Higher Dose of Alglucosidase Alpha for Pompe Disease

August 17, 2021 updated by: Taipei Veterans General Hospital, Taiwan

Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

36

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 60 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The observational population included patients of Pompe disease who were diagnosed and treated in our series.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Forced vital capacity
Time Frame: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Pulmonary function test (Units: percentage of predictive value)
For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Peak expiratory flow
Time Frame: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Pulmonary function test (Units: percentage of predictive value)
For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Polysomnography
Time Frame: For patient older than 6-month-old, first test before study, then every six months, up to 2-years.
Comprehensive test used to diagnose sleep disorders.
For patient older than 6-month-old, first test before study, then every six months, up to 2-years.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
uGLC4
Time Frame: uGLC4 will be tested before the treatment, then every three months, up to 2-years.
Urine glucose tetrasaccharide (Units: mmol/mol Creatinine).
uGLC4 will be tested before the treatment, then every three months, up to 2-years.
CK
Time Frame: CK will be tested before the treatment, then every three months, up to 2-years.
Blood creatine kinase (Units: units per liter, U/L).
CK will be tested before the treatment, then every three months, up to 2-years.
AST
Time Frame: AST will be tested before the treatment, then every three months, up to 2-years.
Blood aspartate aminotransferase (Units: units per liter, U/L).
AST will be tested before the treatment, then every three months, up to 2-years.
ALT
Time Frame: ALT will be tested before the treatment, then every three months, up to 2-years.
Blood alanine aminotransferase (Units: units per liter, U/L).
ALT will be tested before the treatment, then every three months, up to 2-years.
Body weight
Time Frame: The body weight will be monitored before the treatment, then every two weeks, up to 2-years.
Change of body weight (Unit: kilogram, kg)
The body weight will be monitored before the treatment, then every two weeks, up to 2-years.
Body height
Time Frame: The body height will be monitored before the treatment, then every two weeks, up to 2-years.
Change of body height (Units: centimeter, cm)
The body height will be monitored before the treatment, then every two weeks, up to 2-years.
Antibody titers
Time Frame: First test will be one month later after first ERT, then every six months, up to 2-years.
Alglucosidase alfa IgG antibody titer
First test will be one month later after first ERT, then every six months, up to 2-years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Taipei Veterans General Hospital, Taiwan

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

September 1, 2021

Primary Completion (Anticipated)

December 31, 2023

Study Completion (Anticipated)

December 31, 2026

Study Registration Dates

First Submitted

August 5, 2021

First Submitted That Met QC Criteria

August 17, 2021

First Posted (Actual)

August 23, 2021

Study Record Updates

Last Update Posted (Actual)

August 23, 2021

Last Update Submitted That Met QC Criteria

August 17, 2021

Last Verified

August 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2021-02-019A

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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