Plastic BronchitisPhase 2NCT03942926
The Efficacy and Safety of Sirolimus for Plastic Bronchitis
A Phase 2 study testing Sirolimus in people with Plastic Bronchitis, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Plastic Bronchitis
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 6 May 2019to 5 May 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- plastic bronchitis
- pulmonary lymphatic perfusion syndrome demonstrated on 68Ga-NEB positron emission tomography (PET)
Who cannot join
- Pregnancy and breastfeeding
- Severe cardiovascular, hepatic and renal dysfunction
- allergy to sirolimus or 68Ga-NEB
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Kai-Feng Xu, M.D., 86-10-69155039, xukf@pumch.cn
Xinlun Tian, M.D., 86-10-69155039, xinlun_t@sina.com
About this study
Official title: 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.
Read the 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.
- Conditions
- Plastic Bronchitis
- Being tested
- Sirolimus
- Keywords
- plastic bronchitis, sirolimus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called rapamycin
Patients will receive sirolimus for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Experimental | Patients in sirolimus group will receive sirolimus for 6 months. Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
changes of pulmonary lymphatic perfusion distribution
quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study
Measured: 6 months
changes of coughing score
measured by coughing VAS score (0-10) at baseline, 3 months and the end of study
Measured: 6 months
Secondary outcomes (5)
changes of pulmonary function (FEV1, FVC)
measured by spirometry at baseline, 3 months and the end of study
Measured: 6 months
changes of six minutes walking distance
measured by six minutes walking test at baseline, 3 months and the end of study
Measured: 6 months
change of health-related quality of life
measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study
Measured: 6 months
changes of breathlessness score
measured by Borg scale (0-10) at baseline, 3 months and the end of study
Measured: 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
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2019
- Study start6 May 2019 (estimated)
- Primary completion5 May 2020 (estimated)
- Study completion5 May 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Plastic BronchitisStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- North-China Pharmaceutical Company, China
- Responsible party
- Sponsor
- Investigators
- Kai-Feng Xu, MD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03942926
- Sponsor's protocol ID
- PB1
- Status last verified
- May 2019
- First submitted
- 5 May 2019
- Last updated
- 10 May 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The protocol and clinical study report will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety of Sirolimus for Plastic Bronchitis：a Pilot Study
- ClinicalTrials.gov ID
- NCT03942926
- Acronym / sponsor ID
- PB1
- Conditions
- Plastic Bronchitis
- Keywords
- plastic bronchitis, sirolimus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Collaborators
- North-China Pharmaceutical Company, China
- Study start
- 6 May 2019 (estimated)
- Primary completion
- 5 May 2020 (estimated)
- Study completion
- 5 May 2020 (estimated)
- First posted
- 8 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Ten lymphatic bronchitis patients are planned to be recruited for the study. Sirolimus will be administered for 6 months.
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Experimental | Drug: SirolimusPatients in sirolimus group will receive sirolimus for 6 months. |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- plastic bronchitis
- pulmonary lymphatic perfusion syndrome demonstrated on 68Ga-NEB positron emission tomography (PET)
Key exclusion criteria (3 listed)
- Pregnancy and breastfeeding
- Severe cardiovascular, hepatic and renal dysfunction
- allergy to sirolimus or 68Ga-NEB
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: rapamycinPatients will receive sirolimus for 6 months. | Drug | Sirolimus |
Primary outcome
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
5 secondary outcomes
changes of pulmonary function (FEV1, FVC)
Time frame: 6 months
changes of six minutes walking distance
Time frame: 6 months
change of health-related quality of life
Time frame: 6 months
changes of breathlessness score
Time frame: 6 months
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2019.
- Individual participant dataSharing planned: Yes. The protocol and clinical study report will be shared.
Related trials for Plastic Bronchitis
RecruitingObservational study
European Registry Study on Lymphatic Interventions
Lymphatic Disorders, Plastic Bronchitis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kai-Feng Xu, MD principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Plastic Bronchitis, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Kai-Feng Xu (010-69155039, xukf@pumch.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
changes of pulmonary lymphatic perfusion distribution (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2019.