Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations (MAV-RAPA)
Arteriovenous Malformations
About this trial
This is an interventional treatment trial for Arteriovenous Malformations focused on measuring Arteriovenous Malformations, Sirolimus, Maxillofacial Surgery
Eligibility Criteria
Inclusion Criteria:
- 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 Criteria:
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
Sites / Locations
- UCL
- CHU AmiensRecruiting
- CHU Bordeaux
- CHU DijonRecruiting
- CHRU Lille
- HCL LyonRecruiting
- APHM
- CHU Montpellier
- CHU Nancy
- CHU Nice
- APHP
- CHU Strasbourg
- CHU Tours
Arms of the Study
Arm 1
Experimental
Sirolimus treatment
Patients will receive sirolimus (Rapamune). The dose should be adjusted to obtain a residual plasma rate of 8 to 12 ng/ml in 4 weeks. This serum level will be maintained throughout the duration of the study in the absence of side effects. In case of intolerance that do not justify the discontinuation of treatment, the dose may be reduced by maintaining a serum level greater than 3 ng/ml. The starting dose will be 2 mg per day, and will be adapted every week for one month. The preferred dosage form is tablet form. To prevent common side effects in early treatment, corticosteroids based prednisolone (SOLUPRED) will be established at a dose of 0.5 mg/ kg/day for the first week of treatment.