Pompe DiseaseNCT00713245
A Long Term Follow up Study in Late-onset Pompe Disease
An observational study of Pompe Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
This study is to observe the progression of disease in late-onset Pompe disease Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Pompe Disease
- How many people
- 70 participants
- Where
- 1 siteTaiwan
- Study dates
- May 2008to Dec 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: individuals with acid alpha glucosidase deficiency but without obvious symptoms
Who may be able to join
- individuals with acid alpha glucosidase deficiency without obvious symptoms
- parents signed inform consent
Who cannot join
- individuals with acid alpha glucosidase deficiency presenting symptoms
Trial locations and contacts
1 site in 1 country.
Whu-Liang Hwu, MD, PhD contact
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Hospital
Taipei
Yin-Hsiu Chien, MD, 886-2-23123456 ext. 71937
Whu-Liang Hwu, MD, PhD
About this study
This study is to observe the progression of disease in late-onset Pompe disease
- Conditions
- Pompe Disease
- Keywords
- Pompe disease, late onset
- Medical subject headings
- Glycogen Storage Disease Type II
What is being measured
The primary outcome is the main question the study is designed to answer.
all muscle function
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2008 (estimated)
- Study startMay 2008
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Whu-Liang Hwu, MD, PhD, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00713245
- Sponsor's protocol ID
- 200804012R
- Status last verified
- Dec 2013
- First submitted
- 9 Jul 2008
- Last updated
- 4 Dec 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long Term Follow up Study in Late-onset Pompe Disease
- ClinicalTrials.gov ID
- NCT00713245
- Acronym / sponsor ID
- 200804012R
- Conditions
- Pompe Disease
- Keywords
- Pompe disease, late onset
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- May 2008
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 11 Jul 2008 (estimated)
- 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 to observe the progression of disease in late-onset Pompe disease
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- individuals with acid alpha glucosidase deficiency but without obvious symptoms
Key inclusion criteria (2 listed)
- individuals with acid alpha glucosidase deficiency without obvious symptoms
- parents signed inform consent
Key exclusion criteria (1 listed)
- individuals with acid alpha glucosidase deficiency presenting symptoms
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
all muscle function
Time frame: 10 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2013.
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MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
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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. This study is not recruiting at the moment.
Whu-Liang Hwu, MD, PhD contact
Whu-Liang Hwu, MD, PhD principal investigator
National Taiwan University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pompe Disease, run by National Taiwan University Hospital at 1 site in 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 2 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 4 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Whu-Liang Hwu, MD, PhD (hwuwlntu@ntu.edu.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
all muscle function (time frame: 10 years). 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 4 Dec 2013.