Trial Outcomes & Findings for Phase 2 Study of Neoadjuvant Vemurafenib in Melanoma Patients With Untreated Brain Metastases (NCT NCT01781026)

NCT ID: NCT01781026

Last Updated: 2017-03-10

Results Overview

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 2 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2017-03-10

Participant Flow

Participant milestones

Measure	Vemurafenib Administration Vemurafenib 960 mg orally, twice a day
Overall Study STARTED	2
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Vemurafenib Administration Vemurafenib 960 mg orally, twice a day
Overall Study Lack of Efficacy	2

Baseline Characteristics

Phase 2 Study of Neoadjuvant Vemurafenib in Melanoma Patients With Untreated Brain Metastases

Baseline characteristics by cohort

Measure	Vemurafenib Administration n=2 Participants Vemurafenib 960 mg orally, twice a day
Age, Continuous	42 years n=93 Participants
Sex: Female, Male Female	0 Participants n=93 Participants
Sex: Female, Male Male	2 Participants n=93 Participants

PRIMARY outcome

Timeframe: 1 year

Population: Subjects withdrew prior primary outcome measurement therefore no data was obtained to report

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Outcome measures

Outcome data not reported

Adverse Events

Vemurafenib Administration

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Vemurafenib Administration n=2 participants at risk Vemurafenib 960 mg orally, twice a day
Gastrointestinal disorders Nausea/Vomiting/Weakness	50.0% 1/2 • Number of events 1 • Adverse event dating was collected through the duration of the study (approximately 14 months)

Other adverse events

Adverse event data not reported

Additional Information

Harriet Kluger

Yale University

Email: Harriet.Kluger@yale.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place