Trametinib Phase 2 Vascular Anomaly Trial, CHOP
Summary
ClinicalTrials.gov registration for Phase 2 study NCT07549646 at Children's Hospital of Philadelphia evaluating Trametinib in patients aged 2 months to 30 years with Ras/MAPK pathway driven vascular anomalies. The study will enroll participants for up to 3 years with up to 16 clinic visits and study drug administration for at least 2 years. Trametinib is FDA-approved for metastatic melanoma but is experimental for vascular anomalies, as the FDA has not approved this use.
“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).”
About this source
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What changed
ClinicalTrials.gov posted a Phase 2 study registration for NCT07549646 titled "VATCH Trametinib for Ras/MAPK Pathway VAs" conducted at Children's Hospital of Philadelphia. The single-arm study evaluates Trametinib efficacy and safety in patients 2 months to 30 years diagnosed with Ras/MAPK pathway driven vascular anomalies. Participants will take the study drug for at least 2 years up to a maximum of 3 years if there is positive response, attending up to 16 clinic visits lasting approximately 30 minutes to 2 hours. An optional component involves blood collection for biomarker testing.
Healthcare providers and clinical investigators should note that while Trametinib carries FDA approval for metastatic melanoma, its use in treating vascular anomalies is considered experimental under this IND. The study follows standard Phase 2 oncology trial design with regular safety monitoring through questionnaires and study drug diaries.
Archived snapshot
Apr 24, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs
Phase 2 NCT07549646 Kind: PHASE2 Apr 24, 2026
Abstract
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.
Conditions: Ras/MAPK Pathway Vascular Anomalies, Vascular Anomalies, Vascular Anomaly
Interventions: Trametinib
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