Registry Study on Cystic Fibrosis in Chinese Children

• ClinicalTrials.gov Identifier: NCT02753374
• Recruitment Status: Not yet recruiting
• First Posted: April 27, 2016
• Last Update Posted: April 28, 2016
• Sponsor: Beijing Children's Hospital
• Collaborators: Shengjing Hospital; Capital Institute of Pediatrics, China; Shanghai Children's Medical Center; Shenzhen Children's Hospital; The First Affiliated Hospital of Xiamen University; First Affiliated Hospital of Guangxi Medical University
• Information provided by (Responsible Party): Kunling Shen, Beijing Children's Hospital

Study Description

Brief Summary:

This study is a multicenter, prospective cohort study of patients diagnosed with cystic fibrosis, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.

Condition or disease
Cystic Fibrosis

Detailed Description:

All new cases of cystic fibrosis which was confirmed or probably diagnosed at each center from the beginning of the study are made the investigation of the clinical manifestations, sweat test and CFTR mutations by the standard diagnostic process.Then all the patients' following clinical data will be followed for 10 years (once per six month): clinical manifestations, lung function, chest imaging (once per year), quality of life and other indicators.

Study Design

• Study Type: Observational
• Estimated Enrollment: 100 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Registry Study on Cystic Fibrosis in Chinese Children-a Multicenter, Prospective Cohort Study
• Study Start Date: May 2016
• Estimated Primary Completion Date: May 2030
• Estimated Study Completion Date: July 2030

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

1. Change from baseline in lung function on the spirometry [ Time Frame: ten years ]
   forced expiratory volume at one second (FEV1) in Liter

Eligibility Criteria

• Ages Eligible for Study: up to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Children who was confirmed diagnosis as cystic fibrosis at the certain hospitals (sponsor and collaborators)

Criteria

Inclusion Criteria:

• Age 0~18 years old
• Any organ system symptoms consistent with CF, such as chronic sinopulmonary disease, gastrointestinal and nutritional abnormalities, obstructive azoospermia or having siblings with CF
• CFTR dysfunction indicated by elevated sweat chloride levels ≥60 mmol/L twice, or one sweat chloride levels ≥40 mmol/L plus presence of two pathogenic CFTR mutations on different alleles
• Probable CF patients with sweat chloride levels among 40~59 mmol/L plus with presence of 0-1 pathogenic CFTR mutation
• Consent to provide the related clinical specimen to the certain hospital
• The guardians of the patients fully understand the purpose of the study, volunteer their children to participate in this study and sign informed consent.

Exclusion Criteria:

• Subject will be excluded if she or he has one of the following:
• It is unable to provide complete medical records or the current condition can not accept the diagnosis process.
• She or he does not agree to participate in the test.

Contacts and Locations

Contacts

Contact: Baoping Xu, PhD; 861059616308; xubaopingbch@163.com

Sponsors and Collaborators

Beijing Children's Hospital

Shengjing Hospital

Capital Institute of Pediatrics, China

Shanghai Children's Medical Center

Shenzhen Children's Hospital

The First Affiliated Hospital of Xiamen University

First Affiliated Hospital of Guangxi Medical University

Investigators

• Principal Investigator: Kunling Shen, MD, PhD; Beijing Children's Hospital of Capital Medical University, China, China National Clinical Research Center for Respiratory Diseases
• Principal Investigator: Baoping Xu, MD, PhD; Beijing Children's Hospital of Capital Medical University, China, China National Clinical Research Center for Respiratory Diseases

More Information

Publications:

Kerem B, Rommens JM, Buchanan JA, Markiewicz D, Cox TK, Chakravarti A, Buchwald M, Tsui LC. Identification of the cystic fibrosis gene: genetic analysis. Science. 1989 Sep 8;245(4922):1073-80.

Comeau AM, Parad RB, Dorkin HL, Dovey M, Gerstle R, Haver K, Lapey A, O'Sullivan BP, Waltz DA, Zwerdling RG, Eaton RB. Population-based newborn screening for genetic disorders when multiple mutation DNA testing is incorporated: a cystic fibrosis newborn screening model demonstrating increased sensitivity but more carrier detections. Pediatrics. 2004 Jun;113(6):1573-81.

Nazer HM. Early diagnosis of cystic fibrosis in Jordanian children. J Trop Pediatr. 1992 Jun;38(3):113-5. doi: 10.1093/tropej/38.3.113.

Al-Mahroos F. Cystic fibrosis in bahrain incidence, phenotype, and outcome. J Trop Pediatr. 1998 Feb;44(1):35-9. doi: 10.1093/tropej/44.1.35.

Yamashiro Y, Shimizu T, Oguchi S, Shioya T, Nagata S, Ohtsuka Y. The estimated incidence of cystic fibrosis in Japan. J Pediatr Gastroenterol Nutr. 1997 May;24(5):544-7.

Brennan ML, Schrijver I. Cystic Fibrosis: A Review of Associated Phenotypes, Use of Molecular Diagnostic Approaches, Genetic Characteristics, Progress, and Dilemmas. J Mol Diagn. 2016 Jan;18(1):3-14. doi: 10.1016/j.jmoldx.2015.06.010. Epub 2015 Nov 26. Review.

• Responsible Party: Kunling Shen, Chief of China National Clinical Research Center for Respiratory Diseases, Beijing Children's Hospital
• ClinicalTrials.gov Identifier: NCT02753374
• Other Study ID Numbers: BCHlung004
• First Posted: April 27, 2016
• Last Update Posted: April 28, 2016
• Last Verified: April 2016

Keywords provided by Kunling Shen, Beijing Children's Hospital:

cystic fibrosis, Chinese, children

Additional relevant MeSH terms:

Cystic Fibrosis; Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases