24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs

The purpose of this study is to assess the effectiveness and safety of Trametinib (the "Study Drug") in patients with Ras/MAPK pathway driven vascular anomalies (VA). Trametinib has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of metastatic melanoma. Its use in this study is considered experimental because the FDA has not approved the study drug for treating people with VAs. The study will enroll participants 2 months of age up to 30 years of age that have been diagnosed with Ras/MAPK pathway driven vascular anomalies. Study participation will last up to 3 years and will involve regular study visits to Children's Hospital of Philadelphia (CHOP) Philadelphia Campus. Participants will need to take the study drug Trametinib for at least 2 years, or up to 3 years in total, if there is a positive response. Participating in this research means you will attend up to 16 clinic visits. Most visits will take approximately 30 minutes, but some visits will take approximately 2 hours, because you will be asked to complete questionnaires about your experience. Participating in this research also means taking the study drug, having pictures taken, and completing study drug diaries. There is also an optional portion to this study that involves collecting blood for biomarker testing.

NCT Number	NCT07549646
Lead Sponsor	Children's Hospital of Philadelphia Collaborators: Novartis, CaNVAS (Consortium of Investigators of Vascular Anomalies), Baylor College of Medicine, Arkansas Children's Hospital Research Institute, Nationwide Children's Hospital, Nemours Children's Health, Boston Children's Hospital, NYU Langone Health, Children's Healthcare of Atlanta, Oregon Health and Science University, Children's Hospital Colorado, Riley Children's Health, Children's Hospital of Los Angeles (CHLA), Seattle Children's Hospital, Children's Hospital of Orange County, Children's Wisconsin - Milwaukee, University of North Carolina, Children's Hospital Medical Center, Cincinnati, University of California, San Francisco, Columbia University, Children's Hospital of Pittsburg, Dell Children's Medical Center of Central Texas, Vanderbilt University Medical Center, Helen DeVos Children's Hospital, Washington University School of Medicine, Lucile Packard Children's Hospital Stanford
Conditions	Ras/MAPK Pathway Vascular Anomalies, Vascular Anomalies, Vascular Anomaly
Enrollment	45 participants
Start Date	2025-08-27
Primary Completion	2029-08-27 (estimated)
Study Completion	2030-08-27 (estimated)
Updated on ClinicalTrials.gov	2026-04-24