The Efficacy and Safety of Sirolimus for Plastic Bronchitis

NCT03942926 | Unknown Phase 2

• 1 other identifier: PB1
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Plastic Bronchitis

Keywords

plastic bronchitis; sirolimus

Outcome Measures

Primary Outcomes (2)

• changes of pulmonary lymphatic perfusion distribution

  quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study

  6 months

• changes of coughing score

  measured by coughing VAS score (0-10) at baseline, 3 months and the end of study

  6 months

Secondary Outcomes (5)

• changes of pulmonary function (FEV1, FVC)

  6 months

• changes of six minutes walking distance

  6 months

• change of health-related quality of life

  6 months

• changes of breathlessness score

  6 months

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  6 months

Study Arms (1)

Sirolimus

EXPERIMENTAL

Patients in sirolimus group will receive sirolimus for 6 months.

Drug: Sirolimus

Interventions

Sirolimus DRUG

Patients will receive sirolimus for 6 months.

Also known as: rapamycin

Sirolimus

Eligibility Criteria

• Age: 13 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• plastic bronchitis
• pulmonary lymphatic perfusion syndrome demonstrated on 68Ga-NEB positron emission tomography (PET)

You may not qualify if:

• Pregnancy and breastfeeding
• Severe cardiovascular, hepatic and renal dysfunction
• allergy to sirolimus or 68Ga-NEB

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead
• North-China Pharmaceutical Company, China: collaborator

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Interventions

Sirolimus

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Kai-Feng Xu, MD

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kai-Feng Xu

CONTACT

010-69155039; xukf@pumch.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Ten lymphatic bronchitis patients are planned to be recruited for the study. Sirolimus will be administered for 6 months.

Study Record Dates

• First Submitted: May 5, 2019
• First Posted: May 8, 2019
• Study Start: May 6, 2019
• Primary Completion: May 5, 2020
• Study Completion: May 5, 2020
• Last Updated: May 10, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, 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

Locations

CN (1)