What this Trial Is About

Lymphangioleiomyomatosis (LAM) is a rare lung disease affecting women, characterized by cystic lung damage and worsening respiratory failure. Researchers are studying the long-term safety and effectiveness of mTOR inhibitors, such as sirolimus and everolimus, which have shown to stabilize lung function decline during earlier studies. This study, called the Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS), aims to follow about 600 patients with LAM, including adult female patients with tuberous sclerosis complex (TSC) with or without lung cysts, who are taking or considering mTOR inhibitor treatment as part of their clinical care. Participants will receive sirolimus or everolimus under the guidance of their own doctors, with treatments managed outside the study. The study is observational and will track lung function tests and any side effects over a period of at least two years. It includes patients who are currently on chronic therapy, newly treated, previously intolerant to, or having failed mTOR inhibitor therapy. The decision to start or continue treatment lies with the patient and their physician. During the study, participants will attend regular clinical visits at least once per year, where lung function testing and safety assessments will be performed as part of routine care. Researchers will monitor lung function decline, focusing on measures such as forced expiratory volume (FEV1), and record any adverse events related to treatment. This long-term observation will help improve understanding of the durability and safety of mTOR inhibitors for managing LAM and may guide future treatment approaches.

Safety and Durability of Sirolimus for Treatment of LAM