Trametinib tablet: Drug is supplied in 0.5 mg and 2 mg tablets
Study 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.
Eligibility
Sex
ALL
Min age
1 Year
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* -Presenting a complicated vascular anomalies vascular anomalies with the following characteristics:
1. Male and female;
2. Between 1 and 18 years of age;
3. Complicated vascular anomalies vascular anomalies diagnosis was confirmed by local investigators and by consensus of our multidisciplinary vascular anomaly group at the West China Hospital of Sichuan University based on:
Biopsy; Compatible MRI findings; History and clinical features.
Exclusion Criteria:
1. Patients have allergy or contraindication to MEK inhibitor
2. Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;
3. Patients had a history of a major surgery within 2 weeks before enrollment;
4. Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of enrollment;
5. Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of trametinib.
6. Concurrent severe and/or uncontrolled medical diseases that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).
7. Patients with inadequate liver function:
Total bilirubin higher than or equal to 1.5 × the upper limit of the normal (ULN) for age and alanine aminotransferase and aspartate aminotransferase higher than or equal to 2.5 × the ULN for age.
8. Patients with inadequate renal function:
0-5 years of age maximum serum creatinine (mg/dL) of 0.8; 6-10 years of age maximum serum creatinine (mg/dL) of 1.0; 11-14 years of age maximum serum creatinine (mg/dL) of 1.2;
9. Adequate bone marrow function:
Absolute neutrophil count lower than 1 × 109/L;
10. History of a malignancy within 5 years;
11. HIV infection or known immunodeficiency;
12. Patients with an inability to participate in or follow-up during the study treatment and assessment plan;
13. Inability to give informed consent.
Primary outcome measure(s)
The proportion of patients achieving an objective response at month 12 — 12 months Objective response was defined as ≥20% reduction in lesion volume compared to that at baseline.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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