Arteriovenous MalformationsPhase 2MAV-RAPANCT02042326
Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
A Phase 2 study testing Sirolimus in people with Arteriovenous Malformations, run by Centre Hospitalier Universitaire, Amiens at 13 sites in 2 countries.
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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Arteriovenous Malformations
- What is being tested?
- Sirolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 13 sitesin 2 countries
- Study dates
- 12 Sep 2014to Sep 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 21Show fewer
- 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
Trial locations and contacts
13 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
Sylvie TESTELIN, MD, PhD contact
13 sites in 2 countries
France12 sites, 12 recruiting
- Recruiting
CHU Amiens
Amiens, 80000
Bernard DEVAUCHELLE, MD, PhD
Sylvie TESTELIN, MD, PhD
- Not yet recruiting
CHU Bordeaux
Bordeaux, 33000
- Recruiting
CHU Dijon
Dijon, 21000
Pierre VABRES, MD PhD
Pierre VABRES, MD PhD
- Not yet recruiting
CHRU Lille
Lille, 59000
- Recruiting
HCL Lyon
Lyon, 69000
Pierre BRETON, MD PhD, pierre.breton@chu-lyon.fr
Laurent GUIBAUD, MD PhD, laurent.guibaud@chu-lyon.fr
- Not yet recruiting
APHM
Marseille, 13000
Stéphanie MALLET, MD, stephanie.mallet@ap-hm.fr
Jean-Michel BARTOLI
- Not yet recruiting
CHU Montpellier
Montpellier, 34000
- Not yet recruiting
CHU Nancy
Nancy, 54000
- Not yet recruiting
CHU Nice
Nice, 06000
- Not yet recruiting
APHP
Paris, 75000
- Not yet recruiting
CHU Strasbourg
Strasbourg, 67000
- Not yet recruiting
CHU Tours
Tours, 37000
About this study
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.
Read the detailed 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.
- Conditions
- Arteriovenous Malformations
- Being tested
- Sirolimus
- Keywords
- Arteriovenous Malformations, Sirolimus, Maxillofacial Surgery
- Medical subject headings
- Arteriovenous Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune
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.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus treatment | Experimental | Patients will receive sirolimus (Rapamune). The dose should be adjusted to obtain a residual plasma rate of 8 to 12 ng/ml in 4 weeks. This serum level will be maintained throughout the duration of the study in the absence of side effects. In case of intolerance that do not justify the discontinuation of treatment, the dose may be reduced by maintaining a serum level greater than 3 ng/ml.
The starting dose will be 2 mg per day, and will be adapted every week for one month.
The preferred dosage form is tablet form. To prevent common side effects in early treatment, corticosteroids based prednisolone (SOLUPRED) will be established at a dose of 0.5 mg/ kg/day for the first week of treatment. Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: After 12 months of treatment
Secondary outcomes (5)
Treatment efficacy at M3
Measured: After 3 months of treatment
Treatment efficacy at M6
Measured: After 6 months of treatment
Treatment efficacy at M9
Measured: After 9 months of treatment
Treatment tolerability
Number and description of serious advent events
Measured: One year
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.
Measured: Before treatment initiation and after 12 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2014 (estimated)
- Study start12 Sep 2014
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Arteriovenous MalformationsStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire, AmiensThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire, Amiens
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Bernard DEVAUCHELLE, MD, PhD, Study director, CHU AmiensEmmanuel MORELON, MD, PhD, Study chair, HCL Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02042326
- Sponsor's protocol ID
- PHRCN10-PR-DEVAUCHELLE
- Other IDs
- 2011-000321-69
- Acronym
- MAV-RAPA
- Status last verified
- May 2026
- First submitted
- 20 Jan 2014
- Last updated
- 13 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
- ClinicalTrials.gov ID
- NCT02042326
- Acronym / sponsor ID
- MAV-RAPA / PHRCN10-PR-DEVAUCHELLE
- Conditions
- Arteriovenous Malformations
- Keywords
- Arteriovenous Malformations, Sirolimus, Maxillofacial Surgery
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire, AmiensUniversity, hospital or other
- Study start
- 12 Sep 2014 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 22 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus treatment | Experimental | Drug: SirolimusPatients will receive sirolimus (Rapamune). The dose should be adjusted to obtain a residual plasma rate of 8 to 12 ng/ml in 4 weeks. This serum level will be maintained throughout the duration of the study in the absence of side effects. In case of intolerance that do not justify the discontinuation of treatment, the dose may be reduced by maintaining a serum level greater than 3 ng/ml. The starting dose will be 2 mg per day, and will be adapted every week for one month. The preferred dosage form is tablet form. To prevent common side effects in early treatment, corticosteroids based prednisolone (SOLUPRED) will be established at a dose of 0.5 mg/ kg/day for the first week of treatment. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (21 listed)
- Chronic or acquired immunosuppression :
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: RapamuneFor 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. | Drug | Sirolimus treatment |
Primary outcome
Treatment efficacy at M12
Time frame: After 12 months of treatment
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).
5 secondary outcomes
Treatment efficacy at M3
Time frame: After 3 months of treatment
Treatment efficacy at M6
Time frame: After 6 months of treatment
Treatment efficacy at M9
Time frame: After 9 months of treatment
Treatment tolerability
Time frame: One year
Treatment Impact on Quality of life
Time frame: Before treatment initiation and after 12 months of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sylvie TESTELIN, MD, PhD contact
Bernard DEVAUCHELLE, MD, PhD study director
CHU Amiens
Emmanuel MORELON, MD, PhD study chair
HCL Lyon
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Arteriovenous Malformations, run by Centre Hospitalier Universitaire, Amiens at 13 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in France, Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Bernard DEVAUCHELLE, MD, PhD (+33322668325, devauchelle.bernard@chu-amiens.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment efficacy at M12 (time frame: After 12 months of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2026.