NCT02042326 Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
| NCT ID | NCT02042326 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Centre Hospitalier Universitaire, Amiens |
| Condition | Arteriovenous Malformations |
| Study Type | INTERVENTIONAL |
| Enrollment | 50 participants |
| Start Date | 2014-09-12 |
| Primary Completion | 2027-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 50 participants in total. It began in 2014-09-12 with a primary completion date of 2027-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility 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
Contact & Investigator
Bernard DEVAUCHELLE, MD, PhD
STUDY DIRECTOR
CHU Amiens
Frequently Asked Questions
Who can join the NCT02042326 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Arteriovenous Malformations. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT02042326 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT02042326 currently recruiting?
Yes, NCT02042326 is actively recruiting participants. Contact the research team at devauchelle.bernard@chu-amiens.fr for enrollment information.
Where is the NCT02042326 trial being conducted?
This trial is being conducted at Brussels, Belgium, Amiens, France, Bordeaux, France, Dijon, France and 9 additional locations.
Who is sponsoring the NCT02042326 clinical trial?
NCT02042326 is sponsored by Centre Hospitalier Universitaire, Amiens. The principal investigator is Bernard DEVAUCHELLE, MD, PhD at CHU Amiens. The trial plans to enroll 50 participants.