Brain Arteriovenous MalformationsPhase 2NCT06913725
An Exploratory Study to Evaluate the Efficacy and Safety of FCN-159 in Patients With Brain Arteriovenous Malformations
A Phase 2 study testing FCN-159 in people with Brain Arteriovenous Malformations, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Brain Arteriovenous Malformations
- What is being tested?
- FCN-159
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 24 Feb 2025to 30 Sep 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age ≥ 18 years and ≤ 60 years.
- Diagnosis of brain arteriovenous malformation (AVM) confirmed by DSA.
- Spetzler-Martin grade IV-V
- No history of rupture of the AVM vessels.
- No aneurysmal structures that are amenable to interventional embolization.
- No major surgery within the past 3 months.
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- Able to swallow and retain oral medication, with no significant gastrointestinal abnormalities that could affect drug absorption, such as malabsorption syndrome, bowel obstruction, or extensive gastrointestinal resection.
- Karnofsky Performance Score ≥ 50%
- The patient must have adequate organ and bone marrow function, and must not have received blood transfusions or used any supportive medications (such as cytokines or erythropoietin) to elevate white blood cells, platelets, or hemoglobin levels within 7 days prior to screening:
- Absolute neutrophil count ≥ 1.0 × 10^9/L. Hemoglobin ≥ 90 g/L. Platelet count ≥ 100 × 10^9/L. Total serum bilirubin ≤ 1.5 × upper limit of normal (ULN), patients with Gilbert's syndrome may have ≤ 3.0 × ULN.
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN.
- Albumin ≥ 3 g/dL. Creatinine < 1.5 × ULN or creatinine clearance ≥ 50 mL/min. Urine protein < 2+; if urine protein ≥ 2+, then 24-hour urine protein quantification must be ≤ 1g.
- Coagulation function: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 × ULN.
- The patient must voluntarily sign a written informed consent and be able to complete follow-up visits.
- For patients of reproductive potential: the patient must agree to use an effective contraceptive method, such as combined hormonal contraception, progestin-only contraception with ovulation suppression, intrauterine device (IUD), intrauterine system (IUS), bilateral tubal ligation, partner's vasectomy, or complete abstinence during the treatment period and for at least 90 days after the last dose of study treatment. Male patients should agree to avoid sperm donation for at least 90 days after the last dose of treatment.
Who cannot join
- Patients with multiple arteriovenous malformation lesions (excluding segmental lesions such as CAMS syndrome) or extracranial arteriovenous malformation lesions;
- Diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT), Capillary Malformation-Arteriovenous Malformation syndrome (CM-AVM), PTEN Hamartoma Tumor Syndrome (PHTS), or CLOVES syndrome;
- Patients who have received one of the following treatments after birth: a. Stereotactic radiation therapy, surgical treatment, or interventional embolization for BAVM; b. Pharmacological treatment for BAVM; c. Participation in other interventional clinical trials for BAVM;
- History of ruptured bleeding from malformed vascular clusters or other causes of brain hemorrhage, including subarachnoid hemorrhage, before the screening period;
- Patients with malignant tumors currently or within the past three years, except for non-melanoma skin basal cell carcinoma, in situ breast cancer, or in situ cervical cancer;
- Unable to undergo MRI scans and/or have contraindications to MRI (e.g., interference with MRI target lesion volume analysis due to prosthetics, braces, etc.);
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- Unable to undergo DSA and/or have contraindications to DSA (e.g., contrast agent allergy, coagulopathy, etc.);
- Poorly controlled epilepsy;
- Uncontrolled, unstable hypertension: Repeated measurements showing systolic blood pressure >140 mmHg or diastolic blood pressure >90 mmHg with antihypertensive treatment;
- Dysphagia, active gastrointestinal diseases, malabsorption syndrome, or other conditions affecting the absorption of study drugs;
- Piror or current retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), glaucoma, or other significant abnormalities in ophthalmologic examinations;
- Interstitial lung disease, including clinically significant radiation pneumonitis;
