Pompe DiseaseNCT00713453
Muscle Response to Enzyme Replacement Therapy in 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 study the response of muscle cells from Pompe disease after enzyme replacement therapy Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Pompe Disease
- How many people
- 16 participants
- Where
- 1 siteTaiwan
- Study dates
- Jan 2007to Dec 2019
- 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
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: patients with Pompe disease, prepare or on enzyme replacement therapy
Who may be able to join
- patients with Pompe disease, prepare or on enzyme replacement therapy
- signed informed consent
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Hospital
Taipei
Yin-Hsiu Chien, MD, 886-2-23123456 ext. 71937, chienyh@ntu.edu.tw
Whu-Liang Hwu, MD, PhD
About this study
This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy
- Conditions
- Pompe Disease
- Keywords
- Pompe disease, muscle, enzyme replacement therapy
- 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 morphology changes improvement
Measured: 6 months
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 startJan 2007
- Primary completionDec 2019 (estimated)
- Study completionDec 2019 (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
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, National Taiwan University Hospital, National Taiwan University Hospital
- Investigators
- Yin-Hsiu Chien, MD, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00713453
- Sponsor's protocol ID
- 200704066R
- Status last verified
- Mar 2015
- First submitted
- 9 Jul 2008
- Last updated
- 24 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Muscle Response to Enzyme Replacement Therapy in Pompe Disease
- ClinicalTrials.gov ID
- NCT00713453
- Acronym / sponsor ID
- 200704066R
- Conditions
- Pompe Disease
- Keywords
- Pompe disease, muscle, enzyme replacement therapy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- Jan 2007
- Primary completion
- Dec 2019 (estimated)
- Study completion
- Dec 2019 (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 study the response of muscle cells from Pompe disease after enzyme replacement therapy
Study design
- Observational model
- Case only
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients with Pompe disease, prepare or on enzyme replacement therapy
Key inclusion criteria (2 listed)
- patients with Pompe disease, prepare or on enzyme replacement therapy
- signed informed consent
Key exclusion criteria (1 listed)
- -
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 morphology changes improvement
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2015.
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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.
Yin-Hsiu Chien, MD 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 24 Mar 2015. 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 Yin-Hsiu Chien, MD (886-2-23123456 ext. 71937, chienyh@ntu.edu.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
all morphology changes improvement (time frame: 6 months). 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 24 Mar 2015.