281. Klippel-Trenaunay-Weber syndrome Clinical trials / Disease details


Clinical trials : 2 Drugs : 2 - (DrugBank : 1) / Drug target gene : 1 - Drug target pathways : 51

  
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1JPRN-UMIN000030522
2017/11/1422/12/2017A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomaliesA multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies - Sirolimus for intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformationsBody surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Gifu UniversityNULLRecruitingNot applicableNot applicableMale and Female50Not selectedJapan
2NCT01364857
(ClinicalTrials.gov)
November 201031/5/2011French National Cohort of Children With Port Wine StainFrench National Prospective Cohort of Children With Port Wine Stain on a Limb = Cohorte Nationale d'Enfants Avec Angiome Plan de Membre inférieurPort Wine Stain;Klippel Trenaunay Syndrome;Parkes Weber SyndromeGenetic: search for polymorphisms of RASA1 geneUniversity Hospital, ToursNULLActive, not recruiting2 Years12 YearsAll150N/AFrance