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Safety and Durability of Sirolimus for Treatment of LAM

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Observational
Eligible Ages 18 Years and Over
Gender Female
More 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT02432560
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

University of Cincinnati
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Francis X McCormack, MD
Principal Investigator Affiliation University of Cincinnati
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other, NIH
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Lymphangioleiomyomatosis
Additional Details

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.

Arms & Interventions

Arms

: 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

: 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

Interventions

Drug: - Sirolimus

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

Drug: - Everolimus

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

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Stanford University Medical Center, Stanford, California

Status

Recruiting

Address

Stanford University Medical Center

Stanford, California, 94305

Site Contact

Stephen Ruoss, MD

[email protected]

650-723-6381

National Jewish Health, Denver, Colorado

Status

Recruiting

Address

National Jewish Health

Denver, Colorado, 80206

Site Contact

Gregory Downey, MD

[email protected]

303-398-1436 #1881

Mayo Clinic Jacksonville, Jacksonville, Florida

Status

Recruiting

Address

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224

Site Contact

Charles Burger, MD

[email protected]

904-953-2381

Emory University School of Medicine, Atlanta, Georgia

Status

Recruiting

Address

Emory University School of Medicine

Atlanta, Georgia, 33136

Site Contact

Sirhari Veeraraghavan, MD

[email protected]

(513) 558-4376

Maywood, Illinois

Status

Active, not recruiting

Address

Loyola University Medical Center, Chicago

Maywood, Illinois, 60153

Brigham and Women's Hospital, Boston, Massachusetts

Status

Recruiting

Address

Brigham and Women's Hospital

Boston, Massachusetts, 02115

Site Contact

Elizabeth Henske, MD

[email protected]

857-307-0784

University of Michigan, Ann Arbor, Michigan

Status

Recruiting

Address

University of Michigan

Ann Arbor, Michigan, 48109

Site Contact

Mei Lan Han, MD

[email protected]

734-936-5047

Mayo Clinic Rochester, Rochester, Minnesota

Status

Active, not recruiting

Address

Mayo Clinic Rochester

Rochester, Minnesota, 55905

Washington University School of Medicine, Saint Louis, Missouri

Status

Recruiting

Address

Washington University School of Medicine

Saint Louis, Missouri, 63110

Site Contact

Adrian Shifren, MD

[email protected]

314-362-6904

University of Rochester Medical Center, Rochester, New York

Status

Recruiting

Address

University of Rochester Medical Center

Rochester, New York, 14642-8692

Site Contact

MaryAnne Morgan, MD

[email protected]

585-275-4161

University of Cincinnati, Cincinnati, Ohio

Status

Active, not recruiting

Address

University of Cincinnati

Cincinnati, Ohio, 45267

Cleveland Clinic, Cleveland, Ohio

Status

Active, not recruiting

Address

Cleveland Clinic

Cleveland, Ohio, 44195

Oregon Health and Science University, Portland, Oregon

Status

Active, not recruiting

Address

Oregon Health and Science University

Portland, Oregon, 97239

Philadelphia, Pennsylvania

Status

Active, not recruiting

Address

University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, 19104

Medical University of South Carolina, Charleston, South Carolina

Status

Active, not recruiting

Address

Medical University of South Carolina

Charleston, South Carolina, 29425

Vanderbilt University Medical Center, Nashville, Tennessee

Status

Active, not recruiting

Address

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-2650

Dallas, Texas

Status

Active, not recruiting

Address

University of Texas Southwestern Medical Center

Dallas, Texas, 75390

University of Texas Health Center, Houston, Texas

Status

Active, not recruiting

Address

University of Texas Health Center

Houston, Texas, 77030

University of Utah School of Medicine, Salt Lake City, Utah

Status

Active, not recruiting

Address

University of Utah School of Medicine

Salt Lake City, Utah, 84132

Swedish Medical Center, Seattle, Washington

Status

Active, not recruiting

Address

Swedish Medical Center

Seattle, Washington, 98104

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