View clinical trials related to Lymphatic Abnormalities.
Filter by:The main purpose of this study is to assess the change in clinician global impression after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel compared to placebo in approximately 50 participants with microcystic lymphatic malformations.
Recent studies have demonstrated that growth of vascular malformations can be driven by genetic variants in one of 2 signalling pathways. Targeted drugs specific to these pathways have been developed and shown to be effective in treating cancer. This study will describe the effectiveness of (i) 48 weeks of alpelisib therapy for participants with slow-flow vascular malformations and a gene mutation in one of these signalling pathways (module 1) and (ii) 48 weeks of mirdametinib therapy for participants with fast-flow vascular malformations and a gene mutations in the other signalling pathway (module 2).