Ignite Creation Date:
2025-12-25 @ 2:15 AM
Ignite Modification Date:
2026-03-04 @ 9:36 PM
Study NCT ID:
NCT02432560
Status:
RECRUITING
Last Update Posted:
2024-12-24
First Post:
2015-03-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Durability of Sirolimus for Treatment of LAM
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D018192', 'term': 'Lymphangioleiomyomatosis'}], 'ancestors': [{'id': 'D008203', 'term': 'Lymphangiomyoma'}, {'id': 'D018190', 'term': 'Neoplasm, Lymphatic Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D054973', 'term': 'Perivascular Epithelioid Cell Neoplasms'}, {'id': 'D018204', 'term': 'Neoplasms, Connective and Soft Tissue'}, {'id': 'D008232', 'term': 'Lymphoproliferative Disorders'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D007160', 'term': 'Immunoproliferative Disorders'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020123', 'term': 'Sirolimus'}, {'id': 'D000068338', 'term': 'Everolimus'}], 'ancestors': [{'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 600}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2015-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2025-07-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-12-18', 'studyFirstSubmitDate': '2015-03-04', 'studyFirstSubmitQcDate': '2015-05-01', 'lastUpdatePostDateStruct': {'date': '2024-12-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-05-04', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2025-07-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Long term safety of mTOR inhibitor treatment in LAM', 'timeFrame': '2-5 years', 'description': 'Symptoms and adverse events will be recorded'}, {'measure': 'Efficacy - FEV1 slope', 'timeFrame': '2-5 years', 'description': 'Rate of change in FEV1 in ml/month'}, {'measure': 'Efficacy -10% reduction in FEV1', 'timeFrame': '2-5 years', 'description': 'time from enrollment to 10% or greater reduction in FEV1(forced expiratory volume) in months'}], 'secondaryOutcomes': [{'measure': 'Effect of long term sirolimus on quality of life', 'timeFrame': '2-5 years', 'description': 'Evaluate ATAQ-LAM QOL responses over time'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Lymphangioleiomyomatosis', 'LAM', 'rare lung', 'Rare Lung Disease'], 'conditions': ['Lymphangioleiomyomatosis']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': "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."}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Rare Lung Disease Clinical Network Consortium Clinics', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Female or male, age 18 or over\n* Diagnosis of LAM based on ATS/JRS criteria\n* Signed and dated informed consent\n* On chronic therapy, newly treated or may be considered for therapy with mTOR inhibitors or previously intolerant of or having failed mTOR inhibitor therapy\n\nExclusion Criteria:\n\n* Inability to attend at least one RLD Clinic visit per year\n* Inability to give informed consent\n* Inability or unwillingness to perform pulmonary function testing'}, 'identificationModule': {'nctId': 'NCT02432560', 'acronym': 'MIDAS', 'briefTitle': 'Safety and Durability of Sirolimus for Treatment of LAM', 'organization': {'class': 'OTHER', 'fullName': 'University of Cincinnati'}, 'officialTitle': 'Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)', 'orgStudyIdInfo': {'id': 'MIDAS'}, 'secondaryIdInfos': [{'id': '1U54HL127672', 'link': 'https://reporter.nih.gov/quickSearch/1U54HL127672', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Everolimus', 'description': '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', 'interventionNames': ['Drug: Everolimus']}, {'label': 'Sirolimus', 'description': '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', 'interventionNames': ['Drug: Sirolimus']}], 'interventions': [{'name': 'Sirolimus', 'type': 'DRUG', 'otherNames': ['Rapamune, rapamycin'], 'description': "Sirolimus treatment will be part of a participant's clinical care and will be managed by their physician.", 'armGroupLabels': ['Sirolimus']}, {'name': 'Everolimus', 'type': 'DRUG', 'otherNames': ['Afinitor'], 'description': "Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.", 'armGroupLabels': ['Everolimus']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', 'city': 'Stanford', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Stephen Ruoss, MD', 'role': 'CONTACT', 'email': 'ruoss@stanford.edu', 'phone': '650-723-6381'}], 'facility': 'Stanford University Medical Center', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Gregory Downey, MD', 'role': 'CONTACT', 'email': 'gregory.downey@gmail.com', 'phone': '303-398-1436', 'phoneExt': '1881'}], 'facility': 'National Jewish Health', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32224', 'city': 'Jacksonville', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Charles Burger, MD', 'role': 'CONTACT', 'email': 'charles.burger@mayo.edu', 'phone': '904-953-2381'}], 'facility': 'Mayo Clinic Jacksonville', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Atlanta', 'state': 'Georgia', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sirhari Veeraraghavan, MD', 'role': 'CONTACT', 'email': 'veeraraghavan@emory.edu'}], 'facility': 'Emory University School of Medicine', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60153', 'city': 'Maywood', 'state': 'Illinois', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Loyola University Medical Center, Chicago', 'geoPoint': {'lat': 41.8792, 'lon': -87.84312}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Elizabeth Henske, MD', 'role': 'CONTACT', 'email': 'ehenske@partners.org', 'phone': '857-307-0784'}], 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mei Lan Han, MD', 'role': 'CONTACT', 'email': 'mrking@umich.edu', 'phone': '734-936-5047'}], 'facility': 'University of Michigan', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Mayo Clinic Rochester', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Adrian Shifren, MD', 'role': 'CONTACT', 'email': 'ashifren@wustl.edu', 'phone': '314-362-6904'}], 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '14642-8692', 'city': 'Rochester', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'MaryAnne Morgan, MD', 'role': 'CONTACT', 'email': 'maryanne_morgan@urmc.rochester.edu', 'phone': '585-275-4161'}], 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '45267', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'University of Cincinnati', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Oregon Health and Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'University of Pennsylvania Medical Center', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '37232-2650', 'city': 'Nashville', 'state': 'Tennessee', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Vanderbilt University Medical Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'University of Texas Southwestern Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'University of Texas Health Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'University of Utah School of Medicine', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '98104', 'city': 'Seattle', 'state': 'Washington', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Swedish Medical Center', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}], 'centralContacts': [{'name': 'Susan McMahan Sellers, BSN, RN', 'role': 'CONTACT', 'email': 'susan.mcmahan@uc.edu', 'phone': '(513) 558-4376'}, {'name': 'Francis X McCormack, MD', 'role': 'CONTACT', 'email': 'frank.mccormack@uc.edu', 'phone': '(513) 558-0588'}], 'overallOfficials': [{'name': 'Francis X McCormack, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'University of Cincinnati'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Cincinnati', 'class': 'OTHER'}, 'collaborators': [{'name': 'Rare Diseases Clinical Research Network', 'class': 'NETWORK'}, {'name': 'National Institutes of Health (NIH)', 'class': 'NIH'}, {'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}, {'name': 'The LAM Foundation', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Francis McCormack', 'investigatorAffiliation': 'University of Cincinnati'}}}}