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Safety and Durability of Sirolimus for Treatment of LAM (MIDAS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02432560
Recruitment Status : Active, not recruiting
First Posted : May 4, 2015
Last Update Posted : September 30, 2019
Sponsor:
Collaborators:
Rare Diseases Clinical Research Network
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
The LAM Foundation
Information provided by (Responsible Party):
Francis McCormack, University of Cincinnati

Brief Summary:
The MIDAS study aims to follow 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.

Condition or disease Intervention/treatment
Lymphangioleiomyomatosis Drug: Sirolimus Drug: Everolimus

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. The investigators therefore need to know 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 "registry" trial. 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 over periods of at least 2 years. The mTOR inhibitor therapy will be initiated and managed by the participant's clinician. The study is planned to use the collected data from standard of care. 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 Rare Lung Disease Clinical Network Consortium, with data stored and analyzed by the Database Management Coordinating Center (DMCC) as part of the NIH-supported Rare Disease Consortium.

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Study Type : Observational
Estimated Enrollment : 600 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)
Actual Study Start Date : March 2015
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : April 2020

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
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
Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.
Other Name: Afinitor

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
Sirolimus treatment will be part of a participant's clinical care and will be managed by their physician.
Other Name: Rapamune, rapamycin




Primary Outcome Measures :
  1. Long term safety of mTOR inhibitor treatment in LAM [ Time Frame: 2 years ]
    Subjects will report symptoms experienced during the observation period on sirolimus/everolimus therapy

  2. Efficacy - FEV1 slope [ Time Frame: 2 years ]
    forced expiratory volume (FEV1) slope over 2 years

  3. Efficacy -10% reduction in FEV1 [ Time Frame: 2 years ]
    time to 10% reduction in FEV1

  4. Efficacy - 10% reduction in FVC1 [ Time Frame: 2 years ]
    time to 10% reduction in FVC (forced vital capacity) as compared to the placebo group from the MILES trial

  5. Efficacy - annual change in spirometry [ Time Frame: 2 years ]
    absolute annual change in spirometry [FEV1, FVC, TLC (total lung capacity), RV (residual volume) and diffusing capacity]


Secondary Outcome Measures :
  1. Effect of long term sirolimus on quality of life [ Time Frame: 2 years ]
    Evaluate of Quality of life questionnaires over 2 years



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Rare Lung Disease Clinical Network Consortium Clinics
Criteria

Inclusion Criteria:

  • Female, age 18 or over
  • Diagnosis of LAM
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02432560


Locations
Show Show 20 study locations
Sponsors and Collaborators
University of Cincinnati
Rare Diseases Clinical Research Network
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
The LAM Foundation
Investigators
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Study Director: Francis X McCormack, MD University of Cincinnati
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Responsible Party: Francis McCormack, Professor, University of Cincinnati
ClinicalTrials.gov Identifier: NCT02432560    
Other Study ID Numbers: MIDAS
1U54HL127672 ( U.S. NIH Grant/Contract )
First Posted: May 4, 2015    Key Record Dates
Last Update Posted: September 30, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Francis McCormack, University of Cincinnati:
Lymphangioleiomyomatosis
LAM
rare lung
Rare Lung Disease
Additional relevant MeSH terms:
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Lymphangioleiomyomatosis
Lymphangiomyoma
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Sirolimus
Everolimus
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents