Sirolimus for Lymphangioleiomyomatosis

(MILED Trial)

This trial aims to determine if taking a low dose of sirolimus (also known as Rapamune) early and for an extended period can prevent the progression of lymphangioleiomyomatosis (LAM), a rare lung disease that primarily affects women and causes breathing difficulties. The study compares sirolimus to a placebo (a pill with no active drug) to evaluate effectiveness. Women diagnosed with LAM who experience symptoms that hinder daily life may be suitable candidates for this trial. As a Phase 3 trial, it represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

The trial requires that you stop using sirolimus, everolimus, investigational treatments for LAM, and estrogen-containing medications at least 30 days before joining. Other medications are not specifically mentioned, so check with the trial team.

The trial requires that you stop using estrogen-containing medications and sirolimus, everolimus, or any investigational treatment for LAM at least 30 days before joining. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Research has shown that sirolimus has been tested for safety in people with lymphangioleiomyomatosis (LAM). In these studies, sirolimus helped maintain stable lung function and improved symptoms. However, some participants experienced side effects; for instance, one study found that a few patients developed lung inflammation. Despite this, sirolimus is generally well-tolerated for LAM. Notably, the FDA has already approved sirolimus for other conditions, indicating its safety. Prospective trial participants should discuss potential side effects with the research team to understand what to expect.¹²³⁴⁵

Most treatments for lymphangioleiomyomatosis (LAM) primarily aim to manage symptoms and slow disease progression, often using medications like sirolimus or everolimus. However, what makes sirolimus particularly exciting in this study is its potential to specifically target and inhibit the mTOR pathway, which is crucial in LAM progression. This targeted action could mean more effective management of the disease with potentially fewer side effects compared to broader immunosuppressive treatments. Researchers are eager about this treatment because it harnesses a known mechanism with a targeted approach, possibly offering improved outcomes for patients with LAM.

Research has shown that sirolimus can help people with lymphangioleiomyomatosis (LAM) by slowing the disease's progression. In one study, 59% of patients responded positively after five years of treatment. Sirolimus stabilized lung function and improved quality of life. Patients who took sirolimus for one year also experienced stable lung capacity. Long-term use has generally maintained lung function and oxygen levels. In this trial, participants will receive either sirolimus or a placebo to evaluate its effectiveness further. Overall, sirolimus shows promise for managing LAM symptoms and slowing the disease's progression.¹⁶⁷⁸⁹