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Could a common drug tame a rare brain artery disorder?

NCT ID NCT07634120

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This pilot trial tests whether sirolimus, an anti-inflammatory drug, can reduce artery wall swelling in people with vertebrobasilar dolichoectasia (VBD), a condition where arteries at the base of the brain become enlarged and twisted. Twelve participants will take sirolimus daily for 6 months, and doctors will use advanced 5T MRI scans to track changes. The goal is to see if the drug can calm inflammation and possibly slow disease progression.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Sirolimus (an oral drug that calms inflammation)
What this could lead to
If it works, this could point toward a treatment to slow or stop the progression of vertebrobasilar dolichoectasia, a rare artery condition.
What could go wrong
This is a very small pilot study with only 12 people, so results may not apply widely. It is also early-stage and may not show clear benefit.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Age≥18 years, any gender; 2. Patients with VBD confirmed by DSA/CTA/MRA; 3. No history of VBD rupture and no surgical treatment for VBD; 4. mRS\<4; 5. Positive plasma SGK1; 6. History of posterior circulation infarction or accompanied by VBD-related symptoms/signs; 7. 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; 8. Capable of signing an informed consent form with the accompaniment and understanding of a guardian. Exclusion Criteria: 1. History of malignant tumors; 2. Pregnancy or lactation; 3. Sirolimus allergy; 4. Hydrocephalus requiring urgent surgical intervention or respiratory failure requiring life support treatment; 5. 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); 6. History of immunosuppressive therapy; 7. Acute cerebral infarction within the last month or definite high signal on DWI indicating acute or subacute cerebral infarction; 8. Acute stage of intracranial hemorrhage as indicated by CT; 9. History of VBD rupture or surgery; 10. Presence of acute active infection (such as severe bacterial, viral or fungal infection); 11. Uncontrolled diabetes (HbA1c≥7%); 12. History of liver or lung transplantation; 13. Presence of organic heart disease; 14. History of arteriovenous thrombosis; 15. Patients taking only antiplatelet drugs or only statin drugs; 16. 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.); 17. Currently participating in other clinical studies; 18. Presence of contraindications for MRI examination; 19. Other situations not suitable for inclusion.

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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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Huashan Hospital, Fudan University

    Shanghai, Shanghai Municipality, 200040, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.