Actively Recruiting
Prospective Evaluation of the Efficacy of Sirolimus (Rapamune ae) in the Treatment of Severe Arteriovenous Malformations
Led by Centre Hospitalier Universitaire, Amiens · Updated on 2026-05-13
50
Participants Needed
13
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy and safety of oral sirolimus in reducing the volume and symptoms of superficial arteriovenous malformations (AVM) in patients aged 2 years and older. This phase 2 study explores sirolimus, which has immunosuppressant, anti-tumor, and anti-vascular properties. The drug's potential to inhibit blood vessel growth may help treat AVMs, which currently have poorly understood mechanisms. This research is sponsored by Centre Hospitalier Universitaire, Amiens. Participants will receive sirolimus orally, starting at 2 mg per day with doses adjusted weekly over four weeks to reach and maintain a blood level of 8 to 12 ng/ml. If side effects occur but do not require stopping treatment, the dose may be lowered while keeping a minimum blood level of 3 ng/ml. Tablets are preferred, but a liquid form is available for children under 6 years or those with swallowing difficulties. To reduce early side effects, patients will take a corticosteroid, prednisolone, at 0.5 mg/kg daily for the first week. During the 12-month treatment, participants will be monitored for treatment effectiveness and tolerability, with evaluations at 3, 6, 9, and 12 months. Quality of life assessments will be done before treatment and after one year. Researchers will observe changes in AVM volume and symptoms while tracking side effects. The total study duration for each participant is one year, with regular clinical assessments and blood tests to ensure safety and dose management.
CONDITIONS
Brief Title
Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 2 years or older with arteriovenous malformation stage II, III, or IV (active or quiescent, with or without hemorrhagic events)
- Consent form signed by patient or parent/guardian for minors after clear information
- Effective contraception required for patients of childbearing age during the study and 12 weeks after treatment ends
- Negative pregnancy blood test for women of childbearing age
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, peanut, or soya
- Hypersensitivity to sirolimus or any ingredient of the study drug
- 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 less than two years before enrollment
- Surgery within two months before enrollment
- Active viral or bacterial infection at enrollment
- High cholesterol or lipid levels despite treatment
- Uncontrolled diabetes
- Inability to follow the clinical study
- Individuals under guardianship or deprived of liberty
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 months
Participants receive sirolimus medication with dose adjustments during the first month to maintain target blood levels. Corticosteroids are given during the first week to prevent side effects.
Weekly visits during the first month, then regular visits for treatment monitoring
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
Research Team
B
Bernard DEVAUCHELLE, MD, PhD
S
Sylvie TESTELIN, MD, PhD
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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