Sirolimus for Superficial arteriovenous malformation

Inclusion criteria

• {"criterion_text":"-Patients (adults, adolescents and children over 2 years of age), of stage II+, III or IV of the Schöbinger classification (1994) whether their whether the lesion is progressive or quiescent, with or without haemorrhagic phenomena.\n-Patients (parents in the case of minors) must have signed a consent form consent form drawn up after clear information about the the expected risks and benefits of the study.\n-Patients should use effective contraception for the duration of the study for the duration of the study and for up to 12 weeks after the end of treatment.\n-Negative pregnancy test (blood β-HCG) for women of childbearing age."}

Exclusion criteria

• {"criterion_text":"-Acquired or chronic immunodepression\n-Anemia with Hb<9g/dl\n-Elevation of transaminases >2.5 N\n-History of cancer less than two years prior to inclusion\n-Surgery less than 2 months prior to inclusion\n-Active infection (viral and bacterial) at inclusion date\n-Hypercholesterolaemia > 7 mmol/l despite well-conducted medical treatment\n-Hyperlipidaemia > 2 mmol/l despite well-managed medical treatment\n-Uncontrolled diabetes\n-Patients unable to follow a clinical study\n-Adults under guardianship or trusteeship, persons deprived of their liberty\n-Patients with chronic active hepatitis B, hepatitis C or HIV infection\n-Pregnant or breast-feeding women.\n-Allergy to macrolides\n-Allergy to peanuts or soya\n-Hypersensitivity to 'Sirolimus' or to one of the excipients of the investigational product experimental product\n-Contraindication to the performance of an MRI scan\n-Leukopenia less than 1000 GB/mm3\n-Thrombocytopenia less than 80,000 PS/mm3"}

Primary endpoints

• {"endpoint_text":"-the proportion of patients with a reduction in tumour volume of the 30% reduction in AVM tumour volume by angioscannographic criteria during the first year of the study","definition_or_measurement_approach":"Proportion of patients achieving at least a 30% reduction in AVM tumour volume on angioscannographic evaluation comparing volume at one year versus baseline (angio-CT/angioscannographic criteria)."}

Secondary endpoints

• {"endpoint_text":"-Assessment of treatment efficacy at three months, six months, nine months","definition_or_measurement_approach":"Assessment of treatment efficacy at 3, 6 and 9 months (timed efficacy evaluations as stated)."}

France

Earliest CTIS Part Ii Submission Date

10-01-2025

Latest Decision Or Authorization Date

17-01-2025

Processing Time Days

7

Number Of Sites

4

Number Of Participants

50

Primary sponsor

Full Name

Centre Hospitalier Universitaire Amiens Picardie

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France