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Dry Blood Spot Screening Test for Neonatal Cholestasis Patients (DBS-SCReNC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03667534
Recruitment Status : Enrolling by invitation
First Posted : September 12, 2018
Last Update Posted : September 14, 2018
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital

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 aim 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. In phase I study investigator will enroll cholestasis patients at National Taiwan University Children Hospital.Investigator will collect patients' blood on dry blood spot and measure the bilirubin and bile acid using tandem mass spectrometry. The data acquire from tandem mass spectrometry will be compared with patients serum bilirubin and bile acid level to evaluate the accuracy. In phase II study, investigator will enroll the cholestasis patients and healthy newborn without cholestasis. Investigator will review their newborn screening at 3 days old. Newborn screening dry blood spot will be examined for the bilirubin and bile acid level and compared with healthy control without cholestasis.

The findings will aid early diagnosis in the patients and hence improve the survival.


Condition or disease Intervention/treatment
Cholestasis in Newborn Infant Biliary Atresia Diagnostic Test: Dry blood spot screening

Detailed Description:

Aims:

  1. Developed a screening test for neonatal cholestasis using dry blood spot
  2. Improve patient survival by early diagnosis of treatable neonatal cholestasis disease such as biliary atresia and inborn error of bile acid metabolism.

Phase I - Validation of tandem mass for quantification of bilirubin and bile acids Pediatric children with cholestasis diagnose at National Taiwan University Hospital during admission or out-patient clinic will be collected for the Phase I study. 5ml of blood will be collected for measurement of bilirubin and bile acids during routine follow-up at National Taiwan University Hospital outpatient clinic, no extra blood drawn process will be performed.Serum bilirubin and bile acid level will be served as reference and compared with DBS method at different time points. 20 patients will be collected to validate the correlation of serum bilirubin and bile acids quantity with tandem mass method. Another 20 patients samples will be collected and the DBS will be protected from light using light blocking enveloped.

Phase II study In the pilot study, investigator aim to develop dry blood spot method for the screening of neonatal cholestasis. Children with neonatal cholestasis disease diagnosed at Department of Pediatrics, National Taiwan University Children's Hospital from 2007-2018 will be recruited retrospectively from chart review. Patients with indirect type hyperbilirubinemia and healthy neonate will be enrolled as control. Investigator will analyze neonatal cholestasis and control patient dry blood spot screening test after birth to quantified total and direct bilirubin level using tandem mass spectrometry. ROC curve analysis will be conducted on bilirubin quantity to find the best cutoff to screen for neonatal cholestasis. The sample size of case and control study is 40 cases and 40 controls.


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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Dry Blood Spot Screening Test for Neonatal Cholestasis Patients
Actual Study Start Date : September 9, 2018
Estimated Primary Completion Date : April 9, 2019
Estimated Study Completion Date : September 9, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rashes


Intervention Details:
  • Diagnostic Test: Dry blood spot screening
    Dry blood spot measurement of biomarker for screening of cholestasis patients


Primary Outcome Measures :
  1. Cholestasis [ Time Frame: on the time of enrollment ]
    Direct bilirubin > 1.0mg/dl or Direct bilirubin to total bilirubin ratio > 0.2


Biospecimen Retention:   Samples Without DNA
Blood serum sample and urine sample


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.
Criteria

Inclusion Criteria:

  • Patients with jaundice
  • Healthy volunteers

Exclusion Criteria:

  • Subjects who do not agree with study protocol.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03667534


Locations
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Taiwan
National Taiwan University Hospital
Taipei, Taiwan, 100
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
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Principal Investigator: Mei-Hwei Chang, MD National Taiwan University Hospital

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Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT03667534     History of Changes
Other Study ID Numbers: 201804007RIND
First Posted: September 12, 2018    Key Record Dates
Last Update Posted: September 14, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: No sharing plan

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by National Taiwan University Hospital:
cholestasis
new born screening
dry blood spot
biliary atresia

Additional relevant MeSH terms:
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Cholestasis
Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities