Cystic FibrosisNCT02753374
Registry Study on Cystic Fibrosis in Chinese Children
An observational study of Cystic Fibrosis, run by Beijing Children's Hospital.
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- May 2016to Jul 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children who was confirmed diagnosis as cystic fibrosis at the certain hospitals (sponsor and collaborators)
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Registry Study on Cystic Fibrosis in Chinese Children-a Multicenter, Prospective Cohort Study
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.
Read the 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.
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, Chinese, children
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in lung function on the spirometry
forced expiratory volume at one second (FEV1) in Liter
Measured: ten years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2016 (estimated)
- Study startMay 2016
- Primary completionMay 2030 (estimated)
- Study completionJul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Children's Hospital
- Sponsor type
- University, hospital or other
- 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
- Responsible party
- Principal investigator, Kunling Shen, Beijing Children's Hospital
- Investigators
- Kunling Shen, MD, PhD, Principal investigator, Beijing Children's Hospital of Capital Medical University, China, China National Clinical Research Center for Respiratory DiseasesBaoping Xu, MD, PhD, Principal investigator, Beijing Children's Hospital of Capital Medical University, China, China National Clinical Research Center for Respiratory Diseases
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02753374
- Sponsor's protocol ID
- BCHlung004
- Status last verified
- Apr 2016
- First submitted
- 26 Apr 2016
- Last updated
- 28 Apr 2016
- Results posted
- No
- Data monitoring committee
- Yes
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. doi: 10.1126/science.2570460.
PubMed 2570460Comeau 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.
PubMed 15173476Nazer 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.
PubMed 1507302Al-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.
PubMed 9538604Yamashiro 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.
PubMed 9161949Brennan 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.
PubMed 26631874
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Registry Study on Cystic Fibrosis in Chinese Children-a Multicenter, Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT02753374
- Acronym / sponsor ID
- BCHlung004
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, Chinese, children
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Beijing Children's HospitalUniversity, hospital or other
- 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
- Study start
- May 2016
- Primary completion
- May 2030 (estimated)
- Study completion
- Jul 2030 (estimated)
- First posted
- 27 Apr 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children who was confirmed diagnosis as cystic fibrosis at the certain hospitals (sponsor and collaborators)
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change from baseline in lung function on the spirometry
Time frame: ten years
forced expiratory volume at one second (FEV1) in Liter
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2016.
- Background: PubMed 2570460Kerem 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.
- Background: PubMed 15173476Comeau 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.
- Background: PubMed 1507302Nazer 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.
- Background: PubMed 9538604Al-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.
- Background: PubMed 9161949Yamashiro 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.
- Background: PubMed 26631874Brennan 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kunling Shen, MD, PhD principal investigator
Beijing Children's Hospital of Capital Medical University, China, China National Clinical Research Center for Respiratory Diseases
Baoping Xu, MD, PhD principal investigator
Beijing Children's Hospital of Capital Medical University, China, China National Clinical Research Center for Respiratory Diseases
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Beijing Children's Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 28 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Baoping Xu, PhD (861059616308, xubaopingbch@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in lung function on the spirometry (time frame: ten years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02753374, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2016.