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

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 1, 2021

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2023

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 5, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 17, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 23, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 23, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 17, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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