Phase 2 Study of Oral FCN-159 for Adults Aged 18 to 60 With Brain Arteriovenous Malformations Unsuitable for Surgery or Other Treatments

What this Trial Is About

Researchers are evaluating the efficacy and safety of FCN-159, an oral investigational MEK inhibitor, in patients with brain arteriovenous malformations BAVM who are not suitable for surgery, radiotherapy, or embolization. This open-label Phase 2 study aims to address the lack of available drug treatments for BAVM by comparing FCN-159 with a no-treatment control group. The study will recruit 10 participants, stratified by gender, age, lesion site, and Spetzler-Martin grade, who have no prior rupture of the malformation. Participants in the treatment group will receive 8 mg of FCN-159 orally once daily in continuous 28-day cycles until disease progression, unacceptable side effects, or other end-of-treatment criteria are met. The control group will be observed without treatment during the study period. Radiological evaluations using Silence-MRA will occur at 3, 6, and 12 treatment cycles after starting therapy, then every 6 cycles and at the end of treatment to assess response. During the study, participants will undergo various assessments including imaging scans such as Silence-MRA and DSA, MRI evaluations of lesion volume and blood flow, and monitoring for events like intracranial hemorrhage and epilepsy. Safety and adverse events will be tracked throughout the 12-month period to measure overall response rate and other health outcomes. Participants must complete follow-up visits and meet specific health criteria to ensure safety and data quality.

An Exploratory Study to Evaluate the Efficacy and Safety of FCN-159 in Patients With Brain Arteriovenous Malformations