NCT02432560

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.

Trial Health

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Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
600

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Mar 2015

Longer than P75 for all trials

Geographic Reach
1 country

20 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2015

Completed
3 days until next milestone

First Submitted

Initial submission to the registry

March 4, 2015

Completed
2 months until next milestone

First Posted

Study publicly available on registry

May 4, 2015

Completed
10.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2025

Completed
Last Updated

December 24, 2024

Status Verified

December 1, 2024

Enrollment Period

10.4 years

First QC Date

March 4, 2015

Last Update Submit

December 18, 2024

Conditions

Keywords

LymphangioleiomyomatosisLAMrare lungRare Lung Disease

Outcome Measures

Primary Outcomes (3)

  • Long term safety of mTOR inhibitor treatment in LAM

    Symptoms and adverse events will be recorded

    2-5 years

  • Efficacy - FEV1 slope

    Rate of change in FEV1 in ml/month

    2-5 years

  • Efficacy -10% reduction in FEV1

    time from enrollment to 10% or greater reduction in FEV1(forced expiratory volume) in months

    2-5 years

Secondary Outcomes (1)

  • Effect of long term sirolimus on quality of life

    2-5 years

Study Arms (2)

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

Drug: Everolimus

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

Drug: Sirolimus

Interventions

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

Also known as: Rapamune, rapamycin
Sirolimus

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

Also known as: Afinitor
Everolimus

Eligibility Criteria

Age18 Years+
Sexfemale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Rare Lung Disease Clinical Network Consortium Clinics

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Stanford University Medical Center

Stanford, California, 94305, United States

RECRUITING

National Jewish Health

Denver, Colorado, 80206, United States

RECRUITING

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Emory University School of Medicine

Atlanta, Georgia, 33136, United States

RECRUITING

Loyola University Medical Center, Chicago

Maywood, Illinois, 60153, United States

ACTIVE NOT RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

ACTIVE NOT RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

University of Rochester Medical Center

Rochester, New York, 14642-8692, United States

RECRUITING

University of Cincinnati

Cincinnati, Ohio, 45267, United States

ACTIVE NOT RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

ACTIVE NOT RECRUITING

Oregon Health and Science University

Portland, Oregon, 97239, United States

ACTIVE NOT RECRUITING

University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, 19104, United States

ACTIVE NOT RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

ACTIVE NOT RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-2650, United States

ACTIVE NOT RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

ACTIVE NOT RECRUITING

University of Texas Health Center

Houston, Texas, 77030, United States

ACTIVE NOT RECRUITING

University of Utah School of Medicine

Salt Lake City, Utah, 84132, United States

ACTIVE NOT RECRUITING

Swedish Medical Center

Seattle, Washington, 98104, United States

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Lymphangioleiomyomatosis

Interventions

SirolimusEverolimus

Condition Hierarchy (Ancestors)

LymphangiomyomaNeoplasm, Lymphatic TissueNeoplasms by Histologic TypeNeoplasmsPerivascular Epithelioid Cell NeoplasmsNeoplasms, Connective and Soft TissueLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Francis X McCormack, MD

    University of Cincinnati

    STUDY DIRECTOR

Central Study Contacts

Susan McMahan Sellers, BSN, RN

CONTACT

Francis X McCormack, MD

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

March 4, 2015

First Posted

May 4, 2015

Study Start

March 1, 2015

Primary Completion

July 31, 2025

Study Completion

July 31, 2025

Last Updated

December 24, 2024

Record last verified: 2024-12

Locations