Actively Recruiting
An Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular Malformations
Led by Xuanwu Hospital, Beijing · Updated on 2025-09-05
10
Participants Needed
1
Research Sites
21 weeks
Total Duration
On this page
Sponsors
X
Xuanwu Hospital, Beijing
Lead Sponsor
C
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of everolimus monotherapy in adults with vascular malformations. This single-arm exploratory trial is conducted by Xuanwu Hospital, Capital Medical University, and includes adult patients aged 18 to 65 who have vascular malformations confirmed by MRI and are unsuitable for effective surgical treatment. Participants will take oral everolimus 10 mg daily in continuous 28-day treatment cycles. Treatment will continue until disease progression, intolerable side effects, lack of benefit as judged by the investigator, study end, or other criteria for stopping treatment. The study includes assessments at multiple points, including after 3, 6, and 12 treatment cycles, and every 6 cycles thereafter. During the study, participants will undergo MRI scans to measure lesion volume and assess microbleeding, iron deposits, and hemorrhage risk. Clinical signs, symptom scores, and quality of life will also be evaluated. These evaluations are scheduled during screening, after certain treatment cycles, and at the end of treatment. The main outcome measured is the response rate based on MRI after one year of treatment.
CONDITIONS
Brief Title
An Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular Malformations
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 18 to 65 years
- No gender restrictions
- Diagnosed with vascular malformation by MRI
- No major surgery within the past 3 months
- Able to swallow and retain oral medication without significant gastrointestinal issues
- Able to provide peripheral blood samples for biomarker testing
- Adequate organ and bone marrow function as defined by specified lab values
- Voluntarily sign informed consent and able to complete follow-up
- For patients of childbearing potential: agree to use effective contraception or abstinence during treatment and for 90 days after last dose; males must refrain from sperm donation for 90 days after last dose
You will not qualify if you...
- Diagnosed with Hereditary Hemorrhagic Telangiectasia, Arteriovenous Malformation, or PTEN Hamartoma Tumor Syndrome
- Participation in other interventional trials targeting cerebral cavernous malformations
- Malignant tumors currently or within past 3 years, except certain treated skin or cervical cancers
- Unable to undergo MRI or have MRI contraindications
- Modified Rankin Scale score of 5, respiratory failure, or severe bleeding needing life support
- Severe renal failure or recent renal failure without treatment
- Severe hepatic failure or recent uncontrolled hepatic symptoms
- Use of other immunosuppressants or immunodeficiency
- Use of medications interfering with CYP3A4 enzyme or specific drugs like cisapride or metoclopramide
- Dysphagia, active gastrointestinal disorders, or malabsorption affecting drug absorption
- Interstitial pneumonitis or severe asthma
- Uncontrolled diabetes mellitus
- Active drug or alcohol dependence affecting adherence
- First-degree relatives with sudden cardiac death before age 50
- Active infections including hepatitis B, hepatitis C, or HIV
- Pregnant or breastfeeding women
- Known hypersensitivity to everolimus or related compounds
- Other severe diseases or issues 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 - Repeated 28-day cycles until disease progression or discontinuation
Participants receive everolimus orally once daily in continuous 28-day cycles. Treatment continues until disease progression, intolerable toxicity, lack of clinical benefit, study termination, or other discontinuation criteria.
Assessments at the end of treatment cycles 3, 6, 12, every 6 cycles thereafter, and at end-of-treatment visit
Trial Site Locations
Total: 1 location
1
the Department of Neurosurgery, China International Neuroscience Institute, Xuanwu Hospital, Capital Medical University
Beijing, China
Actively Recruiting
Research Team
T
Tao Hong, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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