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| Sponsors and Collaborators: |
Office of Rare Diseases (ORD) Rare Diseases Clinical Research Network |
|---|---|
| Information provided by: | Office of Rare Diseases (ORD) |
| ClinicalTrials.gov Identifier: | NCT00414648 |
Purpose
Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations. It results in the uncontrolled growth and proliferation of an unusual type of smooth muscle cell. These cells invade lung tissue, including the airways, blood vessels, and lymph vessels, and restrict the flow of air, blood, and lymph, respectively. Respiratory failure, lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the disease. This study will evaluate the safety and effectiveness of sirolimus, an immunosuppressive medication, in stabilizing or improving lung function in people with LAM.
| Condition | Intervention | Phase |
|---|---|---|
|
Lymphangioleiomyomatosis |
Drug: Sirolimus Drug: Placebo sirolimus |
Phase III |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double Blind (Subject, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
| Official Title: | Lymphangioleiomyomatosis Efficacy and Safety Trial |
| Estimated Enrollment: | 120 |
| Study Start Date: | December 2006 |
| Estimated Study Completion Date: | December 2011 |
| Estimated Primary Completion Date: | June 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Experimental
Participants will receive sirolimus daily for 1 year followed with serial pulmonary functional tests and 6-minute walk tests over a 2-year period.
|
Drug: Sirolimus
A sirolimus dose of 2 mg will be given in the form of 2 tablets (1 mg/tablet) per day for 1 year.
|
|
2: Placebo Comparator
Participants will receive placebo sirolimus daily for 1 year followed with serial pulmonary functional tests and 6-minute walk tests over a 2-year period.
|
Drug: Placebo sirolimus
A placebo dose of 2 mg will be given in the form of 2 tablets (1 mg/tablet) per day for 1 year.
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LAM is an uncommon, progressive, cystic lung disease that predominantly affects young women. It is believed to be caused by defects within cellular pathways that regulate nutrient uptake, cell size, cell migration, and cell proliferation. The disease is caused by mutations in tuberous sclerosis complex (TSC) genes. Individuals with LAM often experience pneumothorax and chylothorax, as well progressive loss of lung function. LAM is frequently fatal and existing therapies for the disease have not proven effective. Lung transplantation can be considered as a last option, but alternative treatments are needed. Sirolimus is an immunosuppressive drug that is often used in people who have had kidney transplants. It directly affects the genetic pathway that causes LAM. This study will evaluate the safety and effectiveness of sirolimus in stabilizing or improving lung function in people with LAM.
Individuals interested in participating in this two-year, double-blind study will first report to the study sites for pulmonary function testing to determine their eligibility for participation. Participants deemed eligible will be randomly assigned to receive either sirolimus or placebo for one year. Sirolimus or placebo will be administered in 2 tablet doses (2 mg for sirolimus) for the duration of the study. Study visits will occur at baseline, week 3, every three months for 12 months, and Months 18 and 24. Study visits will include a physical exam, questionnaires, a pregnancy test, blood and urine collection, and functional lung tests. A 6-minute walk test will occur at most study visits; a chest x-ray will be taken at baseline and Month 24; and a volumetric computed tomography scan will occur at baseline, Month 12, and Month 24. Adverse events, medication side effects, and lung function will be assessed at each visit.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Female |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Frank McCormack, MD | 513-558-4831 | frank.mccormack@uc.edu |
| United States, California | |
| University of California Los Angeles | Recruiting |
| Los Angeles, California, United States | |
| Principal Investigator: Joseph P. Lynch | |
| United States, Colorado | |
| National Jewish Medical and Research Center | Recruiting |
| Denver, Colorado, United States, 80206 | |
| Principal Investigator: Kevin Brown, MD, PhD | |
| United States, Florida | |
| University of Florida, Gainesville | Recruiting |
| Gainesville, Florida, United States, 32611 | |
| Principal Investigator: Mark Brantly, MD | |
| United States, Maryland | |
| National Heart, Lung, and Blood Institute | Recruiting |
| Bethesda, Maryland, United States, 20892 | |
| Contact: Joel Moss, MD | |
| Principal Investigator: Joel Moss, MD | |
| United States, Massachusetts | |
| Harvard's Brigham and Women's Hospital | Recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Principal Investigator: Edwin K. Silverman, MD, PhD | |
| United States, Ohio | |
| University of Cincinnati Medical Center | Recruiting |
| Cincinnati, Ohio, United States, 45267 | |
| Contact: Frank McCormack, MD 513-558-4831 frank.mccormack@uc.edu | |
| Principal Investigator: Frank McCormack, MD | |
| Cleveland Clinic Foundation | Recruiting |
| Cleveland, Ohio, United States, 44195 | |
| Principal Investigator: James K. Stoller, MD | |
| United States, Oregon | |
| Oregon Health & Science University | Recruiting |
| Portland, Oregon, United States, 97239 | |
| Principal Investigator: Alan F. Barker, MD | |
| United States, South Carolina | |
| Medical University of South Carolina | Recruiting |
| Charleston, South Carolina, United States, 29425 | |
| Contact: Charlie Strange, MD strangec@musc.edu | |
| Principal Investigator: Charlie Strange, MD | |
| United States, Texas | |
| University of Texas Health Center at Tyler | Recruiting |
| Tyler, Texas, United States, 75708 | |
| Principal Investigator: James M. Stocks, MD | |
| Canada, Ontario | |
| Toronto General Hospital | Recruiting |
| Toronto, Ontario, Canada, M5G 2N2 | |
| Principal Investigator: Lianne Singer, MD | |
| Japan, Niigata | |
| Niigata University Medical and Dental Hospital | Recruiting |
| Niigata-Shi, Niigata, Japan, 951-8520 | |
| Contact: Koh Nakata, MD, PhD +81 (25) 227-2022 | |
| Japan, Osaka | |
| National Kinki-Chou Hospital | Recruiting |
| Sakai, Osaka, Japan, 591-8555 | |
| Contact: Yoshikazu Inoue, MD, PhD | |
| Principal Investigator: | Frank McCormack, MD | University of Cincinnati Medical Center Division of Pulmonary and Critical Care Medicine |
| Principal Investigator: | Bruce Trapnell, MD | Cincinnati Children's Hospital Medical Center Division of Pulmonary Biology |
More Information
| Responsible Party: | University of Cincinnati Medical Center ( Frank McCormack, MD ) |
| Study ID Numbers: | RDCRN 5702, RLD 5702, 1 U54 RR019498-01 |
| Study First Received: | December 20, 2006 |
| Last Updated: | December 22, 2008 |
| ClinicalTrials.gov Identifier: | NCT00414648 |
| Health Authority: | United States: Federal Government; United States: Food and Drug Administration |
|
Lymphangiomyomatosis Lung Disease |
|
Sirolimus Lymphatic Diseases Lymphangioleiomyomatosis Immunoproliferative Disorders Clotrimazole |
Miconazole Lung Diseases Tioconazole Lymphangiomyoma Lymphoproliferative Disorders |
|
Anti-Infective Agents Neoplasms by Histologic Type Immune System Diseases Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Antibiotics, Antineoplastic |
Immunosuppressive Agents Pharmacologic Actions Lymphatic Vessel Tumors Anti-Bacterial Agents Neoplasms Therapeutic Uses Antifungal Agents |
