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The Efficacy and Safety of Sirolimus for Plastic Bronchitis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03942926
Recruitment Status : Unknown
Verified May 2019 by Peking Union Medical College Hospital.
Recruitment status was:  Recruiting
First Posted : May 8, 2019
Last Update Posted : May 10, 2019
Sponsor:
Collaborator:
North-China Pharmaceutical Company, China
Information provided by (Responsible Party):
Peking Union Medical College Hospital

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.

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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Acute Bronchitis
Drug Information available for: Sirolimus

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




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



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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03942926


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
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Responsible Party: Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03942926    
Other Study ID Numbers: PB1
First Posted: May 8, 2019    Key Record Dates
Last Update Posted: May 10, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The protocol and clinical study report will be shared.
Supporting Materials: Study Protocol
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

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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
Keywords provided by Peking Union Medical College Hospital:
plastic bronchitis
sirolimus
Additional relevant MeSH terms:
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Bronchitis
Acute Disease
Bronchial Diseases
Respiratory Tract Diseases
Lung Diseases, Obstructive
Lung Diseases
Respiratory Tract Infections
Disease Attributes
Pathologic Processes
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs