ClinicalTrials.gov record NCT02432560

Safety and Durability of Sirolimus for Treatment of LAM

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age18 years old and older
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a prospective, real-world registry that follows adults with lymphangioleiomyomatosis (LAM) who are taking, have previously failed or been intolerant of, or may consider taking sirolimus or everolimus. Adult female tuberous sclerosis complex (TSC) patients may also enroll, with or without lung cysts. The study collects lung function tests and adverse events obtained during routine clinical care, with follow-up planned for at least 2 years.

What it is studying. The study is collecting real-world data to evaluate long-term safety (symptoms and adverse events) and durability of mTOR inhibitor therapy (sirolimus or everolimus) in people with LAM. It will track lung function over time (FEV1 changes and time to a 10% decline) and quality-of-life measures.

What the researchers hope to learn. To describe the long-term safety profile of mTOR inhibitor treatment in LAM; to measure the rate of lung function change (FEV1 slope) and time to a 10% FEV1 decline; to evaluate quality-of-life over 2–5 years; and to help determine whether long-term suppressive sirolimus therapy is associated with prevention of progression to later stages of disease.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Sirolimus
  • Everolimus

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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 Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS). This is an observational, real world registry. The investigators propose to enroll 600 LAM patients who are on, have previously failed or been intolerant of or are considering taking sirolimus or everolimus for clinical reasons in a longitudinal observational study. This registry will follow lung function tests and adverse events that are obtained for clinical purposes over periods of at least 2 years. The decision to treat with mTOR inhibitor therapy is made by the clinician and the patient, and will be managed by the participant's clinician. This study will help us to refine treatment for patients with LAM and determine if long term suppressive therapy with sirolimus can prevent progression to later stages of disease. This research will be accomplished as part of the NIH/NCATS Rare Lung Disease Consortium, with data stored and analyzed by the Database Management Coordinating Center (DMCC) at the University of South Florida.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Provide informed consent and have their routine clinical data collected over time. The study records lung function tests (FEV1), symptoms and adverse events, and quality-of-life questionnaires (ATAQ-LAM) as obtained during clinical care, typically over at least 2 years. Participants must attend at least one RLD Clinic visit per year and be willing to perform pulmonary function testing. Treatment with sirolimus or everolimus, if used, is decided and managed by the participant's own clinician.

How the study is structured. A prospective observational registry (cohort) that follows participants over time using data from routine clinical visits. The study records lung function tests, adverse events, and quality-of-life questionnaires collected during clinical care for at least 2 years. Any sirolimus or everolimus treatment is decided and managed by the participant's own clinician.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on lymphangioleiomyomatosis

Clinical trials10 trials listed for lymphangioleiomyomatosisEvery trial the registry lists for lymphangioleiomyomatosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in lymphangioleiomyomatosis researchLatest monthly update: September 2026. Includes: Pleural fluid drainage. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
March 2015
Primary completion
Jul 31, 2025 (estimated)
Study completion
Jul 31, 2025 (estimated)
Target enrollment
600 participants (estimated)
Last updated on registry
Dec 24, 2024
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
University of Cincinnati
Principal investigator
Francis McCormack, Professor
University of Cincinnati
Collaborators
  • Rare Diseases Clinical Research Network
  • National Institutes of Health (NIH)
  • National Heart, Lung, and Blood Institute (NHLBI)
  • The LAM Foundation

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (20)

Terms used on this page · explanations by MedicalUpdates.ai

informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
registry
An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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