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Vertebrobasilar Dolichoectasia Treatment With Sirolimus

2026년 6월 3일 업데이트: Wei Zhu, Huashan Hospital

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.

연구 개요

상태

아직 모집하지 않음

정황

개입 / 치료

연구 유형

중재적

등록 (추정된)

12

단계

  • 2 단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

연구 장소

  • 중국
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, 중국, 200040
        • Huashan Hospital, Fudan University
        • 연락하다:

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

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.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
간섭 없음: 대조군
실험적: sirolimus group
Participants will receive oral sirolimus 2mg/d continuously for 6 months.
의약품: Sirolimus
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.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
기간: 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.
3 and 6 months
Longitudinal changes of SAWE on 5T HR-VWI VBD following 3 and 6 months of sirolimus treatment.
기간: 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.
3 and 6 months
Longitudinal changes of FAWE in VBD on 5T HR-VWI 3 and 6 months of sirolimus treatment.
기간: 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.
3 and 6 months
Longitudinal changes of WEVR on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
기간: 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.
3 and 6 months

2차 결과 측정

결과 측정
측정값 설명
기간
Longitudinal changes of CAWE on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
기간: 12 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 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.
12 months
Longitudinal changes of SAWE on 5T HR-VWI VBD following 12 months of sirolimus treatment.
기간: 12 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 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.
12 months
Longitudinal changes of FAWE on 5T HR-VWI VBD 12 months of sirolimus treatment.
기간: 12 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 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.
12 months
Longitudinal changes of WEVR on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
기간: 12 months
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.
12 months
Longitudinal changes of vascular dilation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
기간: 3, 6, and 12 months
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.
3, 6, and 12 months
Longitudinal changes of vascular tortuosity on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
기간: 3, 6, and 12 months
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.
3, 6, and 12 months
Longitudinal changes of vascular elongation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
기간: 3, 6, and 12 months
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.
3, 6, and 12 months
Longitudinal changes of thrombus volume in VBD following 3, 6, and 12 months of sirolimus treatment.
기간: 3, 6, and 12 months
Using 5T HR-VWI, we quantify longitudinal changes of thrombus volume in VBD at 3, 6, and 12 months following sirolimus treatment.
3, 6, and 12 months
Longitudinal changes in plasma SGK1 levels following 1, 3, 6, and 12 months of sirolimus treatment in VBD patients.
기간: 1, 3, 6, and 12 months
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.
1, 3, 6, and 12 months
Incidence of ischemic stroke in VBD patients at 3, 6, and 12 months.
기간: 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.
3, 6, and 12 months
Incidence of subarachnoid hemorrhage associated with VBD rupture in VBD patients at 3, 6, and 12 months.
기간: 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.
3, 6, and 12 months
modified Rankin Scale in VBD patients at 3, 6, and 12 months.
기간: 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.
3, 6, and 12 months
Five-level EuroQol five-dimensional questionnaire in VBD patients at 3, 6, and 12 months.
기간: 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.
3, 6, and 12 months
The safety of sirolimus in VDB patients.
기간: 1, 3, 6, and 12 months
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.
1, 3, 6, and 12 months
The tolerability of sirolimus in VDB patients.
기간: 1, 3, 6, and 12 months

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.
1, 3, 6, and 12 months

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Huashan Hospital

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2026년 6월 1일

기본 완료 (추정된)

2027년 6월 1일

연구 완료 (추정된)

2027년 12월 1일

연구 등록 날짜

최초 제출

2026년 5월 20일

QC 기준을 충족하는 최초 제출

2026년 6월 3일

처음 게시됨 (실제)

2026년 6월 8일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 6월 8일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 6월 3일

마지막으로 확인됨

2026년 6월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

  • KY2026-724

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

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미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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