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Safety and Durability of Sirolimus for Treatment of LAM

NCT ID: NCT02432560

Last Updated: 2024-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

600 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-03-31

Study Completion Date

2025-07-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Lymphangioleiomyomatosis

Keywords

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Lymphangioleiomyomatosis LAM rare lung Rare Lung Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Everolimus

women over age 18 who have LAM and are currently taking, have previously failed or been intolerant of, or who are considering taking everolimus as part of their clinical care

Everolimus

Intervention Type DRUG

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

Sirolimus

women over age 18 who have LAM and are currently taking, have previously failed or been intolerant of, or who are considering taking sirolimus as part of their clinical care

Sirolimus

Intervention Type DRUG

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

Interventions

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Sirolimus

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

Intervention Type DRUG

Everolimus

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

Intervention Type DRUG

Other Intervention Names

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Rapamune, rapamycin Afinitor

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Rare Diseases Clinical Research Network

NETWORK

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

The LAM Foundation

OTHER

Sponsor Role collaborator

University of Cincinnati

OTHER

Sponsor Role lead

Responsible Party

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Francis McCormack

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Francis X McCormack, MD

Role: STUDY_DIRECTOR

University of Cincinnati

Locations

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Stanford University Medical Center

Stanford, California, United States

Site Status RECRUITING

National Jewish Health

Denver, Colorado, United States

Site Status RECRUITING

Mayo Clinic Jacksonville

Jacksonville, Florida, United States

Site Status RECRUITING

Emory University School of Medicine

Atlanta, Georgia, United States

Site Status RECRUITING

Loyola University Medical Center, Chicago

Maywood, Illinois, United States

Site Status ACTIVE_NOT_RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status ACTIVE_NOT_RECRUITING

Washington University School of Medicine

St Louis, Missouri, United States

Site Status RECRUITING

University of Rochester Medical Center

Rochester, New York, United States

Site Status RECRUITING

University of Cincinnati

Cincinnati, Ohio, United States

Site Status ACTIVE_NOT_RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status ACTIVE_NOT_RECRUITING

Oregon Health and Science University

Portland, Oregon, United States

Site Status ACTIVE_NOT_RECRUITING

University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, United States

Site Status ACTIVE_NOT_RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status ACTIVE_NOT_RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status ACTIVE_NOT_RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status ACTIVE_NOT_RECRUITING

University of Texas Health Center

Houston, Texas, United States

Site Status ACTIVE_NOT_RECRUITING

University of Utah School of Medicine

Salt Lake City, Utah, United States

Site Status ACTIVE_NOT_RECRUITING

Swedish Medical Center

Seattle, Washington, United States

Site Status ACTIVE_NOT_RECRUITING

Countries

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United States

Central Contacts

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Susan McMahan Sellers, BSN, RN

Role: CONTACT

Phone: (513) 558-4376

Email: [email protected]

Francis X McCormack, MD

Role: CONTACT

Phone: (513) 558-0588

Email: [email protected]

Facility Contacts

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Stephen Ruoss, MD

Role: primary

Gregory Downey, MD

Role: primary

Charles Burger, MD

Role: primary

Sirhari Veeraraghavan, MD

Role: primary

Elizabeth Henske, MD

Role: primary

Mei Lan Han, MD

Role: primary

Adrian Shifren, MD

Role: primary

MaryAnne Morgan, MD

Role: primary

Other Identifiers

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1U54HL127672

Identifier Type: NIH

Identifier Source: secondary_id

View Link

MIDAS

Identifier Type: -

Identifier Source: org_study_id