Safety and Durability of Sirolimus for Treatment of LAM (MIDAS)
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| ClinicalTrials.gov Identifier: NCT02432560 |
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Recruitment Status :
Recruiting
First Posted : May 4, 2015
Last Update Posted : January 13, 2022
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Sponsor:
University of Cincinnati
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
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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 |
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.
| 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 : | August 2023 |
| Estimated Study Completion Date : | August 2024 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Lymphangioleiomyomatosis
| Group/Cohort | Intervention/treatment |
|---|---|
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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
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Drug: Everolimus
Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.
Other Name: Afinitor |
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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
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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 :
- 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
- Efficacy - FEV1 slope [ Time Frame: 2 years ]forced expiratory volume (FEV1) slope over 2 years
- Efficacy -10% reduction in FEV1 [ Time Frame: 2 years ]time to 10% reduction in FEV1
- 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
- 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 :
- Effect of long term sirolimus on quality of life [ Time Frame: 2 years ]Evaluate ATAQ-LAM QOL responses over time
Information from the National Library of Medicine
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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
Contacts
| Contact: Susan McMahan Sellers, BSN, RN | (513) 558-4376 | susan.mcmahan@uc.edu | |
| Contact: Francis X McCormack, MD | (513) 558-0588 | frank.mccormack@uc.edu |
Locations
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
| Study Director: | Francis X McCormack, MD | University of Cincinnati |
| 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: | January 13, 2022 |
| Last Verified: | January 2022 |
| 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:
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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 |
MTOR Inhibitors Protein Kinase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antineoplastic Agents Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antifungal Agents |