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Clinical Trials/NCT04258046
NCT04258046
Completed
Phase 2

Phase II Clinical Trial of MEK Inhibitor Trametinib in the Treatment of Complicated Extracranial Arterial Venous Malformation (VM)

Stanford University2 sites in 1 country25 target enrollmentStarted: December 1, 2020Last updated:
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ConditionsVenous MalformationArterial Disease
InterventionsTrametinib tablet
DrugsTrametinib tablet

Overview

Phase
Phase 2
Status
Completed
Enrollment
25
Locations
2
Primary Endpoint
Disease response rate by investigator assessment at Month 6
OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

Brief Summary

Arteriovenous malformation (AVM) is a congenital vascular anomaly that progresses throughout life and causes complications including tissue destruction due to rapid overgrowth, bleeding, functional deficits, severe deformity and cardiac failure. Unfortunately, traditional managements have transient benefits with more than 90 recurrence rate within a year. Therefore, there is a significant unmet medical need. The purpose of this study is to assess the safety and efficacy of Trametinib in children and adults with Extracranial Arteriovenous Malformation (AVM).

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
12 Years to 60 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •Patient must be ≥ 12 years and ≤ 60 years
  • •Confirmed diagnosis of complicated extracranial AVMs made by a physician who is familiar with this condition.
  • •Genetic testing for mutations within MAP2K1 or remaining RAS/MAPK pathway is preferred but not mandatory
  • •Patient is able to swallow and/or retain oral medication via G tube
  • •All clinical and laboratory studies to determine eligibility will be performed within six weeks prior to enrollment unless otherwise indicated.
  • •Patients who have undergone surgical resection or interventional radiology procedures (sclerotherapy) of their AVM are eligible if they meet all inclusion criteria after these procedures
  • •At least 4 weeks from undergoing any major surgery
  • •Patients with endocrine deficiencies are allowed to receive physiologic or stress doses of steroids if necessary.
  • •Myelosuppressive chemotherapy: None within 4 weeks of entry into this study.
  • •At least 14 days since the completion of therapy with a biologic. For agents that have known adverse events occurring beyond 14 days after administration, this period must be extended beyond the time during which adverse events are known to occur. These patients must be discussed among PI and other investigators on a case-by-case basis.

Exclusion Criteria

  • •AVM due to germline mutation such as PTEN
  • •Prior MEK inhibitor therapy or have allergy or contraindication to MEK inhibitor
  • •Unable to swallow PO drugs or administer the drug via G tube
  • •Patients who have undergone major surgery ≤ 4 weeks prior to starting study treatment or who have not recovered from side effects of such procedure
  • •Patients with evidence of or history of cardiovascular risk
  • •Patients with retinal vein occlusion, hemorrhage or have a history of such conditions.
  • •Patients who are currently on other immunosuppressive medication(s)
  • •Patients who have an uncontrolled infection
  • •Unstable health status that may interfere with completing study
  • •Unable to travel to clinic as requested

Arms & Interventions

Oral Trametinib

Experimental

Patients will receive oral trametinib once daily

Intervention: Trametinib tablet (Drug)

Outcomes

Primary Outcomes

Disease response rate by investigator assessment at Month 6

Time Frame: Month 6

Combining a composite of radiographic, clinical, functional impairment, and quality of life measures.

Secondary Outcomes

  • Change from baseline in MRI Volumetric Scan Measurement of Targeted Disease Area(Month 12)
  • Disease response rate by investigator assessment at Month 12(Month 12)
  • Change from baseline in MRI Volumetric Scan Measurement of Targeted Disease Area(Month 6)

Investigators

Sponsor Class
Other
Responsible Party
Principal Investigator
Principal Investigator

Joyce Teng

Director of Pediatric Dermatology

Stanford University

Study Sites (2)

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