Идёт набор NCT02042326

Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations

Фаза II С лечением Arteriovenous Malformations

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Sirolimus.
Кому может быть актуально: Состояния в реестре: Arteriovenous Malformations. Базовые параметры: от 2 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Бельгия, Франция
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Обзор

The aim of the study is to evaluate the efficacy and safety of sirolimus (oral form), to decrease the volume and symptoms due to superficial arteriovenous malformations (AVM). Sirolimus has properties that reduce the activity of the immune system (immunosuppressant), to fight against the proliferation of cancer cells (anti- tumor) and also reduce the proliferation of blood vessels (anti -vascular). Sirolimus is primarily used in transplant patients to prevent organ transplant rejection. Many animal and laboratory studies were carried out and demonstrate in particular the activity of sirolimus on vessels. It is this anti- vascular effect that could help treat arteriovenous malformations.

Подробное описание

Anti-proliferative and anti-angiogenic properties of Sirolimus (Rapamycin®) are the basis of the rationale to use it in the treatment of arteriovenous malformations, for which the pathophysiology remains poorly understood. The interest of this class of drug is that inhibition of mTOR (mammalian target of rapamycin) may also block growth and / or angiogenic factors (other than VEGF) involved in the development of AVM. More specifically anti-VEGF drugs does not have that potential.

Вмешательства

Препарат Sirolimus
For patients with swallowing problems, and for children under 6 years and / or who have an inability to swallow tablets, the 1mg/ml solution form should be used.

Первичные конечные точки

Treatment efficacy at M12 [Срок оценки: After 12 months of treatment]

Вторичные конечные точки (5)

Treatment efficacy at M3 [Срок оценки: After 3 months of treatment]
Treatment efficacy at M6 [Срок оценки: After 6 months of treatment]
Treatment efficacy at M9 [Срок оценки: After 9 months of treatment]
Treatment tolerability [Срок оценки: One year]
Treatment Impact on Quality of life [Срок оценки: Before treatment initiation and after 12 months of treatment]

Критерии участия

Критерии включения

Patients (adults, adolescents and children older than 2 years), with arteriovenous malformation stage II + III or IV (according to Schöbinger's classification) : active or quiescent, marked or not by hemorrhagic phenomena.
Patients (parents for minors) must sign a consent form established after clear information risks and expected benefits of the study.
Patients (major and minor of childbearing age) must have effective contraception during the study period and continuing until 12 weeks after the end of treatment
Negative pregnancy blood test for women of childbearing age.

Критерии исключения

Chronic or acquired immunosuppression :

patients with transplanted organ or who received a hematopoietic stem cell
patient with congenital immunodeficiency
Patients implanted with chronic active infection associated with hepatitis B , hepatitis C or HIV
Pregnant or nursing woman.
Allergy to macrolides
Allergy to peanut or soya
Hypersensitivity to " Sirolimus " or any of the excipients of the investigational product
Contraindications to performing an MRI
Leukopenia below 1 000 /mm3
Thrombocytopenia lower to 80,000 /mm3
Anemia with Hb < 9 g/dl
Elevated transaminase > 2.5 N
History of cancer less than two years before the inclusion
Surgery older than 2 months before inclusion
Active infection (viral and bacterial ) on the date of inclusion
Hypercholesterolemia > 7 mmol / l despite appropriate medical treatment
Hyperlipidemia > 2 mmol / l despite appropriate medical treatment
Uncontrolled diabetes
Patients unable to follow a clinical study
Major under guardianship, persons deprived of their liberty