Vascular MalformationsPhase 2NCT07037238
An Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular Malformations
A Phase 2 study testing Everolimus 10mg daily in people with Vascular Malformations, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
This study is a single-arm exploratory trial conducted by Xuanwu Hospital, Capital Medical University, aiming to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Vascular Malformations
- What is being tested?
- Everolimus 10mg daily
- 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
- 1 Jul 2025to 31 Dec 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged ≥18 and ≤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, with no significant gastrointestinal abnormalities that may affect drug absorption, such as malabsorption syndrome, intestinal obstruction, or extensive gastrointestinal resection;
- Able to provide peripheral blood samples for biomarker testing at a central laboratory;
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- Patients must have adequate organ and bone marrow function, and must not have received blood transfusions or any supportive treatments (e.g., cytokines or erythropoietin) to increase white blood cells, platelets, or hemoglobin levels within 7 days before screening tests:Absolute neutrophil count ≥1.0×10⁹/L;Hemoglobin ≥90 g/L; Platelets ≥100×10⁹/L;Total bilirubin ≤1.5× upper limit of normal (ULN), or ≤3.0× ULN for patients with Gilbert's syndrome;AST and ALT ≤2.5× ULN;Albumin ≥3 g/dL; Serum creatinine <1.5× ULN or creatinine clearance ≥50 mL/min;Urine protein <2+; if ≥2+, then 24-hour urine protein must be ≤1 g;Coagulation: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤1.5× ULN.
- Patients must voluntarily sign the written informed consent form and be able to complete follow-up;
- For patients of childbearing potential: they must agree to use highly effective contraceptive methods, such as combined hormonal contraception, progestogen-only hormonal contraception associated with ovulation inhibition, intrauterine devices (IUDs), intrauterine hormonal systems (IUS), bilateral tubal occlusion, or partner vasectomy, or to practice sexual abstinence during the treatment period and for at least 90 days after the last dose. Male patients must agree to refrain from sperm donation for at least 90 days after the last dose.
Who cannot join
- Diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT), Arteriovenous Malformation (AVM), or PTEN Hamartoma Tumor Syndrome (PHTS);
- Patients who have previously received any of the following treatments after birth: participation in other interventional clinical trials targeting cerebral cavernous malformations (CM);
- Presence of malignant tumors currently or within the past three years, except for curatively treated non-melanoma skin basal cell carcinoma, ductal carcinoma in situ of the breast, or cervical carcinoma in situ;
- Unable to undergo MRI scans and/or have contraindications for MRI (e.g., interference from prosthetics, orthodontic devices, etc., affecting target lesion volume analysis on MRI);
- Modified Rankin Scale (mRS) score of 5, respiratory failure, or currently experiencing severe bleeding requiring life-support treatment;
- Severe renal failure (e.g., creatinine clearance \[CrCl] < 30 mL/min, or significantly elevated serum creatinine not correctable by other means), recent history (within past 3 months) of renal failure or end-stage renal disease without effective treatment, or currently undergoing dialysis;
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- Severe hepatic failure, including but not limited to: Child-Pugh Class C or higher, recent (within 3 months) uncontrolled symptoms related to hepatic failure such as ascites, jaundice, coagulopathy, or hepatic encephalopathy, or patients requiring liver transplantation;
- Currently using other immunosuppressants or patients with immunodeficiency;
- Patients requiring use of medications that interfere with or inhibit CYP3A4 enzyme activity, or medications such as cisapride or metoclopramide;
- Patients with dysphagia, active gastrointestinal disorders, malabsorption syndrome, or other conditions that may affect the absorption of the investigational drug;
- Interstitial pneumonitis, including clinically significant radiation pneumonitis;
- Severe asthma;
- Uncontrolled diabetes mellitus;
- Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements;
- First-degree relatives with a history of sudden cardiac death before the age of 50. First-degree relatives are defined as those with a direct bloodline, such as parents and children, grandparents and grandchildren, or maternal grandparents and maternal grandchildren;
- Active bacterial, fungal, or viral infections, including active hepatitis B (HBsAg positive with HBV DNA > 1000 IU/mL or meeting local diagnostic criteria for active HBV infection), hepatitis C (HCV RNA positive), or HIV infection (HIV positive);
- Pregnant or breastfeeding women. Any patient who becomes pregnant during the trial must withdraw from the study;
- Known hypersensitivity to everolimus, other rapamycin derivatives, or any of the excipients in this product. Observed allergic reactions to everolimus or related compounds include but are not limited to: hypersensitivity, dyspnea, flushing, chest pain, or angioedema (e.g., airway or tongue swelling with or without respiratory compromise);
- Other factors, as determined by the investigator, that may lead to early study termination, such as presence of other severe diseases (including psychiatric disorders) requiring concomitant treatment, significantly abnormal lab values, or social/family issues that may affect patient 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
Tao Hong, MD, +86-13810000653, hongtao.edu@gmail.com
Jian Ren, MD, +86 13269087795, renjian1129@126.com
About this study
This study is a single-arm exploratory trial conducted by Xuanwu Hospital, Capital Medical University, aiming to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations.
