Ignite Creation Date:
2025-12-24 @ 7:45 PM
Ignite Modification Date:
2025-12-27 @ 9:28 AM
Study NCT ID:
NCT07072403
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-22
First Post:
2025-07-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Trametinib Treatment for Complicated Vascular Anomalies
Sponsor:
West China Hospital
Organization:
Study Overview
Official Title:
Safety and Efficacy of Trametinib for Complicated Vascular Anomalies: A Multicenter Prospective Study
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: