Vertebrobasilar DolichoectasiaPhase 2NCT07634120
Vertebrobasilar Dolichoectasia Treatment With Sirolimus
A Phase 2 study testing Sirolimus in people with Vertebrobasilar Dolichoectasia, run by Huashan Hospital at 1 site in China.
Trial summary
The aim of this pilot trial is to assess the efficacy of sirolimus in reducing wall enhancement in vertebrobasilar dolichoectasia(VBD) on 5 T high-resolution magnetic resonance vessel wall imaging(HR-VWI) via anti-inflammatory mechanisms, clarify the efficacy of sirolimus in delaying the progression of VBD, evaluate… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Vertebrobasilar Dolichoectasia
- What is being tested?
- Sirolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- Jun 2026to Dec 2027
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age≥18 years, any gender;
- Patients with VBD confirmed by DSA/CTA/MRA;
- No history of VBD rupture and no surgical treatment for VBD;
- mRS<4;
- Positive plasma SGK1;
- History of posterior circulation infarction or accompanied by VBD-related symptoms/signs;
- Currently and in the future, need to take antiplatelet and statin drugs simultaneously, or currently and in the future, do not need to take antiplatelet and statin drugs;
- Capable of signing an informed consent form with the accompaniment and understanding of a guardian.
Who cannot join
- History of malignant tumors;
- Pregnancy or lactation;
- Sirolimus allergy;
- Hydrocephalus requiring urgent surgical intervention or respiratory failure requiring life support treatment;
- Abnormal hepatic and/or renal function (serum transaminase > 40 U/L; serum creatinine > 110 μmol/L); and/or abnormal white blood cells/platelets (white blood cells count < 3.5 × 10⁹/L or > 9.5 × 10⁹/L; platelets count < 100 × 10⁹/L or > 300 × 10⁹/L);
- History of immunosuppressive therapy;
Show all 19Show fewer
- Acute cerebral infarction within the last month or definite high signal on DWI indicating acute or subacute cerebral infarction;
- Acute stage of intracranial hemorrhage as indicated by CT;
- History of VBD rupture or surgery;
- Presence of acute active infection (such as severe bacterial, viral or fungal infection);
- Uncontrolled diabetes (HbA1c≥7%);
- History of liver or lung transplantation;
- Presence of organic heart disease;
- History of arteriovenous thrombosis;
- Patients taking only antiplatelet drugs or only statin drugs;
- Patients taking or needing to take CYP3A4 inhibitors (ketoconazole, itraconazole, voriconazole, clarithromycin, erythromycin, telithromycin, ritonavir, atazanavir, diltiazem, verapamil, cyclosporine, amiodarone, sildenafil, grapefruit juice, etc.) or CYP3A4 inducers (rifampicin, rifabutin, phenobarbital, phenytoin, carbamazepine, dexamethasone, St. John's wort, etc.);
- Currently participating in other clinical studies;
- Presence of contraindications for MRI examination;
- Other situations not suitable for inclusion.
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
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, 200040
Wei Zhu, +8615868058299, 19111220090@fudan.edu.cn
About this study
Official title: Vertebrobasilar Dolichoectasia Treatment With Sirolimus: a Pilot Trial Based On 5.0 T MRI
The aim of this pilot trial is to assess the efficacy of sirolimus in reducing wall enhancement in vertebrobasilar dolichoectasia(VBD) on 5 T high-resolution magnetic resonance vessel wall imaging(HR-VWI) via anti-inflammatory mechanisms, clarify the efficacy of sirolimus in delaying the progression of VBD, evaluate the safety of sirolimus in the treatment of VBD.
- Conditions
- Vertebrobasilar Dolichoectasia
- Being tested
- Sirolimus
- Medical subject headings
- Vertebrobasilar Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Sirolimus is an mTORC1/ mTORC2 inhibitor that has received approval from the U.S. Food and Drug Administration (FDA) and has recently been successfully used to treat lymphatic malformations and venous/lymphatic malformations associated with the same PIK3CA GOF mutations. Participants of sirolimus group will receive oral sirolimus 2mg/d continuously for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| sirolimus group | Experimental | Participants will receive oral sirolimus 2mg/d continuously for 6 months. Receives: Drug: Sirolimus |
| control group | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular CAWE (3D circumferential arterial wall enhancement: mean signal intensity in T1+Gd images) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 3 and 6 months
Longitudinal changes of SAWE on 5T HR-VWI VBD following 3 and 6 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular SAWE (specific contrast uptake arterial wall enhancement: the difference in mean signal intensity between T1 and T1+Gd) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 3 and 6 months
Longitudinal changes of FAWE in VBD on 5T HR-VWI 3 and 6 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular FAWE (focal arterial wall enhancement: areas of the diseased artery with increased AWE) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 3 and 6 months
Longitudinal changes of WEVR on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular WEVR (3D arterial wall enhancement volume rate) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 3 and 6 months
Secondary outcomes (15)
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular CAWE (3D circumferential arterial wall enhancement: mean signal intensity in T1+Gd images) at 12 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 12 months
Longitudinal changes of SAWE on 5T HR-VWI VBD following 12 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular SAWE (specific contrast uptake arterial wall enhancement: the difference in mean signal intensity between T1 and T1+Gd) at 12 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 12 months
Longitudinal changes of FAWE on 5T HR-VWI VBD 12 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular FAWE (focal arterial wall enhancement: areas of the diseased artery with increased AWE) at 12 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 12 months
Longitudinal changes of WEVR on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of vascular WEVR (3D arterial wall enhancement volume rate) at 12 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Measured: 12 months
Longitudinal changes of vascular dilation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Using 5T MRA, we quantify longitudinal changes of dilation(maximum diameter of the intracranial segment of the vertebral artery and basilar artery) of diseased vessel in VBD at 3, 6, and 12 months following sirolimus treatment.
Measured: 3, 6, and 12 months
Longitudinal changes of vascular tortuosity on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Using 5T MRA, we quantify longitudinal changes of tortuosity(displacement distance of the intracranial segment of the vertebral artery and basilar artery) of diseased vessel in VBD at 3, 6, and 12 months following sirolimus treatment.
Measured: 3, 6, and 12 months
Longitudinal changes of vascular elongation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Using 5T MRA, we quantify longitudinal changes of elongation(length of the basilar artery) of diseased vessel in VBD at 3, 6, and 12 months following sirolimus treatment.
Measured: 3, 6, and 12 months
Longitudinal changes of thrombus volume in VBD following 3, 6, and 12 months of sirolimus treatment.
Using 5T HR-VWI, we quantify longitudinal changes of thrombus volume in VBD at 3, 6, and 12 months following sirolimus treatment.
Measured: 3, 6, and 12 months
Longitudinal changes in plasma SGK1 levels following 1, 3, 6, and 12 months of sirolimus treatment in VBD patients.
Longitudinal changes in plasma SGK1 levels (quantitative analysis of plasma SGK1 was performed via western blot) following 1, 3, 6, and 12 months of sirolimus treatment in VBD patients.
Measured: 1, 3, 6, and 12 months
Incidence of ischemic stroke in VBD patients at 3, 6, and 12 months.
Incidence of ischemic stroke(newly developed infarct lesion confirmed by CT/MRI after onset of relevant symptoms/signs or newly developed infarct lesion confirmed by follow-up MRI at 3, 6, or 12 months) in VBD patients at 3, 6, and 12 months.
Measured: 3, 6, and 12 months
Incidence of subarachnoid hemorrhage associated with VBD rupture in VBD patients at 3, 6, and 12 months.
Incidence of subarachnoid hemorrhage associated with VBD rupture(newly developed subarachnoid hemorrhage confirmed by CT/MRI after onset of relevant symptoms/signs or newly developed subarachnoid hemorrhage confirmed by follow-up MRI at 3, 6, or 12 months) at 3, 6, and 12 months.
Measured: 3, 6, and 12 months
modified Rankin Scale in VBD patients at 3, 6, and 12 months.
modified Rankin Scale (mRS), a 7-level, clinician-reported, measure of global disability is measured in VBD patients at 3, 6, and 12 months.
