rodriguez-Galindo, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

Inclusion Criteria: - Pediatric patients, 12 to 17 years of age inclusive - Histologically confirmed surgically incurable and unresectable Stage IIIC or Stage IV (AJCC) melanoma - Positive BRAF mutation result (Cobas 4800 BRAF V600 Mutation Test) - Measurable disease according to RECIST criteria - Performance status: Karnofsky (for patients >/= 16 years of age) or Lansky (for patients < 16 years of age) score of >/= 60 - Adequate bone marrow, liver and renal function - Patients must have fully recovered from the acute toxic effects of all prior therapy prior to first administration of study drug Exclusion Criteria: - Active or untreated central nervous system (CNS) lesions - History of or known spinal cord compression or carcinomatous meningitis - Anticipated or ongoing administration of anti-cancer therapies other than those administered in this study - Previous malignancy within the past 5 years except for basal or squamous cell carcinoma of the skin, melanoma in-situ, and carcinoma in-situ of the cervix - Previous treatment with selective/specific BRAF or MEK inhibitor (previous treatment with sorafenib is allowed) - Any previous treatment with study drug (RO5185426) or participation in a clinical trial that includes RO5185426 - Pregnant or lactating females - Known HIV positivity or AIDS-related illness, active hepatitis B virus, or active hepatitis C virus
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