Study of Oral Rapamycin to Treat Cervicofacial Lymphatic Malformations in Children and Teens up to 18 Years

What this Trial Is About

Researchers are evaluating the use of Rapamycin to treat extended cervicofacial lymphatic malformations in children and adolescents up to 18 years old. This Phase 2 study aims to measure how well Rapamycin reduces the volume of these malformations, improves quality of life, and decreases bleeding when mucosal areas are involved. The treatment outcomes are assessed by MRI scans and clinical evaluations over time. Participants receive Rapamycin orally for a period of 6 months. The study monitors response rates at 3 months and continues to observe clinical symptoms and quality of life impacts up to 12 months. Biological tests measure Rapamycin levels and side effects are tracked monthly throughout one year. MRI imaging is used to measure the malformation volume and follow treatment effectiveness. During the study, participants undergo regular clinical assessments and complete Pediatric Quality of Life Inventory PedsQL 4 scales at baseline, 3, 6, and 12 months. Blood tests evaluate biological responses and safety. Researchers monitor side effects monthly for one year to ensure participant safety. The total follow-up lasts at least one year, allowing detailed tracking of treatment effects and participant well-being.

Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations