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Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial

Status
Active
Cancer Type
Cancer-Related Syndrome
Unknown Primary
Trial Phase
Phase III
Eligibility
18 Years and older, Female
Study Type
Treatment
NCT ID
NCT03150914
Protocol IDs
RLDC5713 (primary)
NCI-2019-08422
U01HL131755-01
Study Sponsor
University of Cincinnati Cancer Center-UC Medical Center

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

  1. Female, age 18 or over
  2. Signed and dated informed consent
  3. Diagnosis of LAM as determined by compatible lung CT and one of the following
  4. biopsy (lung, abdominal mass, lymph node or kidney) or cytology from thoracic or abdominal sources revealing LAM, or
  5. tuberous sclerosis, angiomyolipomata (diagnosed by CT, MRI by the site radiologist or biopsy) or chylous pleural effusion (verified by tap), or
  6. VEGF-D level = 800 pg/ml.
  7. Post-bronchodilator forced expiratory volume in one second of > 70%
  8. Presence of markers of non-trivial burden of LAM or likely progression based on one of the following:
  9. pretrial FEV 1 rate of decline of >60cc/yr, comparing enrollment FEV1 to any prior measurement in the past 3 years, or
  10. baseline supplemental oxygen requirement with exercise, or
  11. 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,

Treatment Sites in Georgia

Emory University Hospital - Atlanta


1364 Clifton Road NE
Atlanta, GA 30322
www.emoryhealthcare.org

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