search
Back to results

The Efficacy and Safety of Sirolimus for Plastic Bronchitis

Primary Purpose

Plastic Bronchitis

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Sirolimus
Sponsored by
Peking Union Medical College Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Plastic Bronchitis focused on measuring plastic bronchitis, sirolimus

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Peking Union Medical College HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sirolimus

Arm Description

Patients in sirolimus group will receive sirolimus for 6 months.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

changes of pulmonary function (FEV1, FVC)
measured by spirometry at baseline, 3 months and the end of study
changes of six minutes walking distance
measured by six minutes walking test at baseline, 3 months and the end of study
change of health-related quality of life
measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study
changes of breathlessness score
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
Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion

Full Information

First Posted
May 5, 2019
Last Updated
May 8, 2019
Sponsor
Peking Union Medical College Hospital
Collaborators
North-China Pharmaceutical Company, China
search

1. Study Identification

Unique Protocol Identification Number
NCT03942926
Brief Title
The Efficacy and Safety of Sirolimus for Plastic Bronchitis
Official Title
The Efficacy and Safety of Sirolimus for Plastic Bronchitis：a Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
May 2019
Overall Recruitment Status
Unknown status
Study Start Date
May 6, 2019 (Anticipated)
Primary Completion Date
May 5, 2020 (Anticipated)
Study Completion Date
May 5, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Peking Union Medical College Hospital
Collaborators
North-China Pharmaceutical Company, China

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Plastic Bronchitis
Keywords
plastic bronchitis, sirolimus

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
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)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Sirolimus
Arm Type
Experimental
Arm Description
Patients in sirolimus group will receive sirolimus for 6 months.
Intervention Type
Drug
Intervention Name(s)
Sirolimus
Other Intervention Name(s)
rapamycin
Intervention Description
Patients will receive sirolimus for 6 months.
Primary Outcome Measure Information:
Title
changes of pulmonary lymphatic perfusion distribution
Description
quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study
Time Frame
6 months
Title
changes of coughing score
Description
measured by coughing VAS score (0-10) at baseline, 3 months and the end of study
Time Frame
6 months
Secondary Outcome Measure Information:
Title
changes of pulmonary function (FEV1, FVC)
Description
measured by spirometry at baseline, 3 months and the end of study
Time Frame
6 months
Title
changes of six minutes walking distance
Description
measured by six minutes walking test at baseline, 3 months and the end of study
Time Frame
6 months
Title
change of health-related quality of life
Description
measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study
Time Frame
6 months
Title
changes of breathlessness score
Description
measured by Borg scale (0-10) at baseline, 3 months and the end of study
Time Frame
6 months
Title
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Description
Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kai-Feng Xu
Phone
010-69155039
Email
xukf@pumch.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kai-Feng Xu, MD
Organizational Affiliation
Peking Union Medical College Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100730
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kai-Feng Xu, M.D.
Phone
86-10-69155039
Email
xukf@pumch.cn
First Name & Middle Initial & Last Name & Degree
Xinlun Tian, M.D.
Phone
86-10-69155039
Email
xinlun_t@sina.com
First Name & Middle Initial & Last Name & Degree
Kai-Feng Xu, M.D.

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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

Learn more about this trial

The Efficacy and Safety of Sirolimus for Plastic Bronchitis

We'll reach out to this number within 24 hrs