Registry Study on Cystic Fibrosis in Chinese Children

NCT02753374 | Not Yet Recruiting DMC

• 1 other identifier: BCHlung004
• Study Type: observational
• Target: 100
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis, Chinese, children

Outcome Measures

Primary Outcomes (1)

• Change from baseline in lung function on the spirometry

  forced expiratory volume at one second (FEV1) in Liter

  ten years

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Beijing Children's Hospital: lead
• Shengjing Hospital: collaborator
• Capital Institute of Pediatrics, China: collaborator
• Shanghai Children's Medical Center: collaborator
• Shenzhen Children's Hospital: collaborator
• The First Affiliated Hospital of Xiamen University: collaborator
• First Affiliated Hospital of Guangxi Medical University: collaborator

Related Publications (6)

• 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. doi: 10.1126/science.2570460.

  PMID: 2570460 BACKGROUND

• 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. doi: 10.1542/peds.113.6.1573.

  PMID: 15173476 BACKGROUND

• 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.

  PMID: 1507302 BACKGROUND

• 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.

  PMID: 9538604 BACKGROUND

• 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. doi: 10.1097/00005176-199705000-00010.

  PMID: 9161949 BACKGROUND

• 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.

  PMID: 26631874 BACKGROUND

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Baoping Xu, PhD

CONTACT

861059616308; xubaopingbch@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief of China National Clinical Research Center for Respiratory Diseases

Study Record Dates

• First Submitted: April 26, 2016
• First Posted: April 27, 2016
• Study Start: May 1, 2016
• Primary Completion (Estimated): May 1, 2030
• Study Completion (Estimated): July 1, 2030
• Last Updated: April 28, 2016

Record last verified: 2016-04