The Efficacy and Safety of Sirolimus for Plastic Bronchitis

Study Overview
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Brief Summary
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There is no confirmed drug therapy for plastic bronchitis. The study wish to test the effectiveness and safety of sirolimus (rapamycin) in patients with lymphatic plastic bronchitis.

Detailed Description
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Plastic bronchitis is a rare respiratory disorder characterized by formation of gelatinous or rigid cast of bronchial tree. Confirmed drug therapy for plastic bronchitis is lacking. Recently, part of plastic bronchitis has been found to have dysfunction of lymphatic circulation in the lung. Pulmonary lymphatic perfusion syndrome (PLPS) is regarded as a major mechanism of lymphatic plastic bronchitis. Sirolimus (rapamycin) has not been studied in plastic bronchitis, however, it has been shown effective in several lymphatic disorders, such as lymphangioleiomyomatosis, generalized lymphatic anomaly (GLA) including lymphangiomatosis, etc. We wish to test the effectiveness of sirolimus in lymphatic plastic bronchitis.

Official Title
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The Efficacy and Safety of Sirolimus for Plastic Bronchitis：a Pilot Study

Conditions
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Plastic Bronchitis

Intervention / Treatment
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* Drug: Sirolimus


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: plastic bronchitis pulmonary lymphatic perfusion syndrome demonstrated on 68Ga-NEB positron emission tomography (PET) Exclusion Criteria: Pregnancy and breastfeeding Severe cardiovascular, hepatic and renal dysfunction allergy to sirolimus or 68Ga-NEB

Ages Eligible for Study
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Minimum Age: 13 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Ten lymphatic bronchitis patients are planned to be recruited for the study. Sirolimus will be administered for 6 months.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sirolimus<br>Patients in sirolimus group will receive sirolimus for 6 months. | Drug: Sirolimus<br>* Patients will receive sirolimus for 6 months.<br>* Other names: rapamycin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| changes of pulmonary lymphatic perfusion distribution | quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study | 6 months | 
| changes of coughing score | measured by coughing VAS score (0-10) at baseline, 3 months and the end of study | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| changes of pulmonary function (FEV1, FVC) | measured by spirometry at baseline, 3 months and the end of study | 6 months | 
| changes of six minutes walking distance | measured by six minutes walking test at baseline, 3 months and the end of study | 6 months | 
| change of health-related quality of life | measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study | 6 months | 
| changes of breathlessness score | measured by Borg scale (0-10) at baseline, 3 months and the end of study | 6 months | 
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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plastic bronchitis, sirolimus