The Efficacy and Safety of Sirolimus for Plastic Bronchitis

Plastic Bronchitis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03942926
• Other study ID #: PB1
• Status: Recruiting
• Phase: Phase 2
• Start date: May 6, 2019
• Est. completion date: May 5, 2020

Study information

• Verified date: May 2019
• Source: Peking Union Medical College Hospital
• Contact: Kai-Feng Xu
• Phone: 010-69155039
• Email: xukf[@]pumch.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: May 5, 2020
• Est. primary completion date: May 5, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Acute Disease
• Bronchitis
• Plastic Bronchitis

Intervention

Drug:
• Sirolimus
Patients will receive sirolimus for 6 months.

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing	Beijing

Sponsors (2)

Lead Sponsor	Collaborator
Peking Union Medical College Hospital	North-China Pharmaceutical Company, China

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	changes of pulmonary lymphatic perfusion distribution	quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study	6 months
Primary	changes of coughing score	measured by coughing VAS score (0-10) at baseline, 3 months and the end of study	6 months
Secondary	changes of pulmonary function (FEV1, FVC)	measured by spirometry at baseline, 3 months and the end of study	6 months
Secondary	changes of six minutes walking distance	measured by six minutes walking test at baseline, 3 months and the end of study	6 months
Secondary	change of health-related quality of life	measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study	6 months
Secondary	changes of breathlessness score	measured by Borg scale (0-10) at baseline, 3 months and the end of study	6 months
Secondary	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