Safety and Durability of Sirolimus for Treatment of LAM
Sponsored by University of Cincinnati
About this study
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.
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, and may need to be lifelong. There is therefore a need to understand whether long-term therapy with sirolimus is safe and effective. To accomplish this goal, the investigators will conduct the Multice…
Where this study is enrolling (8)
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Stanford University Medical Center
Stanford, California
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National Jewish Health
Denver, Colorado
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Mayo Clinic Jacksonville
Jacksonville, Florida
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Emory University School of Medicine
Atlanta, Georgia
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Brigham and Women's Hospital
Boston, Massachusetts
Show all 8 locations
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University of Michigan
Ann Arbor, Michigan
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Washington University School of Medicine
St Louis, Missouri
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University of Rochester Medical Center
Rochester, New York
Who can participate
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
Only the research team can confirm whether you qualify. The intake questionnaire is the best way to find out.
Completing a questionnaire on Clinably does not enroll you in a clinical trial or confirm your eligibility. Only the research team can determine whether you qualify to participate. These results are based on the information you provide and are intended to help you start a conversation with the research team.
Trial data sourced from ClinicalTrials.gov.