Long-term Safety and Tolerability Study of Imatinib for Women with Lymphangioleiomyomatosis A Phase 1 Randomized, Double-blind, Placebo-controlled Trial

What this Trial Is About

Lymphangioleiomyomatosis LAM is a rare lung disease that behaves like a slow-growing cancer caused by unregulated activity of the mTOR pathway. Researchers are studying the long-term safety and tolerability of imatinib mesylate, a drug approved for leukemia that may induce death of LAM cells. This Phase 1 trial follows a previous short-term pilot study and aims to provide more information on imatinibs effects in patients with LAM. Participants will be randomly assigned to receive either imatinib mesylate or a placebo over a six-month period. Imatinib is taken orally at a dose of 400 mg twice daily. The trial includes five office visits and check-up phone calls every two weeks to monitor participants. The study is designed as a double-blinded, placebo-controlled trial to compare safety and tolerability between the two groups. During the study, participants will undergo assessments including lung function tests such as Forced Vital Capacity FVC and Forced Expiratory Volume in One Second FEV1, as well as measurement of the biomarker VEGF-D. Quality of life will be evaluated using the St. George Respiratory Questionnaire. The main outcome measured is the incidence of adverse events over one year. Participants will be monitored regularly for safety and adherence throughout the trial duration.

Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)