NCT03528525

Brief Summary

This study is a multicenter, prospective cohort study of patients diagnosed with respiratory monogenic diseases, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 5 years.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P75+ for all trials

Timeline
56mo left

Started Jan 2017

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress67%
Jan 2017Dec 2030

Study Start

First participant enrolled

January 1, 2017

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2018

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

May 6, 2018

Completed
12 days until next milestone

First Posted

Study publicly available on registry

May 18, 2018

Completed
12.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2030

Expected
Last Updated

May 18, 2018

Status Verified

May 1, 2018

Enrollment Period

1 year

First QC Date

May 6, 2018

Last Update Submit

May 6, 2018

Conditions

Keywords

DiseasesChildrenRespiratoryMonogenic diseasesChina

Outcome Measures

Primary Outcomes (1)

  • Change from baseline in lung function on the spirometry

    forced expiratory volume at one second (FEV1) in Liter

    1 year later

Study Arms (1)

Monogenic diseases cases

Eligibility Criteria

Age1 Month - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Children who was confirmed diagnosis as respiratory monogenic diseases at the certain hospitals (sponsor and collaborators)

You may qualify if:

  • Age: 0-18 years, male and female.
  • Any organ system symptoms consistent with the diagnostic points of respiratory monogenic diseases.
  • Agree to retain specimens related to disease research and agree to store the specimens in the sample bank.
  • willing and able to cooperate with long-term follow-up.
  • The guardian of the child has a full understanding of the purpose of the study, a basic understanding of the clinical research program, and voluntarily allows the child to participate in the study and sign an informed consent form.

You may not qualify if:

  • It is unable to provide complete medical records or the current condition can not accept the diagnosis process
  • She or he cannot agree to participate in the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Beijing Children's hospital

Beijing, Beijing Municipality, 100045, China

RECRUITING

MeSH Terms

Conditions

Disease

Condition Hierarchy (Ancestors)

Pathologic ProcessesPathological Conditions, Signs and Symptoms

Central Study Contacts

Kunling Shen, MD,PhD

CONTACT

Baoping Xu, MD,PhD

CONTACT

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Beijing Children's Hospital

Study Record Dates

First Submitted

May 6, 2018

First Posted

May 18, 2018

Study Start

January 1, 2017

Primary Completion

January 1, 2018

Study Completion (Estimated)

December 30, 2030

Last Updated

May 18, 2018

Record last verified: 2018-05

Locations