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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT02432560
Other study ID # MIDAS
Secondary ID 1U54HL127672
Status Recruiting
Phase
First received
Last updated
Start date March 2015
Est. completion date July 31, 2025

Study information

Verified date May 2024
Source University of Cincinnati
Contact Susan McMahan Sellers, BSN, RN
Phone (513) 558-4376
Email susan.mcmahan@uc.edu
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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, 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 600
Est. completion date July 31, 2025
Est. primary completion date July 31, 2024
Accepts healthy volunteers No
Gender Female
Age group 18 Years and older
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

Drug:
Sirolimus
Sirolimus treatment will be part of a participant's clinical care and will be managed by their physician.
Everolimus
Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.

Locations

Country Name City State
United States University of Michigan Ann Arbor Michigan
United States Emory University School of Medicine Atlanta Georgia
United States Brigham and Women's Hospital Boston Massachusetts
United States Medical University of South Carolina Charleston South Carolina
United States University of Cincinnati Cincinnati Ohio
United States Cleveland Clinic Cleveland Ohio
United States University of Texas Southwestern Medical Center Dallas Texas
United States National Jewish Health Denver Colorado
United States University of Texas Health Center Houston Texas
United States Mayo Clinic Jacksonville Jacksonville Florida
United States Loyola University Medical Center, Chicago Maywood Illinois
United States Vanderbilt University Medical Center Nashville Tennessee
United States University of Pennsylvania Medical Center Philadelphia Pennsylvania
United States Oregon Health and Science University Portland Oregon
United States Mayo Clinic Rochester Rochester Minnesota
United States University of Rochester Medical Center Rochester New York
United States Washington University School of Medicine Saint Louis Missouri
United States University of Utah School of Medicine Salt Lake City Utah
United States Swedish Medical Center Seattle Washington
United States Stanford University Medical Center Stanford California

Sponsors (5)

Lead Sponsor Collaborator
University of Cincinnati National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), Rare Diseases Clinical Research Network, The LAM Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Long term safety of mTOR inhibitor treatment in LAM Symptoms and adverse events will be recorded 2-5 years
Primary Efficacy - FEV1 slope Rate of change in FEV1 in ml/month 2-5 years
Primary Efficacy -10% reduction in FEV1 time from enrollment to 10% or greater reduction in FEV1(forced expiratory volume) in months 2-5 years
Secondary Effect of long term sirolimus on quality of life Evaluate ATAQ-LAM QOL responses over time 2-5 years
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