The Efficacy and Safety of Sirolimus for Plastic Bronchitis

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

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.

Study Overview

• Status: Unknown
• Conditions: Plastic Bronchitis
• Intervention / Treatment: Drug: Sirolimus

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.

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Kai-Feng Xu; Phone Number: 010-69155039; Email: xukf@pumch.cn

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100730
      • Recruiting
      • Peking Union Medical College Hospital
      • Contact: Kai-Feng Xu, M.D.; Phone Number: 86-10-69155039; Email: xukf@pumch.cn
      • Contact: Xinlun Tian, M.D.; Phone Number: 86-10-69155039; Email: xinlun_t@sina.com
      • Principal Investigator: Kai-Feng Xu, M.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 13 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Sirolimus; Patients in sirolimus group will receive sirolimus for 6 months.	Drug: Sirolimus; Patients will receive sirolimus for 6 months.; Other Names: rapamycin

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Collaborators: North-China Pharmaceutical Company, China
• Investigators: Principal Investigator: Kai-Feng Xu, MD, Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Anticipated): May 6, 2019
• Primary Completion (Anticipated): May 5, 2020
• Study Completion (Anticipated): May 5, 2020

Study Registration Dates

• First Submitted: May 5, 2019
• First Submitted That Met QC Criteria: May 7, 2019
• First Posted (Actual): May 8, 2019

Study Record Updates

• Last Update Posted (Actual): May 10, 2019
• Last Update Submitted That Met QC Criteria: May 8, 2019
• Last Verified: May 1, 2019

More Information

Terms related to this study

• Keywords: sirolimus; plastic bronchitis
• Additional Relevant MeSH Terms: Pathologic Processes; Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Lung Diseases; Disease Attributes; Bronchial Diseases; Lung Diseases, Obstructive; Acute Disease; Bronchitis; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: PB1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: The protocol and clinical study report will be shared.
• IPD Sharing Time Frame: Data will be shared between time of completion of the study and time of publication of the study.
• IPD Sharing Access Criteria: contact principle investigator Dr Kai-Feng Xu via xukf@pumch.cn
• IPD Sharing Supporting Information Type: Study Protocol; Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No