Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
Centre Hospitalier Universitaire, Amiens
50 participants
Sep 12, 2014
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria4
- 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.
Exclusion Criteria21
- 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
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
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.
Locations(13)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT02042326