Can a cancer drug tame tangled blood vessels?
NCT ID NCT07037238
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This Phase 2 trial tests the oral drug everolimus in 10 adults with vascular malformations—abnormal clusters of blood vessels. The goal is to see if the drug can shrink these lesions and reduce complications like bleeding. Participants take everolimus daily for up to a year, with MRI scans tracking changes.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- everolimus (an oral drug that blocks a protein called mTOR)
- What this could lead to
- If it works, this could point toward a non-surgical treatment option for vascular malformations, potentially reducing lesion size and bleeding risk.
- What could go wrong
- This is a very small, early-phase trial with only 10 participants and no comparison group. The drug may not shrink lesions or could cause side effects like mouth sores, infections, or metabolic changes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 10 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2025
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria * 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; * 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. Exclusion Criteria: * 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; * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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the Department of Neurosurgery, China International Neuroscience Institute, Xuanwu Hospital, Capital Medical University
RECRUITINGBeijing, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New hope for rare blood vessel disorders: everolimus trial launches