Newborn Screening for Adrenoleukodystrophy | NCT02952482

Studies
Newborn Screening for Adrenoleukodystrophy

NCT02952482

Sponsor

National Taiwan University Hospital

Status

Completed

Last Update Posted

Jul 12, 2018Actual

Enrollment

45,796Actual

Phase

Not provided

Conditions

Adrenoleukodystrophy

Interventions

newborns testing for ALD

Countries

Taiwan

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2017

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2016

Primary Completion Date

Aug 2017Actual

Completion Date

Aug 2017Actual

First Submitted Date

Oct 31, 2016

First Submission Date that Met QC Criteria

Oct 31, 2016

First Posted Date

Nov 2, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 10, 2018

Last Update Posted Date

Jul 12, 2018Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

To test if the routine newborn screening dried blood spots can be used to test if elevation of C26:0 lysophosphatidylcholine (C26:0-lyso-PC), a status indicating adrenoleukodystrophy (ALD)

Detailed Description

Parents of newborns will be invited to test if their newborns are affected with ALD. The routine newborn screening dried blood spots sample will be used to test the concentration of C26:0-lyso-PC . If positive of a screening test, further confirmation tests including physical examination and other methodology for ALD confirmation will be provided. Genetic counseling and treatment option will be provided, too.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

45,796Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
newborns testing for ALD		newborns testing for ALD	Procedure: newborns testing for ALD

Interventions

Name	Type	Description	Arm Group Labels	Other Names
newborns testing for ALD	Procedure	Routine newborn screening dried blood spots sample is used to test if elevation of C26:0-lyso-PC	newborns testing for ALD

Measure	Description	Time Frame
numbers of newborn with ALD		12 months

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Babies born in Taiwan receive regular newborn screening suggested by Ministry of Heath and Welfare.
Parents or Legal Guardian sign in the informed consent form.

Exclusion Criteria:

Parents or Legal Guardian do not agree to sign in the informed consent form.

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Days

Maximum Age

3 Months

Standard Ages

Child

Study Population

newborns whose parents agree to be tested

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
National Taiwan University Hospital		Taipei		100	Taiwan

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000326	Adrenoleukodystrophy

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D000074063	ATP Binding Cassette Transporter, Subfamily D, Member 1

Ancestor Terms

ID	Term
D000074061	ATP Binding Cassette Transporter, Subfamily D
D018528	ATP-Binding Cassette Transporters
D008562	Membrane Glycoproteins
D006023	Glycoproteins

Browse Leaves

Not provided

Browse Branches

Not provided