patients with congenital venous malformations (CVM)
Conditions
Interventions
Product Name: Ethanol gel
Product Code: L0122
Pharmaceutical Form: Gel for injection
CAS Number: 64-17-5
Other descriptive name: ETHANOL (96 PER CENT)
Concentration unit: % (W/W) percent weight/weight
Sponsors
ORFAGEN
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Patients of both sexes, of at least 12 years of age, -For women of childbearing potential, negative pregnancy test at baseline, -Patients with one clinically and radiologically (MRI) documented lesion diagnosed as CVM (pure or predominant), -Patients for which an embolosclerotherapy by the percutaneous route is indicated as first line therapy of the test lesion, or for which previous treatments (i.e. surgery, embolosclerotherapy, laser) have been unsuccessful or insufficient, -Patients with CVM lesional size of at least 12 cm3 (maximum craniocaudal dimension X mean dimension of 3 transverse equispaced measurements X mean dimension of 3 deepness equispaced measurements dimension) at MRI, -Patients with focal or multifocal CVM lesion, i.e. with one or several well-interconnecting venous spaces and well-defined margins, -Patients or parents able to follow study instructions and attend study visits, -Written informed consent from the patients or parents. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -Patients under 12 years of age, -Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception from more than 2 months, -Women of childbearing potential with a positive pregnancy test at baseline, -Patients with CVM of non venous predominance, -Patients with CVM that are not reachable by the percutaneous route, -Patients with extensive superficial skin CVM (i.e. with high risk of skin necrosis), -Patients with a test lesion adjacent to major nerves (e.g. facial nerve in the parotid region, intramuscular regions adjacent to major nerves), -Patients with facial CVM or bone involvement, -Patients with small CVM lesion (<12 cm3 at MRI), -Patients requiring more than 1 ml/Kg body weight (b.w.) in USA or more than 0.5 ml/Kg b.w. in france, or more than 30 mL of absolute ethanol to infuse, -Patients with a known allergy to one of the components of the test products, -Patients with a suspected allergy to iodinated products, -Patients with abnormal clotting parameters (platelets, partial thromboplastin, prothrombin time), -Patients with an active inflammatory episode of the test lesion (i.e. acute or subacute swelling of the test lesion), -Patients with complex malformations (e.g. Klippel-Trenaunay syndrome, Blue Rubber Bled Nevus syndrome, Muco-cutaneous familial venous malformations, Mafucci’s syndrome), -Patients in which a surgery, laser therapy or embolosclerotherapy of the test lesion has been performed within the last 12 weeks prior to study entry, -Asthmatic patients who require daily medications, -Patients with a non treated or non stabilized cardiac disease, -Patients with a suspected right-left shunt, -Patients with an intercurrent condition or a concomitant treatment which may interfere with a good conduct or the evaluation parameters of the study, -Patients who participated in a study within the 12 weeks prior to study entry, -Patients or parents who are not able or willing to follow the study instructions, -Patients or parents who refuse to give written informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the systemic exposure to ethanol with the two test products. ;Secondary Objective: - To compare the systemic (cardiopulmonary, hematological, metabolic) and local outcome of the two test products. - To compare the embolosclerosing activity of L0122 gel and absolute ethanol on size reduction of CVM. - To compare the clinical efficacy (patient benefit) of the two test products.;Primary end point(s): Pharmacokinetic parameter (primary study parameter): Cmax The parameter will be determined by plasma monitoring of ethanol just before infusion, then 5 mn, 10 mn, 20 mn, 40 mn, 60 mn, 90 mn, 120 mn after infusion at the first site, and every 60 mn onwards until ethanolemia becomes undetectable. | — |
Countries
France
Outcome results
None listed