Actively Recruiting

Phase 2
Age: 2Years +
All Genders
NCT02042326

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

Led by Centre Hospitalier Universitaire, Amiens · Updated on 2026-05-13

50

Participants Needed

13

Research Sites

676 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 2Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients aged 2 years or older with arteriovenous malformations stage II, III, or IV (active or inactive, with or without bleeding)
  • Signed informed consent by patients or parents for minors
  • Effective contraception during the study and for 12 weeks after treatment for participants of childbearing potential
  • Negative pregnancy blood test for women of childbearing age
Not Eligible

You will not qualify if you...

  • Chronic or acquired immunosuppression, including organ transplant recipients, hematopoietic stem cell recipients, or congenital immunodeficiency
  • Chronic active infection with hepatitis B, hepatitis C, or HIV
  • Pregnant or nursing women
  • Allergy to macrolides, peanuts, or soy
  • Hypersensitivity to sirolimus or its excipients
  • Contraindications to MRI
  • Leukopenia below 1,000/mm3
  • Thrombocytopenia below 80,000/mm3
  • Anemia with hemoglobin less than 9 g/dl
  • Elevated liver transaminases greater than 2.5 times normal
  • History of cancer within 2 years before inclusion
  • Surgery within 2 months before inclusion
  • Active viral or bacterial infection at inclusion
  • Hypercholesterolemia over 7 mmol/l despite treatment
  • Hyperlipidemia over 2 mmol/l despite treatment
  • Uncontrolled diabetes
  • Inability to follow the clinical study
  • Persons under major guardianship or deprived of liberty

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 13 locations

1

UCL

Brussels, Belgium

Not Yet Recruiting

2

CHU Amiens

Amiens, France, 80000

Actively Recruiting

3

CHU Bordeaux

Bordeaux, France, 33000

Not Yet Recruiting

4

CHU Dijon

Dijon, France, 21000

Actively Recruiting

5

CHRU Lille

Lille, France, 59000

Not Yet Recruiting

6

HCL Lyon

Lyon, France, 69000

Actively Recruiting

7

APHM

Marseille, France, 13000

Not Yet Recruiting

8

CHU Montpellier

Montpellier, France, 34000

Not Yet Recruiting

9

CHU Nancy

Nancy, France, 54000

Not Yet Recruiting

10

CHU Nice

Nice, France, 06000

Not Yet Recruiting

11

APHP

Paris, France, 75000

Not Yet Recruiting

12

CHU Strasbourg

Strasbourg, France, 67000

Not Yet Recruiting

13

CHU Tours

Tours, France, 37000

Not Yet Recruiting

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Research Team

B

Bernard DEVAUCHELLE, MD, PhD

CONTACT

S

Sylvie TESTELIN, MD, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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