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Newborn Screening for Adrenoleukodystrophy

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ClinicalTrials.gov Identifier: NCT02952482
Recruitment Status : Completed
First Posted : November 2, 2016
Last Update Posted : July 12, 2018
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital

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
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 Type : Observational
Actual Enrollment : 45796 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Newborn Screening for Adrenoleukodystrophy
Study Start Date : November 2016
Actual Primary Completion Date : August 2017
Actual Study Completion Date : August 2017


Group/Cohort 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




Primary Outcome Measures :
  1. numbers of newborn with ALD [ Time Frame: 12 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 3 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
newborns whose parents agree to be tested
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.

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): NCT02952482


Locations
Taiwan
National Taiwan University Hospital
Taipei, Taiwan, 100
Sponsors and Collaborators
National Taiwan University Hospital

Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT02952482     History of Changes
Other Study ID Numbers: 201603025RIND
First Posted: November 2, 2016    Key Record Dates
Last Update Posted: July 12, 2018
Last Verified: July 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by National Taiwan University Hospital:
adrenoleukodystrophy
C26:0-lyso-PC

Additional relevant MeSH terms:
Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases