Trametinib in the Treatment of Complicated Extracranial Arterial Venous Malformation


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Trial ID: NCT04258046


Arteriovenous malformation (AVM) is a congenital vascular anomaly that progresses throughout life and causes complications including tissue destruction due to rapid overgrowth, bleeding, functional deficits, severe deformity and cardiac failure. Unfortunately, traditional managements have transient benefits with more than 90 recurrence rate within a year. Therefore, there is a significant unmet medical need. The purpose of this study is to assess the safety and efficacy of Trametinib in children and adults with Extracranial Arteriovenous Malformation (AVM).

Official Title

Phase II Clinical Trial of MEK Inhibitor Trametinib in the Treatment of Complicated Extracranial Arterial Venous Malformation (VM)

Stanford Investigator(s)

Joyce Teng, MD, PhD
Joyce Teng, MD, PhD

Professor of Dermatology and, by courtesy, of Pediatrics

Huy M. Do, MD
Huy M. Do, MD

Professor of Radiology (Neuroimaging and Neurointervention) and, by courtesy, of Neurosurgery and of Otolaryngology - Head & Neck Surgery (OHNS)


Inclusion Criteria:

   - Patient must be ≥ 12 years and ≤ 60 years

   - Confirmed diagnosis of complicated extracranial AVMs made by a physician who is
   familiar with this condition.

   - Genetic testing for mutations within MAP2K1 or remaining RAS/MAPK pathway is preferred
   but not mandatory

   - Patient is able to swallow and/or retain oral medication via G tube

   - All clinical and laboratory studies to determine eligibility will be performed within
   six weeks prior to enrollment unless otherwise indicated.

   - Patients who have undergone surgical resection or interventional radiology procedures
   (sclerotherapy) of their AVM are eligible if they meet all inclusion criteria after
   these procedures

   - At least 4 weeks from undergoing any major surgery

   - Patients with endocrine deficiencies are allowed to receive physiologic or stress
   doses of steroids if necessary.

   - Myelosuppressive chemotherapy: None within 4 weeks of entry into this study.

   - At least 14 days since the completion of therapy with a biologic. For agents that have
   known adverse events occurring beyond 14 days after administration, this period must
   be extended beyond the time during which adverse events are known to occur. These
   patients must be discussed among PI and other investigators on a case-by-case basis.

   - Patients must not have received an investigational drug within the prior 4 weeks.

   - Not within 6 months prior to entering study if AVM is within field of radiation

Exclusion Criteria:

   - AVM due to germline mutation such as PTEN

   - Prior MEK inhibitor therapy or have allergy or contraindication to MEK inhibitor

   - Unable to swallow PO drugs or administer the drug via G tube

   - Patients who have undergone major surgery ≤ 4 weeks prior to starting study treatment
   or who have not recovered from side effects of such procedure

   - Patients with evidence of or history of cardiovascular risk

   - Patients with retinal vein occlusion, hemorrhage or have a history of such conditions.

   - Patients who are currently on other immunosuppressive medication(s)

   - Patients who have an uncontrolled infection

   - Unstable health status that may interfere with completing study

   - Unable to travel to clinic as requested

   - Patients unwilling or unable to comply with the protocol, or who in the opinion of the
   investigator may not be able to comply with the safety monitoring requirements of the

   - Females of child-bearing potential must be willing to practice acceptable methods of
   birth control.

   - Additionally, females of childbearing potential must have a negative serum pregnancy
   test result from 7 days prior to the initiation of the medication to 3 months after
   the final administration of the medication. Males or females of reproductive potential
   may not participate unless they have agreed to use an effective contraceptive method
   during the period when they are receiving the study drug and for 3 months thereafter.


drug: Trametinib tablet


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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Pediatric Derm Study