A Phase 2 interventional study of Sirolimus in Arteriovenous Malformations, sponsored by Centre Hospitalier Universitaire, Amiens. Recruiting at 13 sites in 2 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-05-13.
Sponsored by Centre Hospitalier Universitaire, Amiens · Phase 2, Interventional, and Treatment
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.
120 studies on the registry are indexed under Arteriovenous Malformations; 32 are open to participants now.
This study's planned enrollment of 50 is below the median of 59 across 59 interventional studies indexed under Arteriovenous Malformations.
Browse Arteriovenous Malformations studies →Centre Hospitalier Universitaire, Amiens is the lead sponsor of 576 studies on the registry; 178 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Chronic or acquired immunosuppression :
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.
Drug: 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.
Also known as: Rapamune
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
Plan to share: No
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Centre Hospitalier Universitaire, Amiens