University of Cincinnati
Francis McCormack
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
Sirolimus
Everolimus
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.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 600 participants |
| Official Title : | Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS) |
| Actual Study Start Date : | 2015-03 |
| Estimated Primary Completion Date : | 2025-07-31 |
| Estimated Study Completion Date : | 2025-07-31 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | 18 Years |
| Sexes Eligible for Study: | FEMALE |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
RECRUITING
Stanford University Medical Center
Stanford, California, United States, 94305
RECRUITING
National Jewish Health
Denver, Colorado, United States, 80206
RECRUITING
Mayo Clinic Jacksonville
Jacksonville, Florida, United States, 32224
RECRUITING
Emory University School of Medicine
Atlanta, Georgia, United States, 33136
ACTIVE NOT RECRUITING
Loyola University Medical Center, Chicago
Maywood, Illinois, United States, 60153
RECRUITING
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
RECRUITING
University of Michigan
Ann Arbor, Road cancer, United States, 48109
ACTIVE NOT RECRUITING
Mayo Clinic Rochester
Rochester, Minnesota, United States, 55905
RECRUITING
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
RECRUITING
University of Rochester Medical Center
Rochester, New York, United States, 14642-8692
ACTIVE NOT RECRUITING
University of Cincinnati
Cincinnati, Ohio, United States, 45267
ACTIVE NOT RECRUITING
Cleveland Clinic
Cleveland, Ohio, United States, 44195
ACTIVE NOT RECRUITING
Oregon Health and Science University
Portland, Oregon, United States, 97239
ACTIVE NOT RECRUITING
University of Pennsylvania Medical Center
Philadelphia, Pennsylvania, United States, 19104
ACTIVE NOT RECRUITING
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
ACTIVE NOT RECRUITING
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-2650
ACTIVE NOT RECRUITING
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
ACTIVE NOT RECRUITING
University of Texas Health Center
Houston, Texas, United States, 77030
ACTIVE NOT RECRUITING
University of Utah School of Medicine
Salt Lake City, Utah, United States, 84132
ACTIVE NOT RECRUITING
Swedish Medical Center
Seattle, Washington, United States, 98104