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Protocol No. APEC1621G

Principal Investigator DeSantes, Kenneth

Phase II

Age Group Both

Scope National

Sponsor Type National

Title NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Vemurafenib in Patients with Tumors Harboring Braf V600 Mutations

Objective This is a Phase 2 study of a drug called vemurafenib. In a Phase 2 study, the goal is to find out what effects, good and/or bad, a drug has on your tumor or type of cancer. We are using vemurafenib in this study because it has been shown to block the growth of cancer cells with mutations in the BRAF gene in test tubes and animals. The BRAF gene normally regulates the growth and activity of cells in the body. It is frequently mutated in many types of cancers. You are eligible for this study because your tumor was found to have a mutation in this gene. Vemurafenib is FDA approved for adults with melanoma, but not for use in this population. Vemurafenib is considered a study drug in the treatment of your tumor or type of cancer; we do not know if it will work against the type of tumor you have.

Treatment Vemurafenib will be given by mouth twice daily

Key Eligibility
  • Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to MATCH to APEC1621G based on the presence of a BRAF V600 mutation
  • Patients must be ≥ than 12 months and ≤ 21 years of age at the time of study enrollment
  • Patients must have a body surface area ≥ 0.37 m2 at enrollment
  • Patients must have radiographically measurable disease
  • Patients with neuroblastoma who do not have measurable disease but have MIBG+ evaluable disease are eligible
  • Patiets must have a Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age
  • Must have adequate Bone Marrow, Renal, Liver and Cardiac Function
  • Can not be Pregnancy or Breast-Feeding
  • Patients with known active cutaneous squamous cell carcinoma (includes keratoacanthoma or mixed keratocanthoma subtype) are not eligible. Patients who have fully excised lesions with dermatologic confirmation of absence of disease are eligible
  • Patients with low grade glioma patients (WHO grades I and II) are not eligible
  • Patients who have an uncontrolled infection are not eligible
  • Patients who have received a prior solid organ transplantation are not eligible

  • Applicable Disease Sites Anal; Any Site; Bladder; Brain/Central Nervous System; Breast; Cervix; Colon and Rectum; Endocrine cancers; Esophagus; Gastrointestinal cancers, other; Genitourinary cancers, other; Head and Neck; Hematologic cancers, other; Ill-Defined Sites; Kidney; Leukemia; Liver; Lung; Lymphoma; Melanoma/Skin cancer; Multiple Myeloma; Ovary; Pancreas; Prostate; Sarcoma; Stomach; Thyroid; Unknown Sites; Uterus

    Status Open

    Participating Institutions Gundersen Health System; UW Hospital and Clinics