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

Roche

Status and phase: Terminated; Phase 1

Conditions: Malignant Melanoma

Treatments: Drug: vemurafenib

Study type: Interventional

Funder types: Industry

Identifiers

NCT01519323

NO25390

2011-000874-67 (EudraCT Number)

Details and patient eligibility

About

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 participants (aged 12 through 17) with newly diagnosed or recurrent surgically incurable and unresectable Stage IIIC or Stage IV melanoma harboring BRAFV600 mutations. Participants will receive vemurafenib orally twice daily until disease progression or unacceptable toxicity occurs.

Enrollment: 6 patients

Sex: All

Ages: 12 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Pediatric participants, 12 to 17 years of age inclusive
• Histologically confirmed surgically incurable and unresectable Stage IIIC or Stage IV (AJCC) melanoma
• Positive proto-oncogene B-Raf (BRAF) mutation result (Cobas 4800 BRAF V600 Mutation Test)
• Measurable disease according to Response Evaluation Criteria In Solid Tumors (RECIST) criteria
• Performance status: Karnofsky (for participants >/= 16 years of age) or Lansky (for participants < 16 years of age) score of >/= 60
• Adequate bone marrow, liver and renal function
• Participants 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 Methyl Ethyl Ketone (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 human immunodeficiency virus (HIV) positivity or acquired immune deficiency syndrome (AIDS)-related illness, active hepatitis B virus, or active hepatitis C virus

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

Vemurafenib

Experimental group

Description:

Participants received vemurafenib into two separate cohorts with different starting doses based on greater than or equal to (\>=)45 kilogram (kg) and other weighing less than (\<)45 kg. The starting dose for participants (\>=45 kg) was 720 milligram (mg) of vemurafenib by mouth twice daily (BID) and the next dose level for participants in this cohort was 960 mg by mouth BID. The starting dose level for participants weighing \<45 kg was to be 480 mg of vemurafenib by mouth BID, but no participants were enrolled into this cohort.

Treatment:

Drug: vemurafenib