The Efficacy and Safety of Sirolimus for Plastic Bronchitis

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase: Unknown; Phase 2

Conditions: Plastic Bronchitis

Treatments: Drug: Sirolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT03942926

PB1

Details and patient eligibility

About

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.

Full 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.

Enrollment: 10 estimated patients

Sex: All

Ages: 13+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

10 participants in 1 patient group

Sirolimus

Experimental group

Description:

Patients in sirolimus group will receive sirolimus for 6 months.

Treatment:

Drug: Sirolimus