A single-center, open-label, non-randomized controlled exploratory study is conducted in the Xuanwu Hospital Capital Medical University to evaluate the efficacy and safety of FCN-159 in patients with brain arteriovenous malformations (BAVM), aiming to overcome the current clinical challenges caused by the lack of available drugs for this disease.
This open-label Phase 2 study will evaluate the efficacy and safety of FCN-159 in participants with BAVM disease who currently deemed unsuitable for surgery, radiotherapy, interventional embolization, or lacking corresponding surgical treatment indications based on investigator judgment. Eligible patients have no prior rupture. This project plans to recruit 10 patients with BAVMs. They are 1:1 divided into FCN-159 group and no-treatment controlled group. It is stratified by gender, age (±5 years), anatomical site of the lesion, Spetzer-Martin grade. FCN-159 will be administered orally and treatment will be continuous, 28 days as a cycle. The major efficacy outcome measure is overall response rate (ORR) assessed by silence MRA. Participants will be evaluated radiologically at 3, 6, 12 treatment cycles after initiation of treatment, and then every 6 treatment cycles and EOT.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
10
An investigational oral MEK inhibitor
the Department of Neurosurgery, China International Neuroscience Institute, Xuanwu Hospital, Capital Medical University
Beijing, Beijing Municipality, China
RECRUITINGObjective Response Rate (ORR) assessed by Silence-MRA
Time frame: 1 year
DSA-assessed ORR (Objective Response Rate).
Time frame: 1 year
Changes in lesion volume at 3 months on Silence-MRA
Time frame: 3 months
Changes in lesion volume at 6 months on Silence-MRA
Time frame: 6 months
Changes in lesion volume at 12 months on Silence-MRA
Time frame: 12 months
Changes in lesion volume at 12 months assessed by DSA
Time frame: 12 months
Changes in blood flow velocity of feeding arteries assessed by MRI
Time frame: 12 months
Changes in venous drainage flow velocity assessed by MRI
Time frame: 12 months
Changes in microhemorrhage around the malformation assessed by MRI
Time frame: 12 months
Changes in iron deposition around the malformation assessed by MRI.
Time frame: 12 months
Changes in cerebral perfusion assessed by MRI
Time frame: 12 months
Intracranial hemorrhage event.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: 12 months
The occurrence of epilepsy events.
Time frame: 12 months
Change in mRS score compared to baseline
Time frame: 12 months
The types of adverse events occurring during treatment (TEAEs)
Time frame: 12 months
The frequency of adverse events occurring during treatment (TEAEs)
Time frame: 12 months