Registry Study on Respiratory Monogenic Diseases in Chinese Children

Monogenic Diseases Clinical Trial

Official title:

Beijing Children's Hospital

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03528525
• Other study ID #: BCHlung 010
• Status: Recruiting
• Start date: January 1, 2017
• Est. completion date: December 30, 2030

Study information

• Verified date: May 2018
• Source: Beijing Children's Hospital
• Contact: Kunling Shen, MD,PhD
• Phone: 861059616308
• Email: kunlingshen1717[@]163.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Description:

All new cases of respiratory monogenic diseases was confirmed or diagnosed at each center from the beginning of the study are made the investigation of the clinical manifestations and specific tests by the standard diagnostic process.Then all the patients' following clinical data will be followed for 5 years: clinical manifestations, lung function, chest imaging (once per year), gene test, quality of life and other indicators.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: December 30, 2030
• Est. primary completion date: January 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 18 Years

Eligibility

Inclusion Criteria:

1. Age: 0-18 years, male and female.

2. Any organ system symptoms consistent with the diagnostic points of respiratory monogenic diseases.

3. Agree to retain specimens related to disease research and agree to store the specimens in the sample bank.

4. willing and able to cooperate with long-term follow-up.

5. 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.

Exclusion Criteria:

1. It is unable to provide complete medical records or the current condition can not accept the diagnosis process

2. She or he cannot agree to participate in the study.

Related Conditions & MeSH terms

• Monogenic Diseases

Locations

Country	Name	City	State
China	Beijing Children's hospital	Beijing	Beijing

Sponsors (9)

Lead Sponsor

Collaborator

Kunling Shen

Capital Institute of Pediatrics, China, Children's Hospital of Chongqing Medical University, Guangzhou Women and Children's Medical Center, Shengjing Hospital, The Children's Hospital, Zhejiang University School Of Medicine, The first clinical college of Guangzhou medical university, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Yuying Children's Hospital of Wenzhou Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in lung function on the spirometry	forced expiratory volume at one second (FEV1) in Liter	1 year later