Status: RECRUITING
Background
Lymphangioleiomyomatosis (LAM) is an ongoing lung disease causing cysts that affects 30-40% of women with tuberous sclerosis complex (TSC), and about 4-7 women per million in the population at large who do not inherit a genetic disease. Patients with LAM lose the use of their lungs rather quickly.
The average age for diagnosis of LAM is 35 years. Within 10 years of a women having her first symptom, 55% of patients are breathless when walking on flat ground, 20% require supplemental oxygen and 10% are deceased. Recent data suggests the average survival time once symptoms begin to be 15-30 years.
Studies of patients with non-inherited LAM and of those patients who are re-diagnosed with LAM after lung transplant have shown uncontrolled growth of muscle cells in the lungs. A relationship between LAM and the tuberous sclerosis genes have been found. These genes cause irregular cell growth and release of lymphangiogenic growth hormone into the body.
A previous study (MILES) showed an increase in lung function for those patients who were taking sirolimus. Lung function was determined by pulmonary function testing, which measures how well a person moves air into and out of their lungs. When sirolimus was ended, patients' lung function began to deteriorate again. The MILES study showed that sirolimus stabilized lung function and improved quality of life for LAM patients.
The research questions are:
- What are the demographic and clinical details of LAM patients treated with long-term sirolimus or everolimus?
- What safety concerns are there when LAM patients take sirolimus or everolimus long-term? What symptoms do LAM patients who take sirolimus or everolimus have while taking the drug long-term? How often and why do LAM patients stop taking sirolimus or everolimus? How often are LAM patients taking sirolimus or everolimus hospitalized or receive lung transplants? What is the rate of death of LAM patients taking siroliums or everolimus?
- How does lung function compare in LAM patients who take sirolimus or everolimus long-term versus those who do not take either medication?
About this Study
The purpose of this study is to create a registry database of patients diagnosed with LAM who are taking or planning to take sirolimus or everolimus. From this database researchers will be able to collect and analyze data to determine if long-term sirolimus or everolimus use is helpful and safe for LAM patients.
There will be a total of 300 participants with LAM in this study.
Subjects that agree to be in the study will be asked to participate in the following:
- Review, ask questions about and sign an informed consent
- Attend at least one annual visit to an RLD clinic
- Perform all standard testing during their visit(s) to the RLD clinic
- Follow treatment instructions from the clinician at their RLD clinic
Targeted Enrollment
To be eligible to participate, you must be:
- Female, age 18 or over
- Diagnosed with LAM
- Currently taking sirolimus or everolimus, previously having failed or been intolerant of sirolimus or everolimus, newly treated with sirolimus or everolimus, or considering therapy with sirolimus or everolimus
You are not eligible to participate if:
- You are unable to attend at least one RLD clinic visit per year
- You are not able to provide informed consent for participation
- You are not able or willing to perform lung function testing
How to participate:
We greatly appreciate your interest in this study and in helping us understand the long-term treatment of LAM with sirolimus or everolimus.
In order to participate, you must personally contact the study coordinator of any of the participating institutions by phone or by email. Please use the information below to inquire about participation:
California
Susan Jacobs, RN, MS
Nurse Coordinator
Stanford University Medical Center
Phone: 650-725-8083
E-mail: ssjpulm@stanford.edu
Colorado
Carol Bair, RCP, CRC
Site Coordinator
National Jewish Health
Phone: 303-398-1912
E-mail: BairC@NJHealth.org
Florida
Shannon Kennedy, CCRP
Lead Certified Clinical Research Coordinator
Mayo Clinic - Jacksonville
Phone: 904-953-7439
E-mail: Kennedy.Shannon@mayo.edu
Georgia
Tracy Halaby, RN
Research Nurse
Emory University School of Medicine
Phone: 404-712-7458
E-mail: tracy.halaby@emory.edu
Illinois
Josefina Corral, MSN
Clinical Research Nurse
Loyola University - Health Science Center
Phone: 708-216-5744
E-mail: Jcorral@luc.edu
Massachusetts
Shefali Bagwe, MBBS
Clinical Research Coordinator
Brigham and Women's Hospital - Harvard Medical School
Phone: 857-307-0784
E-mail: sbagwe@bwh.Harvard.edu
Michigan
Catherine Meldrum, PhD, RN, MS, CCRC
Study Coordinator
University of Michigan
Phone: 734-647-7840
E-mail: cathymel@umich.edu
Minnesota
Tami Krpata
Study Coordinator
Mayo Clinic - Rochester
Phone: 507-538-1979
E-mail: krpata.tami@mayo.edu
Missouri
Hannah Perkins
Study Coordinator
Washington University School of Medicine
Phone: 314-362-9919
E-mail: hperkins@wustl.edu
New York
Elizabeth Lyda
Study Coordinator
University of Rochester Medical Center
Phone: 585-233-4358
E-mail: elizabeth_lyda@urmc.rochester.edu
Ohio
Joanne Baran, RN
Study Coordinator
Cleveland Clinic Foundation
Phone: 216-444-5023
E-mail: baranj2@ccf.org
Susan McMahan, RN
Research Nurse
University of Cincinnati
Phone: 513-558-2148
E-mail: mcmahasn@ucmail.uc.edu
Oregon
Samantha Ruimy, MS
Study Coordinator
Oregon Health & Science University
Phone: 503-494-0724
E-mail: ruimy@ohsu.edu
Pennsylvania
Carly D’Errico
Study Coordinator
University of Pennsylvania Medical Center
Phone: 215-746-4219
E-mail: derricoc@mail.med.upenn.edu
South Carolina
Suchit Kumbhare, MBBS, MS, CCRP
Study Coordinator
Medical University of South Carolina
Phone: 843-822-4945
E-mail: kumbhare@musc.edu
Tennessee
Errine Garnett
Study Coordinator
Vanderbilt University Medical Center
Phone: 615-343-0539
E-mail: Errine.T.Garnett@vanderbilt.edu
Texas
Tiffany Ostovar-Kermani, MD, MPH
Study Coordinator
The University of Texas Health Science Center at Houston
Phone: 713-500-6851
E-mail: Tiffany.G.OstovarKermani@uth.tmc.edu
Adetoun Sodimu, MPH, CPH
Clinical Research Manager
University of Texas Souhtwestern Medical Center
Phone: 214-645-6493
E-mail: Adetoun.Sodimu@utsouthwestern.edu
Utah
Silvia Smith, Ph.D
Study Coordinator
University of Utah School of Medicine
Phone: 801-581-5811
E-mail: Silvia.smith@hsc.utah.edu
Washington
Julie Wallick
Research Coordinator Lead
Swedish Medical Center
Phone: 206-215-3986
E-mail: julie.wallick@swedish.org