Sirolimus for severe arteriovenous malformations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2 interventional study gives oral sirolimus to children (age ≥2), adolescents, and adults with superficial arteriovenous malformations to measure changes in lesion volume and symptoms. The rationale is that sirolimus inhibits mTOR and has anti-proliferative and anti-angiogenic effects that may slow or reverse abnormal vessel growth. Patients are screened for contraindications (including immunosuppression, active chronic infections, pregnancy, and relevant allergies) and must agree to contraception if of childbearing potential. Safety monitoring is included to track adverse effects and treatment response over the study period.

Why it matters

Who can join: the full rules

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

Where it's running

• CHU Amiens
  Amiens, France Recruiting
• CHU Dijon
  Dijon, France Recruiting
• HCL Lyon
  Lyon, France Recruiting
• Ucl
  Brussels, Belgium Not yet recruiting
• CHU Bordeaux
  Bordeaux, France Not yet recruiting
• CHRU Lille
  Lille, France Not yet recruiting

Study contacts

• Study coordinator: Bernard DEVAUCHELLE, MD, PhD
• Email: devauchelle.bernard@chu-amiens.fr
• Phone: +33322668325

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.