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出境医 / 临床实验 / The Efficacy and Safety of Sirolimus for Plastic Bronchitis

The Efficacy and Safety of Sirolimus for Plastic Bronchitis

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.

Condition or disease Intervention/treatment Phase
Plastic Bronchitis Drug: Sirolimus Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention 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)
Primary Purpose: Treatment
Official Title: The Efficacy and Safety of Sirolimus for Plastic Bronchitis：a Pilot Study
Estimated Study Start Date : May 6, 2019
Estimated Primary Completion Date : May 5, 2020
Estimated Study Completion Date : May 5, 2020
Arms and Interventions
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 Name: rapamycin
Outcome Measures
Primary Outcome Measures :
  1. changes of pulmonary lymphatic perfusion distribution [ Time Frame: 6 months ]
    quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study

  2. changes of coughing score [ Time Frame: 6 months ]
    measured by coughing VAS score (0-10) at baseline, 3 months and the end of study


Secondary Outcome Measures :
  1. changes of pulmonary function (FEV1, FVC) [ Time Frame: 6 months ]
    measured by spirometry at baseline, 3 months and the end of study

  2. changes of six minutes walking distance [ Time Frame: 6 months ]
    measured by six minutes walking test at baseline, 3 months and the end of study

  3. change of health-related quality of life [ Time Frame: 6 months ]
    measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study

  4. changes of breathlessness score [ Time Frame: 6 months ]
    measured by Borg scale (0-10) at baseline, 3 months and the end of study

  5. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 6 months ]
    Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion


Eligibility Criteria
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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations

Contacts
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Contact: Kai-Feng Xu 010-69155039 xukf@pumch.cn

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Kai-Feng Xu, M.D.    86-10-69155039    xukf@pumch.cn   
Contact: Xinlun Tian, M.D.    86-10-69155039    xinlun_t@sina.com   
Principal Investigator: Kai-Feng Xu, M.D.         
Sponsors and Collaborators
Peking Union Medical College Hospital
North-China Pharmaceutical Company, China
Investigators
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Principal Investigator: Kai-Feng Xu, MD Peking Union Medical College Hospital
Tracking Information
First Submitted Date  ICMJE May 5, 2019
First Posted Date  ICMJE May 8, 2019
Last Update Posted Date May 10, 2019
Estimated Study Start Date  ICMJE May 6, 2019
Estimated Primary Completion Date May 5, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 7, 2019)
  • changes of pulmonary lymphatic perfusion distribution [ Time Frame: 6 months ]
    quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study
  • changes of coughing score [ Time Frame: 6 months ]
    measured by coughing VAS score (0-10) at baseline, 3 months and the end of study
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 7, 2019)
  • changes of pulmonary function (FEV1, FVC) [ Time Frame: 6 months ]
    measured by spirometry at baseline, 3 months and the end of study
  • changes of six minutes walking distance [ Time Frame: 6 months ]
    measured by six minutes walking test at baseline, 3 months and the end of study
  • change of health-related quality of life [ Time Frame: 6 months ]
    measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study
  • changes of breathlessness score [ Time Frame: 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 [ Time Frame: 6 months ]
    Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE The Efficacy and Safety of Sirolimus for Plastic Bronchitis
Official Title  ICMJE The Efficacy and Safety of Sirolimus for Plastic Bronchitis：a Pilot Study
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention 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)
Primary Purpose: Treatment
Condition  ICMJE Plastic Bronchitis
Intervention  ICMJE Drug: Sirolimus
Patients will receive sirolimus for 6 months.
Other Name: rapamycin
Study Arms  ICMJE Experimental: Sirolimus
Patients in sirolimus group will receive sirolimus for 6 months.
Intervention: Drug: Sirolimus
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: May 7, 2019) 		10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 5, 2020
Estimated Primary Completion Date May 5, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03942926
Other Study ID Numbers  ICMJE PB1
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: The protocol and clinical study report will be shared.
Supporting Materials: Study Protocol
Supporting Materials: Clinical Study Report (CSR)
Time Frame: Data will be shared between time of completion of the study and time of publication of the study.
Access Criteria: contact principle investigator Dr Kai-Feng Xu via xukf@pumch.cn
Responsible Party Peking Union Medical College Hospital
Study Sponsor  ICMJE Peking Union Medical College Hospital
Collaborators  ICMJE North-China Pharmaceutical Company, China
Investigators  ICMJE
Principal Investigator: Kai-Feng Xu, MD Peking Union Medical College Hospital
PRS Account Peking Union Medical College Hospital
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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