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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03869515
Other study ID # CHFudanU-02
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date March 25, 2019
Est. completion date December 30, 2025

Study information

Verified date March 2024
Source Children's Hospital of Fudan University
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Recruitment of a carefully characterized cohort of chILD patients, to generate a database and biobank via collecting data on chILD in China. Importantly, compatibility with ongoing United States and Europe chILD data base developments will be factored in.


Description:

Children Interstitial lung disease (chILD) is a heterogeneous group of rare respiratory disorders of known and unknown etiologies that are mostly chronic and associated with high morbidity and mortality. ILD are characterized by inflammatory and fibrotic changes of the lung parenchyma structure that typically result in the presence of diffuse infiltrates on lung imaging, and abnormal pulmonary function tests with evidence of a restrictive ventilatory defect and/or impaired gas exchange. Genetic factors are important contributors to chILD. Genetic variations have been mainly described in genes encoding (or interacting with) the surfactant proteins (SP): SP-C (SFTPC) and the ATP-binding cassette-family A-member 3 (ABCA3) (ABCA3), and less frequently in the genes encoding NKX homeobox 2 (NKX2)-1 (NKX2-1), SP-B (SFTPB), SP-A (SFTPA) ,MARS and other genes. To investigate genetic defects and clinical features of chILD in China, wide recruitment and interdisciplinary critical peer review of all diagnoses from discharge diagnosis coding system of Children's Hospital of Fudan University will be included. Each case will be given a diagnosis independently; if no firm diagnosis is possible, the investigators will review the case periodically as new information becomes available. During the first year of the study, clinicians´ decisions according to clinical practice and outcomes will be independently monitored and assessed. The investigators will systematically optimize and clarify the relative weight of a large spectrum of single and composite clinical outcomes, sequential limited chest CT (to minimise radiation exposure), lung function testing, histopathological categorization of lung biopsies, serum markers and genetic tests. Variability, reproducibility and the effects of training on reading images will be investigated. This project will analyse in detail treatment and outcomes within and between subjects using data collected. Analysis of the collected data will support the definition of trial protocols planned in the future.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 271
Est. completion date December 30, 2025
Est. primary completion date December 30, 2022
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group N/A to 18 Years
Eligibility Inclusion Criteria: - The chILD syndrome exists when a child with DLD has had the common causes of DLD excluded as the primary diagnosis and has at least three of the following four criteria: (1) respiratory symptoms (e.g., cough, rapid and/or difficult breathing, or exercise intolerance);(2) respiratory signs (e.g., resting tachypnea, adventitious sounds, retractions, digital clubbing, failure to thrive, or respiratory fail- ure); (3) hypoxemia; and (4) diffuse abnormalities on CXR or a CT scan. Exclusion Criteria: - These include cystic fibrosis, congenital or acquired immunodeficiency, congenital heart disease, bronchopulmonary dysplasia, pulmonary infection, primary ciliary dyskinesia presenting with newborn respiratory distress and recurrent aspiration.

Study Design


Related Conditions & MeSH terms


Intervention

Other:
No intervention
It's an observational study, so no intervention will be carried out.

Locations

Country Name City State
China Children's hospital of Fudan University Shanghai Shanghai

Sponsors (1)

Lead Sponsor Collaborator
Children's Hospital of Fudan University

Country where clinical trial is conducted

China, 

References & Publications (5)

Bromley S, Vizcaya D. Pulmonary hypertension in childhood interstitial lung disease: A systematic review of the literature. Pediatr Pulmonol. 2017 May;52(5):689-698. doi: 10.1002/ppul.23632. Epub 2016 Oct 23. — View Citation

Eldridge WB, Zhang Q, Faro A, Sweet SC, Eghtesady P, Hamvas A, Cole FS, Wambach JA. Outcomes of Lung Transplantation for Infants and Children with Genetic Disorders of Surfactant Metabolism. J Pediatr. 2017 May;184:157-164.e2. doi: 10.1016/j.jpeds.2017.01.017. Epub 2017 Feb 16. — View Citation

Hime NJ, Zurynski Y, Fitzgerald D, Selvadurai H, Phu A, Deverell M, Elliott EJ, Jaffe A. Childhood interstitial lung disease: A systematic review. Pediatr Pulmonol. 2015 Dec;50(12):1383-92. doi: 10.1002/ppul.23183. Epub 2015 Apr 30. — View Citation

Kurland G, Deterding RR, Hagood JS, Young LR, Brody AS, Castile RG, Dell S, Fan LL, Hamvas A, Hilman BC, Langston C, Nogee LM, Redding GJ; American Thoracic Society Committee on Childhood Interstitial Lung Disease (chILD) and the chILD Research Network. A — View Citation

Thacker PG, Vargas SO, Fishman MP, Casey AM, Lee EY. Current Update on Interstitial Lung Disease of Infancy: New Classification System, Diagnostic Evaluation, Imaging Algorithms, Imaging Findings, and Prognosis. Radiol Clin North Am. 2016 Nov;54(6):1065-1076. doi: 10.1016/j.rcl.2016.05.012. Epub 2016 Aug 12. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Diagnosed with specific cause for chILD (yes/no) Specific causes for chILD based on the 2013 Official American Thoracic Society Clinical Practice Guideline: classification, evaluation, and management of childhood interstitial lung disease in infancy 6 years
Secondary Having pathogenic gene mutations (yes/no) Genetic variations have been mainly described in genes encoding (or interacting with) the surfactant proteins (SP): SP-C (SFTPC) and the ATP-binding cassette-family A-member 3 (ABCA3) (ABCA3), and less frequently in the genes encoding NKX homeobox 2 (NKX2)-1 (NKX2-1), SP-B (SFTPB), SP-A (SFTPA) ,MARS and other genes. 6 years
Secondary Hypoxemia (yes/no) Change of PO2 in arterial blood gases from baseline when diagnosed with chILD 6 years
Secondary Deterioration of pulmonary imaging (yes/no) Change of clinical judgment on pulmonary imaging from baseline if X-ray or CT were done 6 years
Secondary Change from baseline in lung function on the spirometry forced expiratory Volume at one second (FEV1) in Liter 6 years
Secondary Abnormal autoantibody at baseline when diagnosed with chILD (yes/ no) 6 years
Secondary Pathological change of lung biopsy (yes/no) Clinical judgment on histopathological categorization of lung biopsy when diagnosed with chILD 6 years
Secondary Deterioration of pulmonary arterial hypertension (yes/no) Change of pulmonary artery pressure from baseline in echocardiagraphy 6 years
Secondary Change of BALF(bronchoalveolar lavage fluid) (yes/no) Cytology analysis on BALF at the baseline when diagnosed with chILD 6 years
Secondary Survival (yes/no) 1 years
Secondary Survival (yes/no) 2 years
Secondary Survival Five-year 5 years
Secondary Abnormal thyroid hormone at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Abnormal myocardial zymogram at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Abnormal serum immunoglobulin at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Abnormal serum creatinine at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Abnormal blood urea nitrogen at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Abnormal alanine transferase at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Abnormal allergen at baseline when diagnosed with chILD (yes/no) 6 years
Secondary Recurrent hospitalization yes:Hospitalization more than twice per year after diagnosed with chILD; no:Hospitalization less than three times per year after diagnosed with chILD; 1 year after diagnosis
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