Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial
Cancer-Related Syndrome
Unknown Primary
18 Years and older, Female
RLDC5713 (primary)
NCI-2019-08422
U01HL131755-01
Summary
This is a study to determine if early, long-term low dose sirolimus is effective for
preventing progression to more advanced stages.
Objectives
The primary objective of the MILED trial is to determine if early, long term (2 yr), low
dose (fixed at 1 mg/day) treatment of patients with well-preserved lung function will
prevent disease progression to more advanced stages. Sixty patients with FEV1>70%
predicted will be enrolled and randomized to receive 1 mg/day sirolimus or placebo, and
followed for a period of 2 years with pulmonary function testing every 4 months. The
primary endpoint will be the between-group (placebo vs. sirolimus) difference in the rate
of change in FEV1 (in liters) over two years. Secondary endpoints will include severity
grade adverse events, time to 200cc or 10% FEV1 decline, forced vital capacity, lung
volumes, diffusing capacity, serum VEGF-D, and early airflow obstruction assessed using
hyper-polarized gas MRI. The study will be conducted through the Rare Lung Disease Clinic
Network, a confederacy of clinics organized by the LAM Foundation that is currently
following over 1300 U.S. LAM patients and conducting the Department of Defense sponsored
Trial of an Aromatase Inhibitor in LAM (TRAIL) trial. The LAM Foundation will assist with
study recruitment and dissemination of results, and the University of South Florida will
function as the Data Coordinating Center. Successful completion of this study will define
the safety and efficacy of low dose sirolimus in patients with normal lung function, and
determine if sirolimus can be used to prevent disease progression to symptomatic stages.
Eligibility
- Female, age 18 or over
- Signed and dated informed consent
- Diagnosis of LAM as determined by compatible lung CT and one of the following
- biopsy (lung, abdominal mass, lymph node or kidney) or cytology from thoracic or abdominal sources revealing LAM, or
- tuberous sclerosis, angiomyolipomata (diagnosed by CT, MRI by the site radiologist or biopsy) or chylous pleural effusion (verified by tap), or
- VEGF-D level = 800 pg/ml.
- Post-bronchodilator forced expiratory volume in one second of > 70%
- Presence of markers of non-trivial burden of LAM or likely progression based on one of the following:
- pretrial FEV 1 rate of decline of >60cc/yr, comparing enrollment FEV1 to any prior measurement in the past 3 years, or
- baseline supplemental oxygen requirement with exercise, or
- pre-menopausal and one of the following (if post-menopausal, must have a VEGF-D level = 600 pg/ml and one of the following) baseline diffusing capacity for carbon monoxide =80% predicted, a) baseline residual volume =120% predicted, b) baseline desaturation by 4% or more on
six minute walk testing on room air c) more than 20 cysts on the carinal cut of the CT
Treatment Sites in Georgia
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