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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT02704455
Other study ID # BCHlung005
Secondary ID
Status Not yet recruiting
Phase N/A
First received March 3, 2016
Last updated March 5, 2016
Start date May 2016
Est. completion date July 2030

Study information

Verified date March 2016
Source Beijing Children's Hospital
Contact Baoping Xu, MD, 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 primary ciliary dyskinesia, 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 primary ciliary dyskinesia which 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 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: A included patient must be coincident with all the following items:

- Age 0~18 years old

- Any organ system symptoms consistent with PCD and being conform to the clinical diagnostic standard of Katergener syndrome or being coincident with at least two following specific tests:

- Abnormal ciliary beat frequency or movement by the high speed photography microscope

- Abnormal ciliary structure through the electronic microscopy

- The nasal NO decreased significantly

- The target gene mutation found

- The clinical diagnostic criteria of the Katergener syndrome: ? bronchial expansion; ? sinusitis or nasal polyps; ? transposition of viscera and (or) dextrocardia.

- If all the typical clinical manifestations but only 1 specific test with positive results, can also be included in the registration of suspected PCD cases

- 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 cannot agree to participate in the study.

Study Design

Observational Model: Cohort, Time Perspective: Prospective


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 (4)

Hogg C, Bush A. Genotyping in primary ciliary dyskinesia: ready for prime time, or a fringe benefit? Thorax. 2012 May;67(5):377-8. doi: 10.1136/thoraxjnl-2011-201320. Epub 2012 Jan 9. — View Citation

Liu Y, Wang L, Tian X, Xu KF, Xu W, Li X, Yue C, Zhang P, Xiao Y, Zhang X. Characterization of gene mutations and phenotypes of cystic fibrosis in Chinese patients. Respirology. 2015 Feb;20(2):312-8. doi: 10.1111/resp.12452. Epub 2015 Jan 8. — View Citation

Noone PG, Leigh MW, Sannuti A, Minnix SL, Carson JL, Hazucha M, Zariwala MA, Knowles MR. Primary ciliary dyskinesia: diagnostic and phenotypic features. Am J Respir Crit Care Med. 2004 Feb 15;169(4):459-67. Epub 2003 Dec 4. — View Citation

Shapiro AJ, Zariwala MA, Ferkol T, Davis SD, Sagel SD, Dell SD, Rosenfeld M, Olivier KN, Milla C, Daniel SJ, Kimple AJ, Manion M, Knowles MR, Leigh MW; Genetic Disorders of Mucociliary Clearance Consortium. Diagnosis, monitoring, and treatment of primary ciliary dyskinesia: PCD foundation consensus recommendations based on state of the art review. Pediatr Pulmonol. 2016 Feb;51(2):115-32. doi: 10.1002/ppul.23304. Epub 2015 Sep 29. — 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 10 years No
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