Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)
University of Cincinnati
Summary
The MIDAS study aims to follow male and female LAM patients who are currently taking, have previously failed or been intolerant of, or may (at some time in the future) take mTOR inhibitors (sirolimus or everolimus) as part of their clinical care. Adult female TSC patients may also enroll, with or without lung cysts.
Description
Lymphangioleiomyomatosis (LAM) is an uncommon disease affecting women. It is associated with cystic lung destruction and progressive respiratory failure. The Multicenter International LAM Efficacy of Sirolimus (MILES) Trial, led by the investigators' research team, demonstrated that mTOR (mammalian target of rapamycin) inhibition with sirolimus was an effective therapy that stabilized decline in FEV1 (forced expiratory volume). However, lung function decline resumed when the drug was stopped at the one year point in MILES, suggesting that therapy is suppressive rather than remission-inducing,…
Eligibility
- Age range
- 18+ years
- Sex
- Female
- Healthy volunteers
- No
Inclusion Criteria: * Female or male, age 18 or over * Diagnosis of LAM based on ATS/JRS criteria * Signed and dated informed consent * On chronic therapy, newly treated or may be considered for therapy with mTOR inhibitors or previously intolerant of or having failed mTOR inhibitor therapy Exclusion Criteria: * Inability to attend at least one RLD Clinic visit per year * Inability to give informed consent * Inability or unwillingness to perform pulmonary function testing
Interventions
- DrugSirolimus
Sirolimus treatment will be part of a participant's clinical care and will be managed by their physician.
- DrugEverolimus
Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.
Locations (20)
- Stanford University Medical CenterStanford, California
- National Jewish HealthDenver, Colorado
- Mayo Clinic JacksonvilleJacksonville, Florida
- Emory University School of MedicineAtlanta, Georgia
- Loyola University Medical Center, ChicagoMaywood, Illinois
- Brigham and Women's HospitalBoston, Massachusetts