Read the detailed description
This study is a single-arm exploratory trial designed to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations.
A total of 10 participants aged 18 to 65 years with a confirmed diagnosis of vascular malformation will be enrolled. Eligible patients must be deemed by the investigator to be unsuitable for effective surgical treatment. After screening, qualified participants will receive oral everolimus once daily in continuous cycles of 28 days. The daily dosage of everolimus for adult patients will be 10 mg. Treatment will continue until disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, study termination, or any other pre-defined discontinuation criteria-whichever occurs first.
The primary objective of this study is to assess the therapeutic efficacy of everolimus in patients with vascular malformations, primarily through evaluation of lesion volume using MRI sequences. All participants will undergo MRI-based target lesion volume analysis and assessments of microbleeding, iron deposition, and hemorrhage risk during the screening period, at the end of treatment cycles 3, 6, and 12, every 6 cycles thereafter, and at the end-of-treatment (EOT) visit. Clinical signs, symptom scores, and quality-of-life improvements will also be evaluated. The acceptable window for these assessments is ±7 days. If a participant has not undergone efficacy evaluation within 3 months, a final assessment should be conducted at the EOT visit.
- Conditions
- Vascular Malformations
- Being tested
- Everolimus 10mg daily
- Keywords
- Vascular malformations, mTOR Inhibitors, Pharmacological Treatment
- Medical subject headings
- Vascular Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Everolimus 10mg dailyDrug
An oral mTOR inhibitor
| Study group | Role | What they receive |
|---|---|---|
| An Open-Label, Single-Arm Exploratory Study of Everolimus | Experimental | Subjects will receive everolimus 10 mg orally once daily in continuous 28-day treatment cycles, until the occurrence of disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, end of study, or other protocol-specified criteria for treatment discontinuation (whichever occurs first). Receives: Drug: Everolimus 10mg daily |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR) assessed by MRI
Measured: 1 year
Secondary outcomes (9)
Duration of Response (DOR) and 1-year DOR rate assessed by MRI
Measured: 1 year
Mean Percentage Reduction in Target Lesion Volume at 3, 6, and 12 Months Assessed by MRI
Measured: 1 year
Changes in Perilesional Microbleeds and Iron Deposition at 3, 6, and 12 Months Assessed by MRI QSM Sequence.
Measured: 1 year
Changes in lesion hemorrhage risk during the follow-up period
Based on MRI, imaging evidence of hemorrhage was defined as changes meeting the following criteria: (1) an increase in lesion size or alteration in its shape; and (2) a change in signal intensity, primarily characterized by a shift from hypointensity to hyperintensity, especially on T1-weighted sequences. Hemorrhage rates were calculated at 3, 6, and 12 months during follow-up to comprehensively evaluate the temporal changes in hemorrhagic risk of the lesion.
Measured: 1 year
The frequency and severity of intracranial hemorrhage events during the follow-up period were assessed through clinical records, imaging examinations (MRI), and the mRS scoring system.
Measured: 1 year
Changes in patients' clinical signs and symptom scores from baseline.
The effectiveness of the medication and patient prognosis were comprehensively assessed by comparing the clinical symptoms and mRS scores of patients after treatment with baseline clinical symptoms and mRS scores. The mRS scale ranges from 0 to 5, with a decrease in score indicating a more favorable prognosis.
Measured: 1 year
Disease Control Rate (DCR) assessed by MRI
Measured: 1 year
Progression-Free Survival (PFS) and 1-year PFS rate assessed by MRI
Measured: 1 year
The frequency and types of seizures during the follow-up period were recorded and assessed through seizure event logs and classification of seizure types.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2025
- Study start1 Jul 2025
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vascular MalformationsStudies of the same condition, open and under way.
- More trials run by Xuanwu Hospital, BeijingThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Model description
- A total of 10 participants aged 18 to 65 years with a confirmed diagnosis of vascular malformation will be enrolled. Eligible patients must be deemed by the investigator to be unsuitable for effective surgical treatment. After screening, qualified participants will receive oral everolimus once daily in continuous cycles of 28 days. The daily dosage of everolimus for adult patients will be 10 mg. Treatment will continue until disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, study termination, or any other pre-defined discontinuation criteria-whichever occurs first.
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07037238
- Sponsor's protocol ID
- hongtao1
- Status last verified
- Sep 2025
- First submitted
- 27 May 2025
- Last updated
- 5 Sep 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 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular Malformations
- ClinicalTrials.gov ID
- NCT07037238
- Acronym / sponsor ID
- hongtao1
- Conditions
- Vascular Malformations
- Keywords
- Vascular malformations, mTOR 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
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
- Study start
- 1 Jul 2025 (actual)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 25 Jun 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.
