The Efficacy and Safety of Sirolimus for Plastic Bronchitis
- Registration Number
- NCT03942926
- Lead Sponsor
- Peking Union Medical College Hospital
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 10
- plastic bronchitis
- pulmonary lymphatic perfusion syndrome demonstrated on 68Ga-NEB positron emission tomography (PET)
- Pregnancy and breastfeeding
- Severe cardiovascular, hepatic and renal dysfunction
- allergy to sirolimus or 68Ga-NEB
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Sirolimus Sirolimus Patients in sirolimus group will receive sirolimus for 6 months.
- Primary Outcome Measures
Name Time Method changes of coughing score 6 months measured by coughing VAS score (0-10) at baseline, 3 months and the end of study
changes of pulmonary lymphatic perfusion distribution 6 months quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study
- Secondary Outcome Measures
Name Time Method changes of breathlessness score 6 months measured by Borg scale (0-10) at baseline, 3 months and the end of study
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 6 months Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion
change of health-related quality of life 6 months measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study
changes of pulmonary function (FEV1, FVC) 6 months measured by spirometry at baseline, 3 months and the end of study
changes of six minutes walking distance 6 months measured by six minutes walking test at baseline, 3 months and the end of study
Trial Locations
- Locations (1)
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China