NCT07634120

Brief 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 the safety of sirolimus in the treatment of VBD.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_2

Timeline
18mo left

Started Jun 2026

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress3%
Jun 2026Dec 2027

First Submitted

Initial submission to the registry

May 20, 2026

Completed
12 days until next milestone

Study Start

First participant enrolled

June 1, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

June 8, 2026

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2027

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Last Updated

June 8, 2026

Status Verified

June 1, 2026

Enrollment Period

1 year

First QC Date

May 20, 2026

Last Update Submit

June 3, 2026

Conditions

Vertebrobasilar Dolichoectasia

Outcome Measures

Primary Outcomes (4)

  • 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.

    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.

    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.

    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.

    3 and 6 months

Secondary Outcomes (15)

  • Longitudinal changes of CAWE on 5T HR-VWI in VBD following 12 months of sirolimus treatment.

    12 months

  • Longitudinal changes of SAWE on 5T HR-VWI VBD following 12 months of sirolimus treatment.

    12 months

  • Longitudinal changes of FAWE on 5T HR-VWI VBD 12 months of sirolimus treatment.

    12 months

  • Longitudinal changes of WEVR on 5T HR-VWI in VBD following 12 months of sirolimus treatment.

    12 months

  • Longitudinal changes of vascular dilation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.

    3, 6, and 12 months

  • +10 more secondary outcomes

Study Arms (2)

sirolimus group

EXPERIMENTAL

Participants will receive oral sirolimus 2mg/d continuously for 6 months.

Drug: Sirolimus

control group

NO INTERVENTION

Interventions

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.

sirolimus group

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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;
  • 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;
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Huashan Hospital, Fudan University

Shanghai, Shanghai Municipality, 200040, China

Location

MeSH Terms

Conditions

Vertebrobasilar Insufficiency

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Brain IschemiaCerebrovascular DisordersBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesVascular DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Central Study Contacts

Liuxun Hu

CONTACT

+861586805829919111220090@fudan.edu.cn

Wei Zhu

CONTACT

+8615868058299drzhuwei@fudan.edu.cn

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

May 20, 2026

First Posted

June 8, 2026

Study Start

June 1, 2026

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

December 1, 2027

Last Updated

June 8, 2026

Record last verified: 2026-06

Locations

CN(1)