This study is a single-arm exploratory trial conducted by Xuanwu Hospital, Capital Medical University, aiming to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations.
Detailed description
This study is a single-arm exploratory trial designed to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations.
A total of 10 participants aged 18 to 65 years with a confirmed diagnosis of vascular malformation will be enrolled. Eligible patients must be deemed by the investigator to be unsuitable for effective surgical treatment. After screening, qualified participants will receive oral everolimus once daily in continuous cycles of 28 days. The daily dosage of everolimus for adult patients will be 10 mg. Treatment will continue until disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, study termination, or any other pre-defined discontinuation criteria-whichever occurs first.
The primary objective of this study is to assess the therapeutic efficacy of everolimus in patients with vascular malformations, primarily through evaluation of lesion volume using MRI sequences. All participants will undergo MRI-based target lesion volume analysis and assessments of microbleeding, iron deposition, and hemorrhage risk during the screening period, at the end of treatment cycles 3, 6, and 12, every 6 cycles thereafter, and at the end-of-treatment (EOT) visit. Clinical signs, symptom scores, and quality-of-life improvements will also be evaluated. The acceptable window for these assessments is ±7 days. If a participant has not undergone efficacy evaluation within 3 months, a final assessment should be conducted at the EOT visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A total of 10 participants aged 18 to 65 years with a confirmed diagnosis of vascular malformation will be enrolled. Eligible patients must be deemed by the investigator to be unsuitable for effective surgical treatment. After screening, qualified participants will receive oral everolimus once daily in continuous cycles of 28 days. The daily dosage of everolimus for adult patients will be 10 mg. Treatment will continue until disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, study termination, or any other pre-defined discontinuation criteria-whichever occurs first.
| Arm | Type | Receives |
|---|---|---|
| An Open-Label, Single-Arm Exploratory Study of Everolimus | Experimental | Drug: Everolimus 10mg dailySubjects will receive everolimus 10 mg orally once daily in continuous 28-day treatment cycles, until the occurrence of disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, end of study, or other protocol-specified criteria for treatment discontinuation (whichever occurs first). |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients aged ≥18 and ≤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, with no significant gastrointestinal abnormalities that may affect drug absorption, such as malabsorption syndrome, intestinal obstruction, or extensive gastrointestinal resection;
Key exclusion criteria (19 listed)
- Diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT), Arteriovenous Malformation (AVM), or PTEN Hamartoma Tumor Syndrome (PHTS);
- Patients who have previously received any of the following treatments after birth: participation in other interventional clinical trials targeting cerebral cavernous malformations (CM);
- Presence of malignant tumors currently or within the past three years, except for curatively treated non-melanoma skin basal cell carcinoma, ductal carcinoma in situ of the breast, or cervical carcinoma in situ;
- Unable to undergo MRI scans and/or have contraindications for MRI (e.g., interference from prosthetics, orthodontic devices, etc., affecting target lesion volume analysis on MRI);
- Modified Rankin Scale (mRS) score of 5, respiratory failure, or currently experiencing severe bleeding requiring life-support treatment;
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 |
|---|---|---|
| Everolimus 10mg dailyAn oral mTOR inhibitor | Drug | An Open-Label, Single-Arm Exploratory Study of Everolimus |
Primary outcome
Objective Response Rate (ORR) assessed by MRI
Time frame: 1 year
9 secondary outcomes
Duration of Response (DOR) and 1-year DOR rate assessed by MRI
Time frame: 1 year
Mean Percentage Reduction in Target Lesion Volume at 3, 6, and 12 Months Assessed by MRI
Time frame: 1 year
Changes in Perilesional Microbleeds and Iron Deposition at 3, 6, and 12 Months Assessed by MRI QSM Sequence.
Time frame: 1 year
Changes in lesion hemorrhage risk during the follow-up period
Time frame: 1 year
The frequency and severity of intracranial hemorrhage events during the follow-up period were assessed through clinical records, imaging examinations (MRI), and the mRS scoring system.
Time frame: 1 year
Changes in patients' clinical signs and symptom scores from baseline.
Time frame: 1 year
Disease Control Rate (DCR) assessed by MRI
Time frame: 1 year
Progression-Free Survival (PFS) and 1-year PFS rate assessed by MRI
Time frame: 1 year
The frequency and types of seizures during the follow-up period were recorded and assessed through seizure event logs and classification of seizure types.
Time frame: 1 year
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 5 Sep 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 Everolimus 10mg daily in people with Vascular 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 9 inclusion and 19 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 5 Sep 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 MRI (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 5 Sep 2025.