Health TrialsbyHealthPanel

Safety and Durability of Sirolimus for Treatment of LAM

ConditionLymphangioleiomyomatosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexFemale
Age18+
SponsorUniversity of Cincinnati

About this trial

The MIDAS study aims to follow male and female 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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Female or male, age 18 or over

Diagnosis of LAM based on ATS/JRS criteria

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

Disqualifiers

Inability to attend at least one RLD Clinic visit per year

Inability to give informed consent

Inability or unwillingness to perform pulmonary function testing

Trial population

Rare Lung Disease Clinical Network Consortium Clinics

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Sirolimus

    Drug

    Sirolimus treatment will be part of a participant's clinical care and will be managed by their physician.

  • Everolimus

    Drug

    Everolimus treatment will be part of a participant's clinical care and will be managed by their physician.

Treatment groups

600 Participants
are divided into 2 treatment groups
Group A: Everolimus1 intervention
Group B: Sirolimus1 intervention

Trial outcomes

Primary outcomes

1

Long term safety of mTOR inhibitor treatment in LAM

Symptoms and adverse events will be recorded

Time frame
2-5 years
2

Efficacy - FEV1 slope

Rate of change in FEV1 in ml/month

Time frame
2-5 years
3

Efficacy -10% reduction in FEV1

time from enrollment to 10% or greater reduction in FEV1(forced expiratory volume) in months

Time frame
2-5 years

Secondary outcomes

1

Effect of long term sirolimus on quality of life

Evaluate ATAQ-LAM QOL responses over time

Time frame
2-5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Cincinnati

Lead sponsor

Rare Diseases Clinical Research Network

Collaborator

National Institutes of Health (NIH)

Collaborator

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

The LAM Foundation

Collaborator

Source ClinicalTrials.gov
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