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Vemurafenib (R05185426) in Poor Performance Status Patients With Unresectable Locally Advanced or Metastatic Melanoma Harboring a V600E/K Mutation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01474551
Recruitment Status : Terminated (Lack of accrual)
First Posted : November 18, 2011
Results First Posted : April 30, 2015
Last Update Posted : November 18, 2015
Genentech, Inc.
Information provided by (Responsible Party):
Memorial Sloan Kettering Cancer Center

Brief Summary:
The purpose of this study is to find out what effects, good and/or bad, vemurafenib has on the patient and the melanoma. Specifically, the investigators want to know how well vemurafenib shrinks melanoma. The investigators also want to find out how well vemurafenib can improve how well the patient functions.

Condition or disease Intervention/treatment Phase
Melanoma Drug: vemurafenib Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Center Phase II Trial of Vemurafenib (R05185426) in Poor Performance Status Patients With Unresectable Locally Advanced or Metastatic Melanoma Harboring a V600E/K Mutation
Study Start Date : November 2011
Actual Primary Completion Date : March 2013
Actual Study Completion Date : March 2013

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Melanoma
MedlinePlus related topics: Melanoma
Drug Information available for: Vemurafenib

Arm Intervention/treatment
Experimental: vemurafenib
This is a single institution phase II trial in stage III or IV melanoma patients with poor ECOG performance status (3 or 4). Patients must have melanoma with a BRAFV600E or BRAFV600K or mutation with measurable disease not curable by surgery.
Drug: vemurafenib
All patients would be treated with vemurafenib given orally at 960 mg twice a day, which was the phase III dose. One cycle is 4 weeks long.

Primary Outcome Measures :
  1. Overall Objective Response [ Time Frame: 2 years ]
    The Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 will be used to determine treatment response. In order to be considered evaluable for response, a patient must have completed at least 1 cycle of therapy. Patients who do not complete a cycle of therapy can be replaced.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   19 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age >18 years old.
  • Histologic proof of melanoma reviewed and confirmed by MSKCC.
  • A confirmed EBRAFV600E or KBRAFV600K mutation.
  • Stage IV melanoma, or advanced stage III not curable by surgery. Patients with active CNS metastases will be allowed on the study.
  • Measurable disease by RECIST v1.1.
  • ECOG performance status 3 or 4. The basis for the grading of performance is strict; there must be clear justification of the performance status grade (e.g. patient is confined to bed > 50% of time, or cannot carry out ADLs, or is otherwise disabled by burden of disease such as requiring supplemental O2).
  • Patients must be able to swallow pills
  • Adequate hematologic, hepatic and renal function as defined by the following:
  • Absolute Neutrophil Count ≥ 1.0 x 109/L
  • Hemoglobin ≥8.0g/dL, occasional transfusions are acceptable as vemurafenib does not have significant hematologic toxicities.
  • Total bilirubin ≤2.0x the upper limit of normal, ≤3.0x the upper limit of normal if the patient has Gilbert's Syndrome.
  • Alkaline phosphatase ≤2.0x the upper limit of normal.
  • AST and ALT ≤2.0x the upper limit of normal.
  • Serum creatinine ≤ 1.5x the upper limit of normal.

Exclusion Criteria:

  • Uveal melanoma as primary.
  • Concurrent chemotherapy, immunotherapy, or radiotherapy.
  • Prior treatment with a RAF inhibitor. Other prior chemotherapy, immunotherapy, or radiotherapy will be allowed including prior treatment with a MEK inhibitor Patients must have had complete recovery from any adverse events or toxicities of prior cancer-directed therapies.
  • Pregnant or lactating women.
  • A second active malignancy. Prior malignancy will be allowed as long as the patient is known to be free of disease for at least 2 years. Patients with indolent B-cell malignancies will not be eligible.
  • QTc interval > 500 msec.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01474551

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United States, New York
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
Genentech, Inc.
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Principal Investigator: Paul Chapman, MD Memorial Sloan Kettering Cancer Center
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Memorial Sloan Kettering Cancer Center Identifier: NCT01474551    
Other Study ID Numbers: 11-091
First Posted: November 18, 2011    Key Record Dates
Results First Posted: April 30, 2015
Last Update Posted: November 18, 2015
Last Verified: October 2015
Keywords provided by Memorial Sloan Kettering Cancer Center:
skin cancer
Stage IV
advanced stage III
Additional relevant MeSH terms:
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Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Nevi and Melanomas
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action