Skip to main content
OpenTrials
Recruiting

NCT Number: NCT02042326

Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Arteriovenous Malformations Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Vascular Diseases Vascular Malformations

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

UCL, Brussels, Belgium

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

sirolimus

Drug

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.

Other names: Rapamune

Primary outcomes

  1. Treatment efficacy at M12

    Time frame: After 12 months of treatment

    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).

Secondary outcomes

  1. Treatment efficacy at M3

    Time frame: After 3 months of treatment

  2. Treatment efficacy at M6

    Time frame: After 6 months of treatment

  3. Treatment efficacy at M9

    Time frame: After 9 months of treatment

  4. Treatment tolerability

    Time frame: One year

    Number and description of serious advent events

  5. Treatment Impact on Quality of life

    Time frame: Before treatment initiation and after 12 months of treatment

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Bernard DEVAUCHELLE, MD, PhD

CONTACT

[email protected]

+33322668325

Sylvie TESTELIN, MD, PhD

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Centre Hospitalier Universitaire, Amiens

Other

Registry information

Acronym: MAV-RAPA

Important dates

Study start
2014
Primary completion
2027
Study completion
2027
First posted
Jan 22, 2014
Registry last updated
May 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.