The Efficacy and Safety of Sirolimus for Plastic Bronchitis

NCT ID: NCT03942926

Last Updated: 2019-05-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-05-06

Study Completion Date

2020-05-05

Brief Summary

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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

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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

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Plastic Bronchitis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Ten lymphatic bronchitis patients are planned to be recruited for the study. Sirolimus will be administered for 6 months.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Sirolimus

Patients in sirolimus group will receive sirolimus for 6 months.

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Patients will receive sirolimus for 6 months.

Interventions

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Sirolimus

Patients will receive sirolimus for 6 months.

Intervention Type DRUG

Other Intervention Names

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rapamycin

Eligibility Criteria

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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
Minimum Eligible Age

13 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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North-China Pharmaceutical Company, China

UNKNOWN

Sponsor Role collaborator

Peking Union Medical College Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kai-Feng Xu, MD

Role: PRINCIPAL_INVESTIGATOR

Peking Union Medical College Hospital

Locations

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Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Kai-Feng Xu

Role: CONTACT

010-69155039

Facility Contacts

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Kai-Feng Xu, M.D.

Role: primary

86-10-69155039

Xinlun Tian, M.D.

Role: backup

86-10-69155039

Other Identifiers

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PB1

Identifier Type: -

Identifier Source: org_study_id

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