Vertebrobasilar Dolichoectasia Treatment With Sirolimus
3 июня 2026 г. обновлено: 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.
Обзор исследования
Статус
Еще не набирают
Условия
Вмешательство/лечение
Тип исследования
Интервенционный
Регистрация (Оцененный)
12
Фаза
- Фаза 2
Контакты и местонахождение
В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.
Контакты исследования
- Имя: Liuxun Hu
- Номер телефона: +8615868058299
- Электронная почта: 19111220090@fudan.edu.cn
Учебное резервное копирование контактов
- Имя: Wei Zhu
- Номер телефона: +8615868058299
- Электронная почта: drzhuwei@fudan.edu.cn
Места учебы
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Shanghai Municipality
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Shanghai, Shanghai Municipality, Китай, 200040
- Huashan Hospital, Fudan University
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Контакт:
- Wei Zhu
- Номер телефона: +8615868058299
- Электронная почта: 19111220090@fudan.edu.cn
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-
Критерии участия
Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.
Критерии приемлемости
Возраст, подходящий для обучения
- Взрослый
- Пожилой взрослый
Принимает здоровых добровольцев
Нет
Описание
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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;
- 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.
Учебный план
В этом разделе представлена подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.
Как устроено исследование?
Детали дизайна
- Основная цель: Уход
- Распределение: Рандомизированный
- Интервенционная модель: Параллельное назначение
- Маскировка: Нет (открытая этикетка)
Оружие и интервенции
Группа участников / Армия |
Вмешательство/лечение |
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Без вмешательства: контрольная группа
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Экспериментальный: sirolimus group
Participants will receive oral sirolimus 2mg/d continuously for 6 months.
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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.
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Что измеряет исследование?
Первичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
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Longitudinal changes of CAWE on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
Временное ограничение: 3 and 6 months
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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.
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3 and 6 months
|
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Longitudinal changes of SAWE on 5T HR-VWI VBD following 3 and 6 months of sirolimus treatment.
Временное ограничение: 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
|
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Longitudinal changes of FAWE in VBD on 5T HR-VWI 3 and 6 months of sirolimus treatment.
Временное ограничение: 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.
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3 and 6 months
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Longitudinal changes of WEVR on 5T HR-VWI in VBD following 3 and 6 months of sirolimus treatment.
Временное ограничение: 3 and 6 months
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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.
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3 and 6 months
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Вторичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
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Longitudinal changes of CAWE on 5T HR-VWI in VBD following 12 months of sirolimus treatment.
Временное ограничение: 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.
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12 months
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Longitudinal changes of SAWE on 5T HR-VWI VBD following 12 months of sirolimus treatment.
Временное ограничение: 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.
Временное ограничение: 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.
Временное ограничение: 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.
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12 months
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Longitudinal changes of vascular dilation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Временное ограничение: 3, 6, and 12 months
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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.
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3, 6, and 12 months
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Longitudinal changes of vascular tortuosity on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Временное ограничение: 3, 6, and 12 months
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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.
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3, 6, and 12 months
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Longitudinal changes of vascular elongation on 5T MRA in VBD following 3, 6, and 12 months of sirolimus treatment.
Временное ограничение: 3, 6, and 12 months
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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.
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3, 6, and 12 months
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Longitudinal changes of thrombus volume in VBD following 3, 6, and 12 months of sirolimus treatment.
Временное ограничение: 3, 6, and 12 months
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Using 5T HR-VWI, we quantify longitudinal changes of thrombus volume in VBD at 3, 6, and 12 months following sirolimus treatment.
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3, 6, and 12 months
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Longitudinal changes in plasma SGK1 levels following 1, 3, 6, and 12 months of sirolimus treatment in VBD patients.
Временное ограничение: 1, 3, 6, and 12 months
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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.
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1, 3, 6, and 12 months
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Incidence of ischemic stroke in VBD patients at 3, 6, and 12 months.
Временное ограничение: 3, 6, and 12 months
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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.
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3, 6, and 12 months
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Incidence of subarachnoid hemorrhage associated with VBD rupture in VBD patients at 3, 6, and 12 months.
Временное ограничение: 3, 6, and 12 months
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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.
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3, 6, and 12 months
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modified Rankin Scale in VBD patients at 3, 6, and 12 months.
Временное ограничение: 3, 6, and 12 months
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modified Rankin Scale (mRS), a 7-level, clinician-reported, measure of global disability is measured in VBD patients at 3, 6, and 12 months.
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3, 6, and 12 months
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Five-level EuroQol five-dimensional questionnaire in VBD patients at 3, 6, and 12 months.
Временное ограничение: 3, 6, and 12 months
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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.
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3, 6, and 12 months
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The safety of sirolimus in VDB patients.
Временное ограничение: 1, 3, 6, and 12 months
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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.
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1, 3, 6, and 12 months
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The tolerability of sirolimus in VDB patients.
Временное ограничение: 1, 3, 6, and 12 months
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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
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Соавторы и исследователи
Здесь вы найдете людей и организации, участвующие в этом исследовании.
Спонсор
Даты записи исследования
Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.
Изучение основных дат
Начало исследования (Оцененный)
1 июня 2026 г.
Первичное завершение (Оцененный)
1 июня 2027 г.
Завершение исследования (Оцененный)
1 декабря 2027 г.
Даты регистрации исследования
Первый отправленный
20 мая 2026 г.
Впервые представлено, что соответствует критериям контроля качества
3 июня 2026 г.
Первый опубликованный (Действительный)
8 июня 2026 г.
Обновления учебных записей
Последнее опубликованное обновление (Действительный)
8 июня 2026 г.
Последнее отправленное обновление, отвечающее критериям контроля качества
3 июня 2026 г.
Последняя проверка
1 июня 2026 г.
Дополнительная информация
Термины, связанные с этим исследованием
Дополнительные соответствующие термины MeSH
- Цереброваскулярные расстройства
- Заболевания головного мозга
- Заболевания центральной нервной системы
- Заболевания нервной системы
- Сосудистые заболевания
- Сердечно-сосудистые заболевания
- Ишемия головного мозга
- Вертебробазилярная недостаточность
- Органические химические вещества
- Макролиды
- Лактоны
- Сиролимус
Другие идентификационные номера исследования
- KY2026-724
Информация о лекарствах и устройствах, исследовательские документы
Изучает лекарственный продукт, регулируемый FDA США.
Нет
Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.
Нет
Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .