A Long-term Study for the Outcome of Pompe Disease

NCT ID: NCT02399748

Last Updated: 2017-07-18

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

RECRUITING

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2007-04-30

Study Completion Date

2026-12-31

Brief Summary

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Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.

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

Eligibility Criteria

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Inclusion Criteria

Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy

Exclusion Criteria

No
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

Principal Investigators

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Yin-Hsiu Chien

Role: PRINCIPAL_INVESTIGATOR

National Taiwan University Hospital

Locations

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National Taiwan University Hospital

Taipei, , Taiwan

Site Status RECRUITING

Countries

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Taiwan

Central Contacts

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Yin-Hsiu Chien

Role: CONTACT

+886223123456 ext. 71937

Facility Contacts

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Yin-Hsiu Chien, MD, PhD

Role: primary

+886223123456 ext. 71937

References

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Hsu YK, Chien YH, Shinn-Forng Peng S, Hwu WL, Lee WT, Lee NC, Po-Yu Huang E, Weng WC. Evaluating brain white matter hyperintensity, IQ scores, and plasma neurofilament light chain concentration in early-treated patients with infantile-onset Pompe disease. Genet Med. 2023 Jan;25(1):27-36. doi: 10.1016/j.gim.2022.10.005. Epub 2022 Nov 18.

Reference Type DERIVED
PMID: 36399131 (View on PubMed)

Other Identifiers

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200703045R

Identifier Type: -

Identifier Source: org_study_id

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