At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 352 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
In plain English
This is a registry study for people with Pompe disease, also called Glycogen Storage Disease Type II. A registry collects information about patients over time to learn more about their condition. It is run by Genzyme, a Sanofi company.
Who can take part
- You have a confirmed diagnosis of Pompe disease. This means a test showing low levels of the GAA enzyme in blood, skin or muscle tissue, and/or paperwork showing 2 changes (mutations) in the GAA gene.
- You (or a parent or guardian, if needed) have signed the consent and permission forms.
Who may not be able to
- The study has no exclusion criteria. This means no one is kept out of the registry based on other health conditions or treatments.
What taking part involves
- • Not stated — ask the trial team. Registries usually collect your routine health information rather than give a new treatment, but this is not stated in the study details.
Time commitment: Not stated — ask the trial team how many visits are needed, how long the registry runs, or what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- Not specified
- Who
- All
- Number of participants
- 2,000
- Started
- 2004-09-15
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for glycogen storage disease type ii
- • Clinical study - 2,000 participants
- • The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not
Who can take part?
- • Adults
- • Diagnosed with glycogen storage disease type ii
Where?
- • Cambridge - Investigational Site Number : 151340
- • Cardiff - Investigational Site Number : 152334
- • Birmingham - Investigational Site Number : 152287
- • Birmingham - Investigational Site Number : 154240
- • +7 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Pompe disease population. * To evaluate the long-term effectiveness of alglucosidase alfa.
More detail
Study Design Time Perspective: Retrospective and Prospective
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: All genders
Who cannot take part
- ✗There are no exclusion criteria in this Registry
See the full criteria
Where Is This Study? (11 UK sites)
Investigational Site Number : 151340
Cambridge CB2 2QQ, United Kingdom
Investigational Site Number : 152334
Cardiff CF14 4XW, United Kingdom
Investigational Site Number : 152287
Birmingham B15 2TH, United Kingdom
Investigational Site Number : 154240
Birmingham B4 6NH, United Kingdom
Investigational Site Number : 151123
London NW3 2QG, United Kingdom
Investigational Site Number : 152156
Glasgow G51 4TF, United Kingdom
Investigational Site Number : 151646
London WC1N 3BG, United Kingdom
Investigational Site Number : 150303
London WC1N 3JH, United Kingdom
Investigational Site Number : 150964
Salford M6 8HD, United Kingdom
Investigational Site Number : 150455
Manchester M86rb, United Kingdom
Investigational Site Number : 152087
Newcastle upon Tyne NE1 7XE, United Kingdom
How to Get in Touch
Trial Transparency email recommended (Toll free number for US & Canada)
Sponsor contactCONTACT
Pompe Registry HelpLine
Sponsor contactCONTACT