Recruiting

SirolimusSafety and Durability of Sirolimus for Treatment of LAM

Sponsor:

University of Cincinnati

Code:

NCT02432560

Conditions

Lymphangioleiomyomatosis

Eligibility Criteria

Sex: Female

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Sirolimus

Everolimus

Study Details

Brief 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.

Conditions

Lymphangioleiomyomatosis

Study ID

NCT02432560

Start date

Mar, 2015

Status verified date

Dec, 2024

Completion date

Jul 31, 2025

Anticipated

Primary completion date

Jul 31, 2025

Anticipated

Eligibility Criteria

Sex: Female

Age: 18+

Healthy Volunteers: Not accepted

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

Enrollment

600 participants

Anticipated

Interventions and Outcome Measures

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

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.

Primary outcome measure

Long term safety of mTOR inhibitor treatment in LAM [ Time Frame: 2-5 years ]
Efficacy - FEV1 slope [ Time Frame: 2-5 years ]
Efficacy -10% reduction in FEV1 [ Time Frame: 2-5 years ]

Central Contacts and Locations

Central contacts

Susan McMahan Sellers, BSN, RN

(513) 558-4376 susan.mcmahan@uc.edu

Francis X McCormack, MD

(513) 558-0588 frank.mccormack@uc.edu

Locations

Stanford University Medical Center

Recruiting

Stanford, California, United States, 94305

Contacts

Stephen Ruoss, MD

650-723-6381 ruoss@stanford.edu

National Jewish Health

Recruiting

Denver, Colorado, United States, 80206

Contacts

Gregory Downey, MD

303-398-1436 gregory.downey@gmail.com

Mayo Clinic Jacksonville

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Charles Burger, MD

904-953-2381 charles.burger@mayo.edu

Emory University School of Medicine

Recruiting

Atlanta, Georgia, United States, 33136

Contacts

Sirhari Veeraraghavan, MD

veeraraghavan@emory.edu

Brigham and Women's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Elizabeth Henske, MD

857-307-0784 ehenske@partners.org

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Mei Lan Han, MD

734-936-5047 mrking@umich.edu

Washington University School of Medicine

Recruiting

Saint Louis, Missouri, United States, 63110

Contacts

Adrian Shifren, MD

314-362-6904 ashifren@wustl.edu

University of Rochester Medical Center

Recruiting

Rochester, New York, United States, 14642-8692

Contacts

MaryAnne Morgan, MD

585-275-4161 maryanne_morgan@urmc.rochester.edu

More Information

Sponsor

University of Cincinnati

Last update posted

Dec 24, 2024

Last verified

Dec, 2024

Keywords

Lymphangioleiomyomatosis
LAM
rare lung
Rare Lung Disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by University of Cincinnati on 2024-12-24.