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NCT01758354 Clinical Trial

Newborn Screening Assay of Pompe's Disease

Pompe Disease Clinical Trial

Official title:
Newborn Screening Assay of Pompe's Disease

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT01758354
Other study ID # 9461700818
Secondary ID
Status Completed
Phase N/A
First received July 9, 2008
Last updated December 26, 2012
Start date September 2005
Est. completion date December 2009

Study information
Verified date November 2012
Source National Taiwan University Hospital
Contact n/a
Is FDA regulated No
Health authority Taiwan: Department of Health
Study type Interventional

Clinical Trial Summary

The purpose of this study is to test the feasibility of a newborn screen assay for Pompe disease

Description:

DBS from newborn will be tested for acid alpha-glucosidase (GAA) activity. Babies with low GAA activity will be confirmed for Pompe disease.

Recruitment information / eligibility
Status Completed
Enrollment 236536
Est. completion date December 2009
Est. primary completion date December 2009
Accepts healthy volunteers Accepts Healthy Volunteers
Gender Both
Age group N/A and older
Eligibility Inclusion Criteria:

- newborns receiving newborn screening in Newborn Screening center of National Taiwan Univeristy Hospital

- parents signed inform consent for this study

Exclusion Criteria:

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Screening

Related Conditions & MeSH terms

Intervention

Other:
• Pompe disease newborn screening
DBS will be tested for acid alpha-glucosidase (GAA)activity. Newborns with low GAA activity will received a confirmatory blood sampling and clinical evaluation for the presence of cardiomyopathy.

Locations
Country Name City State
Taiwan National Taiwan University Hospital Taipei

Sponsors (1)
Lead Sponsor Collaborator
National Taiwan University Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome
Type Measure Description Time frame Safety issue
Primary detect patients with infantile onset Pompe disease 3 months No
See also
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Completed NCT01380743 - Drug-drug Interaction Study Phase 2
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Completed NCT02240407 - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease Phase 1
Completed NCT05073783 - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A
Completed NCT01410890 - Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease Phase 4