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

May 12, 2021 updated by: National Taiwan University Hospital

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 method for quantification of disease markers on dry blood spot.

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 venous blood will be collected for measurement of disease markers during routine follow-up at National Taiwan University Hospital outpatient clinic. Dried blood spot (DBS) specimens are collected from patients by applying a whole blood, drawn by lancet from the finger, heel or toe, a provided filter paper. To evaluate the stability of disease markers on DBS, a total 10 blood spots (each blood spout contain 50 - 75 microL of blood) will be applied on the filter paper at day 0. The collected blood spots samples of a single patient at day 0 will be evaluated at the day of DBS collection and followed by day 3, day 7, 6 months and 1 year after sample collection. The purpose of repeat measurement of single patient sample at different time point is to evaluate the possible effect of time effect on the accuracy of the data. 5 blood spots are separated and protected from light with a light blocking envelope to compare the light effect on disease markers degradation (Table 1). Serum disease markers measured with conventional method will be served as reference and compared with DBS samples at different time points. 30 patients will be collected to validate the correlation between serum and DBS disease markers level.

Phase II - Pilot study In the pilot study, we aim to develop dry blood spot method for the screening of neonatal cholestasis. Children with neonatal cholestasis disease previously diagnosed at Department of Pediatrics, National Taiwan University Children's Hospital from 2007 or newly diagnosed children will be recruited retrospectively from chart review. Patients with indirect type hyperbilirubinemia and healthy neonates will be enrolled as control. We will analyze neonatal cholestasis and control patients dry blood spot screening test after birth to quantify disease markers level. 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 20 cases and 20 controls.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Taiwan
      • Taipei, Taiwan, 100
        • National Taiwan University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

Inclusion Criteria:

  • Patients with jaundice
  • Healthy volunteers

Exclusion Criteria:

  • Subjects who do not agree with study protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Cholestasis
Time Frame: on the time of enrollment
Direct bilirubin > 1.0mg/dl or Direct bilirubin to total bilirubin ratio > 0.2
on the time of enrollment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
National Taiwan University Hospital

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Mei-Hwei Chang, MD, National Taiwan University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 9, 2018

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 8, 2021

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 9, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 10, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 10, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 12, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 14, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 12, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 201804007RIND

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

IPD Plan Description

No sharing plan

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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