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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
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.
UCL
Brussels, Belgium
NOT_YET_RECRUITINGCHU Amiens
Amiens, France
RECRUITINGCHU Bordeaux
Bordeaux, France
NOT_YET_RECRUITINGCHU Dijon
Dijon, France
RECRUITINGCHRU Lille
Lille, France
NOT_YET_RECRUITINGHCL Lyon
Lyon, France
RECRUITINGAPHM
Marseille, France
NOT_YET_RECRUITINGCHU Montpellier
Montpellier, France
NOT_YET_RECRUITINGCHU Nancy
Nancy, France
NOT_YET_RECRUITINGCHU Nice
Nice, France
NOT_YET_RECRUITING...and 3 more locations
Treatment efficacy at M12
The efficacy of treatment is a composite criteria based on: * The proportion of patients with no evolution of the AVM during the study period, * The proportion of patients with a reduction in tumor volume of the AVM at least 30% of CT Angiography (CTA) criteria during the first year of the study (comparison of the volume of the AVM a year versus pre-inclusion).
Time frame: After 12 months of treatment
Treatment efficacy at M3
Time frame: After 3 months of treatment
Treatment efficacy at M6
Time frame: After 6 months of treatment
Treatment efficacy at M9
Time frame: After 9 months of treatment
Treatment tolerability
Number and description of serious advent events
Time frame: One year
Treatment Impact on Quality of life
Quality of life will be assessed before and at the end of the first year of treatment using a questionnaire given to patients. There is no questionnaire specifically tailored to vascular malformations in the literature. Thus the investigators adapted a document based on an evaluation of the quality of life for survivors of burn injury.
Time frame: Before treatment initiation and after 12 months of treatment
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.