NCT07072403

Trametinib Treatment for Complicated Vascular Anomalies

Safety and Efficacy of Trametinib for Complicated Vascular Anomalies: A Multicenter Prospective Study

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: West China Hospital · Academic / Other
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

Summary

Complicated vascular anomalies have diverse etiologies and variable clinical manifestations, and no standardized treatment protocol has been established. Since most patients present with diffuse lesions that are difficult to resect surgically, identifying effective therapeutic strategies is of critical importance. This study aims to evaluate the safety and efficacy of systemic trametinib therapy in patients with complicated vascular anomalies.

Detailed description

In recent years, with the widespread application of molecular diagnostic technologies and the growing awareness of complicated vascular anomalies among clinicians, an increasing number of molecular alterations have been identified in these disorders. This progress has brought new hope for patients who previously lacked standardized treatment options. Multiple somatic point mutations have been discovered in complicated vascular anomalies, and case reports have demonstrated significant therapeutic benefits from mutation-based targeted therapies-particularly in patients with NRAS mutations treated with MEK inhibitors. However, to date, there have been no prospective studies evaluating the safety and efficacy of trametinib in pediatric patients with complicated vascular anomalies. Such research is urgently needed to provide robust clinical evidence to guide treatment decisions for this challenging patient population.

Conditions

• Vascular Anomalies

Interventions

Type	Name	Description
DRUG	Trametinib tablet	Drug is supplied in 0.5 mg and 2 mg tablets

Timeline

• Start date: 2025-07-07
• Primary completion: 2027-07-01
• Completion: 2027-09-01
• First posted: 2025-07-18
• Last updated: 2025-07-22

Locations

1 site across 1 country: China