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Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus Trial (The MILES Trial)

Phase 3
Completed
Conditions
ymphangioleiomyomatosis (LAM)
Registration Number
JPRN-jRCT2091220011
Lead Sponsor
Frank McCormack, M.D., University of Cincinnati Medical Center Director, Division of Pulmonary and Critical Care Medicine
Brief Summary

Sirolimus inhibits the progression of LAM and stabilize the disease activity.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
120
Inclusion Criteria

1 Age 18 or over
2 Signed and dated informed consent
3 Diagnosis of LAM as determined by biopsy; or chest CT scan findings compatible with LAM in the setting of tuberous sclerosis, angiomyolipoma or chylous pleural effusion
4 Abnormal lung function i.e., postbronchodilator FEV1 less than or equal to 70% of predicted

Exclusion Criteria

1 History of myocardial infarction or stroke related to atherosclerosis
2 Pregnancy or breast feeding
3 Inadequate contraception
4 Significant hematologic or hepatic abnormality
5 Intercurrent infection at initiation of sirolimus
6 Recent surgery (involving entry into a body cavity) within 2 months of initiation of sirolimus
7 Use of an investigational drug within the last 30 days
8 Uncontrolled hyperlipidemia
9 Previous lung transplantation
10 Inability to attend scheduled clinic visits
11 Inability to give informed consent
12 Inability to perform pulmonary function testing
13 Creatinine > 2.5 mg/dl
14 Chylous ascites sufficient to affect diaphragmatic function
15 Pleural effusion sufficient to affect pulmonary function
16 Acute pneumothorax within the past 2 months
17 Malignancy other than uncomplicated skin cancer in the past 2 years
18 Previous or current use of estrogen therapy

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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