Dry Blood Spot Screening Test for Neonatal Cholestasis Patients

NCT03667534 | Unknown

• 1 other identifier: 201804007RIND
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

Cholestatic jaundice with multitude of causes affects approximately 1 in every 2,500 infants. Of the many conditions that cause neonatal cholestasis, the most commonly identifiable are biliary atresia (BA) (25%-35%). The incidence in Taiwan was 1.2 to 2.0 per 10,000 live birth. Prognosis and survival are improved if bile drainage is restored by a Kasai portoenterostomy. Stool color card was introduced to Taiwan in 2002 and national screening program was started in 2004. The rate of age at Kasai operation \<60 days improved from 49.4% before year 2002 to 65.7% after introducing nationwide stool color card screening in Taiwan. There was a great improvement in early diagnosis for biliary atresia after stool color card screening and there were still many researches tried to improve the timing of diagnosis. A prospective cohort observational study on neonates under 27 days old at London showed that serum conjugated bilirubin \> 18 micromol/l in plasma measured at 6-10 days is a reliable marker for neonatal cholestasis liver disease with sensitivity 100%, specificity 99.59% (95% CI 99.5-99.67), PPV 10.3% (95% CI 4.50-16.0). Our study aims to developed a screening test for neonatal cholestasis using dry blood spot to improve patient survival by early diagnosis of treatable neonatal cholestasis disease, such as biliary atresia and inborn error of bile acid metabolism. The diseases markers for neonatal cholestasis we aims including basic clinical blood test examination profiles, inflammatory markers, fibrosis markers, immune profiles. In phase I study we will enroll cholestasis patients at National Taiwan University Children Hospital. We will collect patients' blood on dry blood spot and measure the disease markers for cholestasis disease. In phase II study, we will enroll the cholestasis patients and healthy newborn without cholestasis. We will review their newborn screening at 3 days old. Newborn screening dry blood spot will be examined for the disease markers and compared with healthy control without cholestasis. ROC curve analysis will be performed to find the best cut-off to screen for neonatal cholestasis patients. The findings will aid early diagnosis in the patients and hence improve the survival.

Conditions

Cholestasis in Newborn Infant; Biliary Atresia

Keywords

cholestasis; new born screening; dry blood spot; biliary atresia

Outcome Measures

Primary Outcomes (1)

• Cholestasis

  Direct bilirubin \> 1.0mg/dl or Direct bilirubin to total bilirubin ratio \> 0.2

  on the time of enrollment

Interventions

Dry blood spot screening DIAGNOSTIC_TEST

Dry blood spot measurement of biomarker for screening of cholestasis patients

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with jaundice and healthy volunteers are included for testing the efficacy and accuracy of the screening test.

You may qualify if:

• Patients with jaundice
• Healthy volunteers

You may not qualify if:

• Subjects who do not agree with study protocol.

Sponsors & Collaborators

• National Taiwan University Hospital: lead

Study Sites (1)

National Taiwan University Hospital

Taipei, 100, Taiwan

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood serum sample and urine sample

MeSH Terms

Conditions

Biliary Atresia; Cholestasis

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Digestive System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Mei-Hwei Chang, MD

  National Taiwan University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2018
• First Posted: September 12, 2018
• Study Start: September 9, 2018
• Primary Completion: June 8, 2021
• Study Completion: September 9, 2021
• Last Updated: May 14, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1)