Registry Study on Cystic Fibrosis in Chinese Children

Cystic Fibrosis Clinical Trial

Official title:

Registry Study on Cystic Fibrosis in Chinese Children-a Multicenter, Prospective Cohort Study

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02753374
• Other study ID #: BCHlung004
• Status: Not yet recruiting
• Phase: N/A
• First received: April 26, 2016
• Last updated: April 27, 2016
• Start date: May 2016
• Est. completion date: July 2030

Study information

• Verified date: April 2016
• Source: Beijing Children's Hospital
• Contact: Baoping Xu, PhD
• Phone: 861059616308
• Email: xubaopingbch[@]163.com
• Is FDA regulated: No
• Health authority: China: Ministry of Science and TechnologyChina: Ethics Committee
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 100
• Est. completion date: July 2030
• Est. primary completion date: May 2030
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 18 Years

Eligibility

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.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
n/a

Sponsors (7)

Lead Sponsor	Collaborator
Beijing Children's Hospital	Capital Institute of Pediatrics, China, First Affiliated Hospital of Guangxi Medical University, Shanghai Children's Medical Center, Shengjing Hospital, Shenzhen Children's Hospital, The First Affiliated Hospital of Xiamen University

References & Publications (6)

Al-Mahroos F. Cystic fibrosis in bahrain incidence, phenotype, and outcome. J Trop Pediatr. 1998 Feb;44(1):35-9. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Nazer HM. Early diagnosis of cystic fibrosis in Jordanian children. J Trop Pediatr. 1992 Jun;38(3):113-5. — View Citation

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. — View Citation

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	ten years	No