Actively Recruiting
An Exploratory Study to Evaluate the Efficacy and Safety of FCN-159 in Patients With Brain Arteriovenous Malformations
Led by Xuanwu Hospital, Beijing · Updated on 2025-04-06
10
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
Sponsors
X
Xuanwu Hospital, Beijing
Lead Sponsor
S
Shanghai Fosun Pharmaceutical Industry Development Co., Ltd
Collaborating Sponsor
AI-Summary
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.
CONDITIONS
Brief Title
An Exploratory Study to Evaluate the Efficacy and Safety of FCN-159 in Patients With Brain Arteriovenous Malformations
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years and older, up to 60 years.
- Confirmed diagnosis of brain arteriovenous malformation (AVM) by DSA.
- Spetzler-Martin grade IV-V.
- No history of rupture of AVM vessels.
- No aneurysmal structures suitable for interventional embolization.
- No major surgery within the past 3 months.
- Able to swallow and retain oral medication without significant gastrointestinal abnormalities.
- Karnofsky Performance Score of 50% or higher.
- Adequate organ and bone marrow function with specific blood count and liver, kidney, coagulation parameters met.
- Voluntary written informed consent and ability to complete follow-up visits.
- Agreement to use effective contraception during treatment and for at least 90 days after last dose if of reproductive potential.
You will not qualify if you...
- Multiple arteriovenous malformation lesions or extracranial AVM lesions.
- Diagnosis of Hereditary Hemorrhagic Telangiectasia, Capillary Malformation-Arteriovenous Malformation syndrome, PTEN Hamartoma Tumor Syndrome, or CLOVES syndrome.
- Prior treatments for BAVM including stereotactic radiation, surgery, embolization, pharmacological therapy, or participation in other interventional trials.
- History of ruptured bleeding or brain hemorrhage before screening.
- Malignant tumors within the past 3 years except certain non-invasive cancers.
- Inability or contraindications to undergo MRI or DSA scans.
- Poorly controlled epilepsy or unstable hypertension.
- Gastrointestinal conditions affecting drug absorption.
- Significant ophthalmologic abnormalities.
- Interstitial lung disease or radiation pneumonitis.
- Cardiac conditions including prolonged QTcF, heart failure, arrhythmias, coronary artery disease, low ejection fraction, or bradycardia.
- Family history of sudden cardiac death before age 50.
- Active infections including hepatitis B, hepatitis C, or HIV.
- Pregnant or breastfeeding women.
- Known allergies to study drug or related compounds.
- Other severe diseases or conditions affecting safety or data collection as determined by investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Continuous 28-day cycles until disease progression or end-of-treatment
Participants in the FCN-159 group receive an oral MEK inhibitor daily in continuous 28-day cycles until disease progression, unacceptable toxicity, or other end-of-treatment criteria. Participants in the no-treatment control group are observed without intervention.
Radiological evaluations at 3, 6, 12 cycles after treatment initiation, and then every 6 cycles until end of treatment
Trial Site Locations
Total: 1 location
1
the Department of Neurosurgery, China International Neuroscience Institute, Xuanwu Hospital, Capital Medical University
Beijing, Beijing Municipality, China, No. 45, Changchun Street
Actively Recruiting
Research Team
T
Tao Hong, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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