- Cardiac function or comorbidities that meet one of the following conditions: a. Three 12-lead ECG measurements conducted during the screening period at the study center, with an average QTcF >470 ms calculated using the QTcF formula for the instruments employed; presence of risk factors for QTcF prolongation, such as uncorrectable hypokalemia, inherited long QT syndrome, or taking drugs known to prolong QTcF (mainly Class Ia, Ic, III antiarrhythmic drugs). New York Heart Association (NYHA) functional classification ≥3 congestive heart failure; c. Clinically significant arrhythmias, including but not limited to complete left bundle branch block; d. Documented, clinically significant coronary artery disease, cardiomyopathy, or severe valvular heart disease; e. Echocardiogram showing left ventricular ejection fraction (LVEF) <50%; f. Bradycardia with a heart rate <50 beats per minute;
- Family history of sudden cardiac death before the age of 50 in a first-degree relative;
- Active bacterial, fungal, or viral infections, including active hepatitis B (positive hepatitis B surface antigen and hepatitis B virus DNA >1000 IU/ml, or meeting the study center's criteria for active hepatitis B infection), hepatitis C (positive hepatitis C virus RNA), or HIV infection (HIV positive);
- Pregnant or breastfeeding women;
- Known allergy to the study drug, other MEK1/2 inhibitors, or excipients;
- Any other factors determined by the investigator that may lead to forced discontinuation of the trial, such as severe diseases (including psychiatric disorders) requiring concomitant treatment, significant laboratory abnormalities, family or social factors that may affect the subject's safety or data collection.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
the Department of Neurosurgery, China International Neuroscience Institute, Xuanwu Hospital, Capital Medical University
Beijing, Beijing Municipality, No. 45, Changchun Street
Tao Hong, MD, +86-13810000653, hongtao.edu@gmail.com
Jiaxing Yu, MD, xwyujiaxing@163.com
About this study
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.
Read the detailed description
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.
- Conditions
- Brain Arteriovenous Malformations
- Being tested
- FCN-159
- Keywords
- Brain Arteriovenous Malformation, MEK Inhibitors, Pharmacological Treatment
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FCN-159Drug
An investigational oral MEK inhibitor
| Study group | Role | What they receive |
|---|---|---|
| Open label FCN-159 | Experimental | Participants receive 8mg FCN-159 orally once daily. Participants continue to reveive FCN-159 until disease progression, unacceptable toxicity or other end-of-treatment criteria. Receives: Drug: FCN-159 |
| No-treatment control | No intervention | Patients in the control group will undergo conservative observation throughout the study period. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR) assessed by Silence-MRA
Measured: 1 year
Secondary outcomes (15)
DSA-assessed ORR (Objective Response Rate).
Measured: 1 year
Changes in lesion volume at 3 months on Silence-MRA
Measured: 3 months
Changes in lesion volume at 6 months on Silence-MRA
Measured: 6 months
Changes in lesion volume at 12 months on Silence-MRA
Measured: 12 months
Changes in lesion volume at 12 months assessed by DSA
Measured: 12 months
Changes in blood flow velocity of feeding arteries assessed by MRI
Measured: 12 months
Changes in venous drainage flow velocity assessed by MRI
Measured: 12 months
Changes in microhemorrhage around the malformation assessed by MRI
Measured: 12 months
Changes in iron deposition around the malformation assessed by MRI.
Measured: 12 months
Changes in cerebral perfusion assessed by MRI
Measured: 12 months
Intracranial hemorrhage event.
Measured: 12 months
The occurrence of epilepsy events.
Measured: 12 months
Change in mRS score compared to baseline
Measured: 12 months
The types of adverse events occurring during treatment (TEAEs）
Measured: 12 months
The frequency of adverse events occurring during treatment (TEAEs)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2025
- Study start24 Feb 2025
- Primary completion31 Mar 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain Arteriovenous MalformationsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- The experimental group will receive the medication. In this study, the patients in the experimental group will be matched 1:1 with control group patients based on factors such as gender, age (±5 years), the anatomical location of the BAVM lesion, and the Spetzler-Martin grade. Patients in the control group will receive no treatment during the study period.
