Higher Dose of Alglucosidase Alpha for Pompe Disease

NCT ID: NCT05017402

Last Updated: 2021-08-23

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

NOT_YET_RECRUITING

Total Enrollment

36 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-09-01

Study Completion Date

2026-12-31

Brief Summary

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This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.

Detailed Description

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Conditions

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Glycogen Storage Disease Type II

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Interventions

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Alglucosidase Alfa

Standard dose: 20-25 mg/kg every other week; Higher dose: \>25 mg/kg every other week.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Diagnosis of Pompe disease
2. Currently receiving ERT with regular clinic visits
3. Agree to sign informed consent

Exclusion Criteria

1. Lost follow-up for clinical visits
2. Allergy to Myozyme
3. Refuse to sign informed consent
Minimum Eligible Age

0 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Taipei Veterans General Hospital, Taiwan

OTHER_GOV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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2021-02-019A

Identifier Type: -

Identifier Source: org_study_id

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