Safety and Durability of Sirolimus for Treatment of LAM

• Status: Active, not recruiting
• Trial end date: 2021-08-01
• Target enrollment: 0
• Participant gender: Female

Summary

The MIDAS study aims to follow LAM patients who are currently taking, have previously failed or been intolerant of, or may (at some time in the future) take mTOR inhibitors (sirolimus or everolimus) as part of their clinical care. Adult female TSC patients may also enroll, with or without lung cysts.

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati; University of Cincinnati
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health (NIH); Rare Diseases Clinical Research Network; The LAM Foundation
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Female, age 18 or over

• Diagnosis of LAM

• Signed and dated informed consent

• On chronic therapy, newly treated or may be considered for therapy with mTOR inhibitors or previously intolerant of or having failed mTOR inhibitor therapy

Exclusion Criteria:

• Inability to attend at least one RLD Clinic visit per year

• Inability to give informed consent

• Inability or unwillingness to perform pulmonary function testing