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High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics

NCT ID: NCT04981210

Last Updated: 2023-04-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

1500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-08-01

Study Completion Date

2024-07-31

Brief Summary

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Establish of high-risk screening criteria to earlier identify possible childhood LOPD for early treatment and better prognosis. Therefore, validation of the high-risk screening criteria for childhood LOPD will be critical for identifying children of LOPD in Taiwan.

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Detailed Description

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Conditions

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Pompe Disease

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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suspect LOPD

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Children who are \>1 year and \< 18 year of age from pediatric neurology, pediatric orthopedics and pediatric rehabilitation in NTUH

Exclusion Criteria

* Children who have been diagnosed as specific neuromuscular disorders.
Minimum Eligible Age

1 Year

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Taiwan University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Wen-Chin Weng

Taipei, , Taiwan

Site Status RECRUITING

Countries

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Taiwan

Facility Contacts

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Wen-Chin Weng

Role: primary

[email protected]
+886-2-23123456 ext. 71619

Other Identifiers

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202012243RIPC

Identifier Type: -

Identifier Source: org_study_id

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