Measured: 3, 6, and 12 months
Five-level EuroQol five-dimensional questionnaire in VBD patients at 3, 6, and 12 months.
Five-level EuroQol five-dimensional questionnaire(EQ-5D-5L), a 5-level, measure of quality of life, is performed in VBD patients at 3, 6, and 12 months.
Measured: 3, 6, and 12 months
The safety of sirolimus in VDB patients.
A serious adverse event (SAE) is any untoward medical occurrence that meets one or more of the following criteria, regardless of suspected causal relationship to the study intervention: (1) results in death; (2) is life-threatening; (3) requires inpatient hospitalization or prolongation of existing hospitalization; (4) results in persistent or significant disability or incapacity, or substantially disrupts normal life functions; or (5) constitutes an important medical event that, based on appropriate medical judgment, may jeopardize the patient's health or require medical or surgical intervention to prevent one or more of the outcomes listed in (1)-(4). SAEs will be actively monitored and systematically assessed at all scheduled follow-up visits (at 1, 3, 6, and 12 months post-baseline). In addition, participants are instructed to report any suspected SAE immediately to the study team via a dedicated 24/7 telephone hotline. To ensure timely detection and documentation.
Measured: 1, 3, 6, and 12 months
The tolerability of sirolimus in VDB patients.
Based on prior clinical experience and sirolimus trial reports, adverse events (AEs) include oral ulcers, upper respiratory tract infections, headaches, respiratory diseases, stomatitis, seizures, and fever. Other commonly reported AEs also encompass nausea, vomiting, fatigue, dizziness, diarrhea, and mild allergic reactions such as rash or pruritus. In addition, mild infections, mild gastrointestinal disturbances, and fluctuations in blood pressure or heart rate have been observed.
Unanticipated Adverse Device Effect (UADE): Any serious adverse effect on health or safety, life-threatening event, or death caused by or associated with sirolimus, where the nature, severity, or incidence of such effect, event, or death was not previously identified in the investigational plan; or any other unanticipated serious problem related to sirolimus that concerns the rights, safety, or welfare of subjects.
Measured: 1, 3, 6, and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2026
- Study startJun 2026 (estimated)
- Primary completionJun 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vertebrobasilar DolichoectasiaStudies of the same condition, open and under way.
- More trials run by Huashan HospitalThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Huashan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Wei Zhu, Huashan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07634120
- Sponsor's protocol ID
- KY2026-724
- Status last verified
- Jun 2026
- First submitted
- 20 May 2026
- Last updated
- 8 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vertebrobasilar Dolichoectasia Treatment With Sirolimus: a Pilot Trial Based On 5.0 T MRI
- ClinicalTrials.gov ID
- NCT07634120
- Acronym / sponsor ID
- KY2026-724
- Conditions
- Vertebrobasilar Dolichoectasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Huashan HospitalUniversity, hospital or other
- Study start
- Jun 2026 (estimated)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 8 Jun 2026 (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.
The aim of this pilot trial is to assess the efficacy of sirolimus in reducing wall enhancement in vertebrobasilar dolichoectasia(VBD) on 5 T high-resolution magnetic resonance vessel wall imaging(HR-VWI) via anti-inflammatory mechanisms, clarify the efficacy of sirolimus in delaying the progression of VBD, evaluate the safety of sirolimus in the treatment of VBD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| sirolimus group | Experimental | Drug: SirolimusParticipants will receive oral sirolimus 2mg/d continuously for 6 months. |
| control group | No intervention | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age≥18 years, any gender;
- Patients with VBD confirmed by DSA/CTA/MRA;
- No history of VBD rupture and no surgical treatment for VBD;
- mRS<4;
- Positive plasma SGK1;
Key exclusion criteria (19 listed)
- History of malignant tumors;
- Pregnancy or lactation;
- Sirolimus allergy;
- Hydrocephalus requiring urgent surgical intervention or respiratory failure requiring life support treatment;
- Abnormal hepatic and/or renal function (serum transaminase > 40 U/L; serum creatinine > 110 μmol/L); and/or abnormal white blood cells/platelets (white blood cells count < 3.5 × 10⁹/L or > 9.5 × 10⁹/L; platelets count < 100 × 10⁹/L or > 300 × 10⁹/L);
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 |
|---|---|---|
| SirolimusSirolimus is an mTORC1/ mTORC2 inhibitor that has received approval from the U.S. Food and Drug Administration (FDA) and has recently been successfully used to treat lymphatic malformations and venous/lymphatic malformations associated with the same PIK3CA GOF mutations. Participants of sirolimus group will receive oral sirolimus 2mg/d continuously for 6 months. | Drug | sirolimus group |
Primary outcome
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
Time frame: 3 and 6 months
Using 5T HR-VWI, we quantify longitudinal changes of vascular CAWE (3D circumferential arterial wall enhancement: mean signal intensity in T1+Gd images) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Longitudinal changes of SAWE on 5T HR-VWI VBD following 3 and 6 months of sirolimus treatment.
Time frame: 3 and 6 months
Using 5T HR-VWI, we quantify longitudinal changes of vascular SAWE (specific contrast uptake arterial wall enhancement: the difference in mean signal intensity between T1 and T1+Gd) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Longitudinal changes of FAWE in VBD on 5T HR-VWI 3 and 6 months of sirolimus treatment.
Time frame: 3 and 6 months
Using 5T HR-VWI, we quantify longitudinal changes of vascular FAWE (focal arterial wall enhancement: areas of the diseased artery with increased AWE) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
Longitudinal changes of WEVR on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
Time frame: 3 and 6 months
Using 5T HR-VWI, we quantify longitudinal changes of vascular WEVR (3D arterial wall enhancement volume rate) at 3 and 6 months following initiation of sirolimus therapy. The primary objective of this study is to determine whether sirolimus exerts an anti-inflammatory effect on the diseased vertebrobasilar arterial wall in patients with VBD.
15 secondary outcomes
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
Time frame: 12 months
Longitudinal changes of SAWE on 5T HR-VWI VBD following 12 months of sirolimus treatment.
Time frame: 12 months
Longitudinal changes of FAWE on 5T HR-VWI VBD 12 months of sirolimus treatment.
Time frame: 12 months
Longitudinal changes of WEVR on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
Time frame: 12 months
Longitudinal changes of vascular dilation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Time frame: 3, 6, and 12 months
Longitudinal changes of vascular tortuosity on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Time frame: 3, 6, and 12 months
Longitudinal changes of vascular elongation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Time frame: 3, 6, and 12 months
Longitudinal changes of thrombus volume in VBD following 3, 6, and 12 months of sirolimus treatment.
Time frame: 3, 6, and 12 months
Longitudinal changes in plasma SGK1 levels following 1, 3, 6, and 12 months of sirolimus treatment in VBD patients.
Time frame: 1, 3, 6, and 12 months
Incidence of ischemic stroke in VBD patients at 3, 6, and 12 months.
Time frame: 3, 6, and 12 months
Incidence of subarachnoid hemorrhage associated with VBD rupture in VBD patients at 3, 6, and 12 months.
Time frame: 3, 6, and 12 months
modified Rankin Scale in VBD patients at 3, 6, and 12 months.
Time frame: 3, 6, and 12 months
Five-level EuroQol five-dimensional questionnaire in VBD patients at 3, 6, and 12 months.
Time frame: 3, 6, and 12 months
The safety of sirolimus in VDB patients.
Time frame: 1, 3, 6, and 12 months
The tolerability of sirolimus in VDB patients.
Time frame: 1, 3, 6, and 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 8 Jun 2026.
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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 Sirolimus in people with Vertebrobasilar Dolichoectasia, run by Huashan Hospital 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 8 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: opening soon, as of the sponsor's last update on 8 Jun 2026. 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 Liuxun Hu (+8615868058299, 19111220090@fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment. (time frame: 3 and 6 months). The study lists 4 primary outcomes in all. 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 8 Jun 2026.