Status:
COMPLETED
Efficacy and Safety of Sirolimus in LAM
Lead Sponsor:
University of Cincinnati
Collaborating Sponsors:
Office of Rare Diseases (ORD)
FDA Office of Orphan Products Development
Conditions:
Lymphangioleiomyomatosis
Eligibility:
FEMALE
18+ years
Phase:
PHASE3
Brief Summary
Lymphangioleiomyomatosis (LAM) is a rare lung disease of women that is caused by genetic mutations. It results in the uncontrolled growth of an unusual type of smooth muscle cell in the lung. These ce...
Detailed Description
LAM is an uncommon, progressive, cystic lung disease that predominantly affects young women. The disease is caused by mutations in tuberous sclerosis complex (TSC) genes, which regulate cellular pathw...
Eligibility Criteria
Inclusion
- Age 18 or older
- Signed and dated informed consent
- Diagnosis of LAM based on compatible chest CT scan and a) biopsy or cytology consistent with LAM, or b) presence of tuberous sclerosis, angiomyolipoma or chylous pleural effusion; or c) a VEGF-D level of at least 800 pg/ml
- Forced expiratory volume in one second (FEV1) of 70% or less of predicted value after administration of a bronchodilator
Exclusion
- Known allergy to sirolimus
- History of heart attack, angina, or stroke due to clogging, narrowing, and hardening of the arteries and blood vessels
- Significant hematologic or hepatic abnormality (transaminase levels greater than three times the upper limit of normal, HCT less than 30%, platelets less than 80,000/cubic mm, adjusted absolute neutrophil count less than 1,000/cubic mm, total white blood cell count less than 3,000/cubic mm)
- Intercurrent infection at the time treatment with sirolimus begins
- Any surgery involving entry into a body cavity or requiring three or more sutures within 8 weeks of initiation of study drug
- Use of an investigational drug within the 30 days prior to random assignment
- Uncontrolled hyperlipidemia
- Previous lung transplant or currently on lung transplant list
- Unable to attend scheduled study visits
- Unable to perform pulmonary function tests
- Creatinine levels greater than 2.5 mg/dl
- Chylous ascites severe enough to affect diaphragmatic function
- Pleural effusion severe enough to affect pulmonary function, as determined by the study physician
- History of acute pneumothorax within the 2 months prior to study entry
- History of malignancy within the 2 years prior to study entry (except for squamous or basal cell skin cancer)
- Use of estrogen containing medication within the thirty days prior to randomization
- Unable or unwilling to use adequate contraception
- Pregnant, breastfeeding, or plans to become pregnant within the next 2 years
Key Trial Info
Start Date :
December 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2011
Estimated Enrollment :
89 Patients enrolled
Trial Details
Trial ID
NCT00414648
Start Date
December 1 2006
End Date
February 1 2011
Last Update
November 2 2023
Active Locations (13)
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1
University of California Los Angeles
Los Angeles, California, United States, 90024
2
National Jewish Medical and Research Center
Denver, Colorado, United States, 80206
3
University of Florida, Gainesville
Gainesville, Florida, United States, 32611
4
National Heart, Lung, and Blood Institute
Bethesda, Maryland, United States, 20892