Trametinib for Arterial Venous Malformation

Recruiting in Palo Alto (17 mi)

Overseen by Joyce Teng, MD, PhD, FAAD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Stanford University

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing Trametinib, a medication that may help treat arteriovenous malformation (AVM), a condition where blood vessels grow abnormally. The study focuses on children and adults with AVM because current treatments often fail, with symptoms returning quickly. Trametinib works by blocking proteins that cause abnormal blood vessel growth.

Research Team

Joyce Teng, MD, PhD, FAAD

Principal Investigator

Stanford University

Eligibility Criteria

This trial is for individuals aged 12-60 with a confirmed diagnosis of complicated extracranial AVMs. They must be able to take oral medication and have not received certain treatments like biologics, major surgery, or investigational drugs within specific time frames before enrollment. Women of childbearing potential must agree to use birth control.

Inclusion Criteria

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

I am taking steroids for hormone deficiencies as needed.

I haven't had chemotherapy that lowers my blood cell counts in the last 4 weeks.

Exclusion Criteria

I have previously taken or am allergic to MEK inhibitor therapy.

I have AVM caused by a genetic change.

I am currently taking medication that suppresses my immune system.

Treatment Details

Interventions

• Trametinib (MEK Inhibitor)

Trial Overview The study tests the safety and effectiveness of Trametinib tablets in treating Extracranial Arteriovenous Malformation (AVM), which can cause severe complications. The goal is to find a better treatment option than current therapies that often result in high recurrence rates.

Participant Groups

Group I: Oral Trametinib Experimental Treatment 1 Intervention

Patients will receive oral trametinib once daily

Trametinib is already approved in Canada, Japan for the following indications:

Approved in Canada as Mekinist for:

• Melanoma
• Non-small cell lung cancer

Approved in Japan as Mekinist for:

• Melanoma

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

Dr. Richard A. Miller

Stanford University

Chief Executive Officer since 2023

Stanford University, MD

Dr. Robert Schott

Stanford University

Chief Medical Officer since 2021

University of Michigan, MD

Boston Children's Hospital

Collaborator

Trials

801

Recruited

5,584,000+

Dr. Kevin B. Churchwell

Boston Children's Hospital

Chief Executive Officer since 2021

MD from Vanderbilt Medical School

Dr. Sarah Pitts

Boston Children's Hospital

Chief Medical Officer since 2019

MD from Harvard Medical School