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Phase 2Recruiting

24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs

VATCH (Vascular Anomaly Analysis for Therapy Choice): A Phase II Study of Trametinib Treatment in Subjects With Ras/MAPK Pathway Driven Vascular Anomalies

Sponsor
Children's Hospital of Philadelphia (Academic or other)
Enrollment
45 planned
Conditions
Ras/MAPK Pathway Vascular Anomalies; Vascular Anomalies; Vascular Anomaly
Interventions
Trametinib
Ages
children, adults
Registry
NCT07549646

Full record on ClinicalTrials.gov

From the registry

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.…

Primary outcomes

  • Percentage of subjects with a Substantial Response or Intermediate Response to Trametinib using an Individualized Response Criteria
  • Relative size of target lesion(s) as determined by radiologic assessment
  • Quality of life as evaluated by PROMIS Patient Reported Outcome (PRO) questionnaire T-scores

History

  1. Study completion expected

  2. Primary completion expected

  3. Last registry update

  4. Registered

  5. Started