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

• Register: ClinicalTrials.gov
• Last refreshed on: 20 February 2023
• Main ID: NCT02042326
• Date of registration: 20/01/2014
• Prospective Registration: Yes
• Primary sponsor: Centre Hospitalier Universitaire, Amiens
• Public title: Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations MAV-RAPA
• Scientific title: Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
• Date of first enrolment: September 12, 2014
• Target sample size: 50
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02042326
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium, France

Contacts

• Name: Bernard DEVAUCHELLE, MD, PhD

• Name: Emmanuel MORELON, MD, PhD
• Affiliation: HCL Lyon

• Name: Bernard DEVAUCHELLE, MD, PhD
• Affiliation: CHU Amiens

Key inclusion & exclusion criteria

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



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Arteriovenous Malformations

Intervention(s)

Drug: Sirolimus

Primary Outcome(s)

Treatment efficacy at M12 [Time Frame: After 12 months of treatment]

Secondary Outcome(s)

Treatment tolerability [Time Frame: One year]

Treatment Impact on Quality of life [Time Frame: Before treatment initiation and after 12 months of treatment]

Treatment efficacy at M9 [Time Frame: After 9 months of treatment]

Treatment efficacy at M6 [Time Frame: After 6 months of treatment]

Treatment efficacy at M3 [Time Frame: After 3 months of treatment]

Secondary ID(s)

2011-000321-69

PHRCN10-PR-DEVAUCHELLE

Source(s) of Monetary Support

Please refer to primary and secondary sponsors