LymphangioleiomyomatosisMIDASNCT02432560
Safety and Durability of Sirolimus for Treatment of LAM
An observational study of Lymphangioleiomyomatosis, run by University of Cincinnati at 20 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Lymphangioleiomyomatosis
- What is being tested?
- Sirolimus and Everolimus
- How many people
- 600 participants
- Where
- 20 sitesUnited States
- Study dates
- Mar 2015to 31 Jul 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Study population: Rare Lung Disease Clinical Network Consortium Clinics
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
20 sites in 1 country. Contact the study team or a site near you to ask about taking part.
20 sites in 1 country
United States20 sites, 8 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Mei Lan Han, MD, 734-936-5047, mrking@umich.edu
- Recruiting
Emory University School of Medicine
Atlanta, Georgia, 33136
Sirhari Veeraraghavan, MD, veeraraghavan@emory.edu
- Recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Elizabeth Henske, MD, 857-307-0784, ehenske@partners.org
- Active, not recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
- Active, not recruiting
University of Cincinnati
Cincinnati, Ohio, 45267
- Active, not recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
- Active, not recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
National Jewish Health
Denver, Colorado, 80206
Gregory Downey, MD, 303-398-1436 ext. 1881, gregory.downey@gmail.com
- Active, not recruiting
University of Texas Health Center
Houston, Texas, 77030
- Recruiting
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
Charles Burger, MD, 904-953-2381, charles.burger@mayo.edu
- Active, not recruiting
Loyola University Medical Center, Chicago
Maywood, Illinois, 60153
- Active, not recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-2650
- Active, not recruiting
University of Pennsylvania Medical Center
Philadelphia, Pennsylvania, 19104
- Active, not recruiting
Oregon Health and Science University
Portland, Oregon, 97239
- Active, not recruiting
Mayo Clinic Rochester
Rochester, Minnesota, 55905
- Recruiting
University of Rochester Medical Center
Rochester, New York, 14642-8692
MaryAnne Morgan, MD, 585-275-4161, maryanne_morgan@urmc.rochester.edu
- Active, not recruiting
University of Utah School of Medicine
Salt Lake City, Utah, 84132
- Active, not recruiting
Swedish Medical Center
Seattle, Washington, 98104
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Adrian Shifren, MD, 314-362-6904, ashifren@wustl.edu
- Recruiting
Stanford University Medical Center
Stanford, California, 94305
Stephen Ruoss, MD, 650-723-6381, ruoss@stanford.edu
About this study
Official title: Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)
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.
Read the detailed description
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
- Lymphangioleiomyomatosis
- Being tested
- Sirolimus, Everolimus
- Keywords
- Lymphangioleiomyomatosis, LAM, rare lung, Rare Lung Disease
- Medical subject headings
- Lymphangioleiomyomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune, rapamycin
Sirolimus treatment will be part of a participant's clinical care and will be managed by their physician.
EverolimusDrug
Also called Afinitor
Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long term safety of mTOR inhibitor treatment in LAM
Symptoms and adverse events will be recorded
Measured: 2-5 years
Efficacy - FEV1 slope
Rate of change in FEV1 in ml/month
Measured: 2-5 years
Efficacy -10% reduction in FEV1
time from enrollment to 10% or greater reduction in FEV1(forced expiratory volume) in months
Measured: 2-5 years
Secondary outcomes (1)
Effect of long term sirolimus on quality of life
Evaluate ATAQ-LAM QOL responses over time
Measured: 2-5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2015 (estimated)
- Study startMar 2015
- Primary completion31 Jul 2025 (estimated)
- Study completion31 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LymphangioleiomyomatosisStudies of the same condition, open and under way.
- More trials run by University of CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 600 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Rare Diseases Clinical Research Network, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), The LAM Foundation
- Responsible party
- Principal investigator, Francis McCormack, University of Cincinnati
- Investigators
- Francis X McCormack, MD, Study director, University of Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02432560
- Sponsor's protocol ID
- MIDAS
- Other IDs
- 1U54HL127672
- Acronym
- MIDAS
- Status last verified
- Dec 2024
- First submitted
- 4 Mar 2015
- Last updated
- 24 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)
- ClinicalTrials.gov ID
- NCT02432560
- Acronym / sponsor ID
- MIDAS / MIDAS
- Conditions
- Lymphangioleiomyomatosis
- Keywords
- Lymphangioleiomyomatosis, LAM, rare lung, Rare Lung Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 600 participants (estimated)
- Lead sponsor
- University of CincinnatiUniversity, hospital or other
- Collaborators
- Rare Diseases Clinical Research Network, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), The LAM Foundation
- Study start
- Mar 2015 (actual)
- Primary completion
- 31 Jul 2025 (estimated)
- Study completion
- 31 Jul 2025 (estimated)
- First posted
- 4 May 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Everolimus | – | Drug: Everolimuswomen 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 | – | Drug: Sirolimuswomen 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Rare Lung Disease Clinical Network Consortium Clinics
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- Inability to attend at least one RLD Clinic visit per year
- Inability to give informed consent
- Inability or unwillingness to perform pulmonary function testing
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: Rapamune, rapamycinSirolimus treatment will be part of a participant's clinical care and will be managed by their physician. | Drug | Sirolimus |
| EverolimusAlso: AfinitorEverolimus treatment will be part of a participant's clinical care and will be managed by their physician. | Drug | Everolimus |
Primary outcome
Long term safety of mTOR inhibitor treatment in LAM
Time frame: 2-5 years
Symptoms and adverse events will be recorded
Efficacy - FEV1 slope
Time frame: 2-5 years
Rate of change in FEV1 in ml/month
Efficacy -10% reduction in FEV1
Time frame: 2-5 years
time from enrollment to 10% or greater reduction in FEV1(forced expiratory volume) in months
1 secondary outcome
Effect of long term sirolimus on quality of life
Time frame: 2-5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Francis X McCormack, MD study director
University of Cincinnati
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lymphangioleiomyomatosis, run by University of Cincinnati at 20 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Susan McMahan Sellers, BSN, RN ((513) 558-4376, susan.mcmahan@uc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long term safety of mTOR inhibitor treatment in LAM (time frame: 2-5 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Dec 2024.