Long-term safety and effectiveness of sirolimus for treating LAM
Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)
This study is testing how safe and effective sirolimus is for people with lymphangioleiomyomatosis (LAM) over a long period of time while keeping track of their lung health and any side effects.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 600 (estimated) |
| Ages | 18 Years and up |
| Sex | Female |
| Sponsor | University of Cincinnati Academic / other |
| Locations | 20 sites (Stanford, California and 19 other locations) |
| Trial ID | NCT02432560 on ClinicalTrials.gov |
What this trial studies
The MIDAS study is an observational registry that aims to follow patients with lymphangioleiomyomatosis (LAM) who are currently on or considering treatment with mTOR inhibitors like sirolimus or everolimus. The study will enroll 600 participants to monitor lung function and adverse events over a minimum of two years. This research builds on previous findings that indicated sirolimus can stabilize lung function decline but may require ongoing treatment. The decision to initiate therapy will be made collaboratively between the clinician and the patient.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and over diagnosed with LAM who are currently on or considering mTOR inhibitor therapy.
Not a fit: Patients unable to attend regular clinic visits or those who cannot provide informed consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide insights into the long-term safety and effectiveness of sirolimus, potentially improving treatment strategies for LAM patients.
How similar studies have performed: Previous studies have shown that mTOR inhibitors can stabilize lung function in LAM patients, but this study aims to explore long-term outcomes, making it a continuation rather than a novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Stanford, California and 19 other locations
- Stanford University Medical Center — Stanford, California, United States (Recruiting)
- National Jewish Health — Denver, Colorado, United States (Recruiting)
- Mayo Clinic Jacksonville — Jacksonville, Florida, United States (Recruiting)
- Emory University School of Medicine — Atlanta, Georgia, United States (Recruiting)
- Loyola University Medical Center, Chicago — Maywood, Illinois, United States (Active_not_recruiting)
- Brigham and Women's Hospital — Boston, Massachusetts, United States (Recruiting)
- University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
- Mayo Clinic Rochester — Rochester, Minnesota, United States (Active_not_recruiting)
- Washington University School of Medicine — St Louis, Missouri, United States (Recruiting)
- University of Rochester Medical Center — Rochester, New York, United States (Recruiting)
- University of Cincinnati — Cincinnati, Ohio, United States (Active_not_recruiting)
- Cleveland Clinic — Cleveland, Ohio, United States (Active_not_recruiting)
- Oregon Health and Science University — Portland, Oregon, United States (Active_not_recruiting)
- University of Pennsylvania Medical Center — Philadelphia, Pennsylvania, United States (Active_not_recruiting)
- Medical University of South Carolina — Charleston, South Carolina, United States (Active_not_recruiting)
- Vanderbilt University Medical Center — Nashville, Tennessee, United States (Active_not_recruiting)
- University of Texas Southwestern Medical Center — Dallas, Texas, United States (Active_not_recruiting)
- University of Texas Health Center — Houston, Texas, United States (Active_not_recruiting)
- University of Utah School of Medicine — Salt Lake City, Utah, United States (Active_not_recruiting)
- Swedish Medical Center — Seattle, Washington, United States (Active_not_recruiting)
Study contacts
- Study coordinator: Susan McMahan Sellers, BSN, RN
- Email: susan.mcmahan@uc.edu
- Phone: (513) 558-4376
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.