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06913725
- Sponsor's protocol ID
- hongtao
- Status last verified
- Mar 2025
- First submitted
- 18 Mar 2025
- Last updated
- 6 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: From 6 months post analysis and article publication, the following will be made available long-term for use by future researchers from a recognised research institution whose proposed use of the data has been ethically reviewed and approved by an independent committee and who accept Department of Neurosurgery, Xuanwu Hospital, Capital Medical University conditions for access: * Individual participant data that underlie the results reported in this article after de- identification * Trial protocol,Statistical Analysis Plan,PICF The Sponsor-Investigator will be the long-term custodian after the archive period has finished.
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study to Evaluate the Efficacy and Safety of FCN-159 in Patients With Brain Arteriovenous Malformations
- ClinicalTrials.gov ID
- NCT06913725
- Acronym / sponsor ID
- hongtao
- Conditions
- Brain Arteriovenous Malformations
- Keywords
- Brain Arteriovenous Malformation, MEK Inhibitors, Pharmacological Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Collaborators
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
- Study start
- 24 Feb 2025 (actual)
- Primary completion
- 31 Mar 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 6 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The experimental group will receive the medication. In this study, the patients in the experimental group will be matched 1:1 with control group patients based on factors such as gender, age (±5 years), the anatomical location of the BAVM lesion, and the Spetzler-Martin grade. Patients in the control group will receive no treatment during the study period.
| Arm | Type | Receives |
|---|---|---|
| Open label FCN-159 | Experimental | Drug: FCN-159Participants receive 8mg FCN-159 orally once daily. Participants continue to reveive FCN-159 until disease progression, unacceptable toxicity or other end-of-treatment criteria. |
| No-treatment control | No intervention | –Patients in the control group will undergo conservative observation throughout the study period. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (15 listed)
- Age ≥ 18 years and ≤ 60 years.
- Diagnosis of brain arteriovenous malformation (AVM) confirmed by DSA.
- Spetzler-Martin grade IV-V
- No history of rupture of the AVM vessels.
- No aneurysmal structures that are amenable to interventional embolization.
Key exclusion criteria (18 listed)
- Patients with multiple arteriovenous malformation lesions (excluding segmental lesions such as CAMS syndrome) or extracranial arteriovenous malformation lesions;
- Diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT), Capillary Malformation-Arteriovenous Malformation syndrome (CM-AVM), PTEN Hamartoma Tumor Syndrome (PHTS), or CLOVES syndrome;
- Patients who have received one of the following treatments after birth: a. Stereotactic radiation therapy, surgical treatment, or interventional embolization for BAVM; b. Pharmacological treatment for BAVM; c. Participation in other interventional clinical trials for BAVM;
- History of ruptured bleeding from malformed vascular clusters or other causes of brain hemorrhage, including subarachnoid hemorrhage, before the screening period;
- Patients with malignant tumors currently or within the past three years, except for non-melanoma skin basal cell carcinoma, in situ breast cancer, or in situ cervical cancer;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FCN-159An investigational oral MEK inhibitor | Drug | Open label FCN-159 |
Primary outcome
Objective Response Rate (ORR) assessed by Silence-MRA
Time frame: 1 year
15 secondary outcomes
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.
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2025.
- Individual participant dataSharing planned: Yes. From 6 months post analysis and article publication, the following will be made available long-term for use by future researchers from a recognised research institution whose proposed use of the data has been ethically reviewed and approved by an independent committee and who accept Department of Neurosurgery, Xuanwu Hospital, Capital Medical University conditions for access: * Individual participant data that underlie the results reported in this article after de- identification * Trial protocol,Statistical Analysis Plan,PICF The Sponsor-Investigator will be the long-term custodian after the archive period has finished.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FCN-159 in people with Brain Arteriovenous Malformations, run by Xuanwu Hospital, Beijing at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tao Hong, MD (+86-13810000653, hongtao.edu@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) assessed by Silence-MRA (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2025.