Newborn Screening for Adrenoleukodystrophy

• Sponsor: National Taiwan University Hospital (Other)
• Overall Status: Completed
• CT.gov ID: NCT02952482
• Collaborator: (none)
• Enrollment: 45,796
• Location: 1
• Duration (Months): 9
• Patients Per Site Per Month: 5105.9

Study Details

Study Description

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)

Condition or Disease	Intervention/Treatment	Phase
Adrenoleukodystrophy	Procedure: newborns testing for 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.

Study Design

• Study Type: Observational
• Actual Enrollment: 45796 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Newborn Screening for Adrenoleukodystrophy
• Study Start Date: Nov 1, 2016
• Actual Primary Completion Date: Aug 1, 2017
• Actual Study Completion Date: Aug 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
newborns testing for ALD newborns testing for ALD	Procedure: newborns testing for ALD; Routine newborn screening dried blood spots sample is used to test if elevation of C26:0-lyso-PC

Outcome Measures

Primary Outcome Measures

1. numbers of newborn with ALD [12 months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Days to 3 Months
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	National Taiwan University Hospital	Taipei		Taiwan	100

Sponsors and Collaborators

• National Taiwan University Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: National Taiwan University Hospital
• ClinicalTrials.gov Identifier: NCT02952482
• Other Study ID Numbers: 201603025RIND
• First Posted: Nov 2, 2016
• Last Update Posted: Jul 12, 2018
• Last Verified: Jul 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by National Taiwan University Hospital: adrenoleukodystrophy; C26:0-lyso-PC
• Additional relevant MeSH terms: Adrenoleukodystrophy

Study Results

No Results Posted as of Jul 12, 2018