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Efficacy and Safety of Sirolimus in LAM

NCT00414648 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 89

Last updated 2023-11-02

Study results available
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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 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 inhibitor of the mTOR pathway, in stabilizing or improving lung function in people with LAM.

Conditions

  • Lymphangioleiomyomatosis

Interventions

DRUG

Sirolimus

A sirolimus dose of 2 tablets (1 mg/tablet) per day for 1 year.

DRUG

Placebo

A placebo dose of 2 tablets per day for 1 year.

Sponsors & Collaborators

  • Office of Rare Diseases (ORD)

    collaborator NIH

  • FDA Office of Orphan Products Development

    collaborator FED

  • National Center for Research Resources (NCRR)

    collaborator NIH

  • University of Cincinnati

    lead OTHER

Principal Investigators

  • Francis X McCormack, MD · University of Cincinnati Medical Center Division of Pulmonary and Critical Care Medicine

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-12-31
Primary Completion
2010-09-30
Completion
2011-02-28
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Japan

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00414648 on ClinicalTrials.gov