Glycogen Storage Disease Type IINCT05017402
Higher Dose of Alglucosidase Alpha for Pompe Disease
An observational study of Glycogen Storage Disease Type II, run by Taipei Veterans General Hospital, Taiwan.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 0 to 60 years
- Conditions the study is for
- Glycogen Storage Disease Type II
- What is being tested?
- Alglucosidase Alfa
- How many people
- 36 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2021to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The observational population included patients of Pompe disease who were diagnosed and treated in our series.
Who may be able to join
- Diagnosis of Pompe disease
- Currently receiving ERT with regular clinic visits
- Agree to sign informed consent
Who cannot join
- Lost follow-up for clinical visits
- Allergy to Myozyme
- Refuse to sign informed consent
About this study
Official title: 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.
- Conditions
- Glycogen Storage Disease Type II
- Being tested
- Alglucosidase Alfa
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alglucosidase AlfaDrug
Standard dose: 20-25 mg/kg every other week; Higher dose: >25 mg/kg every other week.
What is being measured
The primary outcome is the main question the study is designed to answer.
Forced vital capacity
Pulmonary function test (Units: percentage of predictive value)
Measured: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Peak expiratory flow
Pulmonary function test (Units: percentage of predictive value)
Measured: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Polysomnography
Comprehensive test used to diagnose sleep disorders.
Measured: For patient older than 6-month-old, first test before study, then every six months, up to 2-years.
Secondary outcomes (7)
uGLC4
Urine glucose tetrasaccharide (Units: mmol/mol Creatinine).
Measured: uGLC4 will be tested before the treatment, then every three months, up to 2-years.
CK
Blood creatine kinase (Units: units per liter, U/L).
Measured: CK will be tested before the treatment, then every three months, up to 2-years.
AST
Blood aspartate aminotransferase (Units: units per liter, U/L).
Measured: AST will be tested before the treatment, then every three months, up to 2-years.
ALT
Blood alanine aminotransferase (Units: units per liter, U/L).
Measured: ALT will be tested before the treatment, then every three months, up to 2-years.
Body weight
Change of body weight (Unit: kilogram, kg)
Measured: The body weight will be monitored before the treatment, then every two weeks, up to 2-years.
Body height
Change of body height (Units: centimeter, cm)
Measured: The body height will be monitored before the treatment, then every two weeks, up to 2-years.
Antibody titers
Alglucosidase alfa IgG antibody titer
Measured: First test will be one month later after first ERT, then every six months, up to 2-years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2021
- Study start1 Sep 2021 (estimated)
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IIStudies of the same condition, open and under way.
- More trials run by Taipei Veterans General Hospital, TaiwanThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Taipei Veterans General Hospital, Taiwan
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05017402
- Sponsor's protocol ID
- 2021-02-019A
- Status last verified
- Aug 2021
- First submitted
- 5 Aug 2021
- Last updated
- 23 Aug 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study
- ClinicalTrials.gov ID
- NCT05017402
- Acronym / sponsor ID
- 2021-02-019A
- Conditions
- Glycogen Storage Disease Type II
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Taipei Veterans General Hospital, TaiwanOther government
- Study start
- 1 Sep 2021 (estimated)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 23 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Other
Who may qualify
- Ages
- 0 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The observational population included patients of Pompe disease who were diagnosed and treated in our series.
Key inclusion criteria (3 listed)
- Diagnosis of Pompe disease
- Currently receiving ERT with regular clinic visits
- Agree to sign informed consent
Key exclusion criteria (3 listed)
- Lost follow-up for clinical visits
- Allergy to Myozyme
- Refuse to sign informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Alglucosidase AlfaStandard dose: 20-25 mg/kg every other week; Higher dose: >25 mg/kg every other week. | Drug | – |
Primary outcome
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)
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)
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.
7 secondary outcomes
uGLC4
Time frame: 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.
AST
Time frame: 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.
Body weight
Time frame: 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.
Antibody titers
Time frame: First test will be one month later after first ERT, then every six months, up to 2-years.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type II, run by Taipei Veterans General Hospital, Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 23 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Forced vital capacity (time frame: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Aug 2021.