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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT02042326
Other study ID # PHRCN10-PR-DEVAUCHELLE
Secondary ID 2011-000321-69
Status Recruiting
Phase Phase 2
First received
Last updated
Start date September 12, 2014
Est. completion date September 2024

Study information

Verified date February 2023
Source Centre Hospitalier Universitaire, Amiens
Contact Bernard DEVAUCHELLE, MD, PhD
Phone +33322668325
Email devauchelle.bernard@chu-amiens.fr
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Description:

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 50
Est. completion date September 2024
Est. primary completion date September 2024
Accepts healthy volunteers No
Gender All
Age group 2 Years and older
Eligibility 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

Study Design


Intervention

Drug:
Sirolimus
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.

Locations

Country Name City State
Belgium UCL Bruxelles
France CHU Amiens Amiens
France CHU Bordeaux Bordeaux
France CHU Dijon Dijon
France CHRU Lille Lille
France HCL Lyon Lyon
France APHM Marseille
France CHU Montpellier Montpellier
France CHU Nancy Nancy
France CHU Nice Nice
France APHP Paris
France CHU Strasbourg Strasbourg
France CHU Tours Tours

Sponsors (1)

Lead Sponsor Collaborator
Centre Hospitalier Universitaire, Amiens

Countries where clinical trial is conducted

Belgium,  France, 

Outcome

Type Measure Description Time frame Safety issue
Primary Treatment efficacy at M12 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).
After 12 months of treatment
Secondary Treatment efficacy at M3 After 3 months of treatment
Secondary Treatment efficacy at M6 After 6 months of treatment
Secondary Treatment efficacy at M9 After 9 months of treatment
Secondary Treatment tolerability Number and description of serious advent events One year
Secondary Treatment Impact on Quality of life 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. Before treatment initiation and after 12 months of treatment
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