Actively Recruiting

Age: 18Years +
FEMALE
ID02432560

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

Led by University of Cincinnati · Updated on 2024-12-24

600

Participants Needed

20

Research Sites

N/A

Total Duration

On this page

Sponsors

U

University of Cincinnati

Lead Sponsor

R

Rare Diseases Clinical Research Network

Collaborating Sponsor

AI-Summary

What this Trial Is About

Lymphangioleiomyomatosis (LAM) is a rare lung disease that mainly affects women and causes lung cysts and progressive breathing problems. Researchers are studying the long-term safety and effects of mTOR inhibitors, specifically sirolimus and everolimus, in treating LAM. This observational study aims to follow patients who are currently using, have previously stopped, or may start these treatments as part of their medical care. The study is conducted by the University of Cincinnati and supported by the NIH Rare Lung Disease Consortium. Participants include women and men aged 18 or older with LAM, who are on chronic mTOR inhibitor therapy, newly treated, or may be considered for treatment. The study tracks patients receiving sirolimus or everolimus as prescribed by their doctors, without assigning treatments. It observes real-world use of these drugs over several years, focusing on safety and lung function. During the study, researchers will collect lung function tests and monitor adverse events for at least 2 years, with some outcomes followed up to 5 years. Patients will be seen at least once per year at a Rare Lung Disease Clinic, and pulmonary function testing will be performed. The main outcomes measured are the safety of long-term mTOR inhibitor use and changes in lung function, including forced expiratory volume (FEV1). Quality of life will also be evaluated. Participation requires informed consent and willingness to attend clinic visits and perform lung tests.

CONDITIONS

Brief Title

Safety and Durability of Sirolimus for Treatment of LAM

Who Can Participate

Age: 18Years +
FEMALE

Eligibility Criteria

Eligible

You may qualify if you...

  • 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
Not Eligible

You will not qualify if you...

  • Inability to attend at least one RLD Clinic visit per year
  • Inability to give informed consent
  • Inability or unwillingness to perform pulmonary function testing

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Long-term Monitoring

Duration - At least 2 years

Participants who are on, have previously failed or been intolerant of, or are considering mTOR inhibitor therapy for LAM are observed over time with periodic lung function tests and adverse event assessments as part of their clinical care.

At least 1 visit per year

Trial Site Locations

Total: 20 locations

1

Stanford University Medical Center

Stanford, California, United States, 94305

Actively Recruiting

2

National Jewish Health

Denver, Colorado, United States, 80206

Actively Recruiting

3

Mayo Clinic Jacksonville

Jacksonville, Florida, United States, 32224

Actively Recruiting

4

Emory University School of Medicine

Atlanta, Georgia, United States, 33136

Actively Recruiting

5

Loyola University Medical Center, Chicago

Maywood, Illinois, United States, 60153

Active, Not Recruiting

6

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

Actively Recruiting

7

University of Michigan

Ann Arbor, Michigan, United States, 48109

Actively Recruiting

8

Mayo Clinic Rochester

Rochester, Minnesota, United States, 55905

Active, Not Recruiting

9

Washington University School of Medicine

St Louis, Missouri, United States, 63110

Actively Recruiting

10

University of Rochester Medical Center

Rochester, New York, United States, 14642-8692

Actively Recruiting

11

University of Cincinnati

Cincinnati, Ohio, United States, 45267

Active, Not Recruiting

12

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Active, Not Recruiting

13

Oregon Health and Science University

Portland, Oregon, United States, 97239

Active, Not Recruiting

14

University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, United States, 19104

Active, Not Recruiting

15

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

Active, Not Recruiting

16

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37232-2650

Active, Not Recruiting

17

University of Texas Southwestern Medical Center

Dallas, Texas, United States, 75390

Active, Not Recruiting

18

University of Texas Health Center

Houston, Texas, United States, 77030

Active, Not Recruiting

19

University of Utah School of Medicine

Salt Lake City, Utah, United States, 84132

Active, Not Recruiting

20

Swedish Medical Center

Seattle, Washington, United States, 98104

Active, Not Recruiting

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Research Team

S

Susan McMahan Sellers, BSN, RN

F

Francis X McCormack, MD

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

2

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