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 319 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 sub-registry (an extra part of a bigger study) that collects information about pregnancies in women with Pompe disease, also called Glycogen Storage Disease Type II (GSD-II). It is for women already taking part in the main Pompe registry, which has the clinical trial number NCT00231400. The study is run by Genzyme, a Sanofi company.
Who can take part
- You are already enrolled in the main Pompe registry (clinical trial number NCT00231400).
- You are pregnant now, or you have been pregnant in the past and medical records about the pregnancy are available.
- You sign a consent form and any authorisation forms before any information is collected.
- If you have more than one pregnancy, you are encouraged to take part for each pregnancy.
Who may not be able to
- There are no exclusion criteria, so nothing is listed that would stop you taking part.
What taking part involves
- • This is not a treatment study. Taking part means letting the team collect information (data) about your pregnancy.
- • Information about your pregnancy is collected whether or not your baby joins.
- • Exactly what information is collected is not stated - ask the trial team.
Time commitment: You sign a consent form and let the team collect information about your pregnancy; how many visits, how often, or how long it lasts is not stated - ask the trial team.
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
- Female
- Number of participants
- 20
- Started
- 2007-06-18
- Last checked
- 2026-04
Plain English Summary
What is this study?
- • Testing a new treatment for glycogen storage disease type ii (gsd-ii)
- • Clinical study - 20 participants
- • This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated
Who can take part?
- • Adults
- • Diagnosed with glycogen storage disease type ii (gsd-ii)
- • Female only
Where?
- • Cardiff - Investigational Site Number : 87147
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase alfa or avalglucosidase 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: Female only
What the study is looking for
- ✓Eligible women must:
- ✓be enrolled in the Pompe registry (NCT00231400)
- ✓be pregnant, or have been pregnant with appropriate medical documentation available.
- ✓provide a signed agreement to take part and authorization form(s) to participate in the Sub-Registry prior to any...
Who cannot take part
- ✗There are no exclusion criteria for this Sub-Registry
See the full criteria
Where Is This Study? (1 UK site)
Investigational Site Number : 87147
Cardiff CF14 4XW, United Kingdom
How to Get in Touch
Trial Transparency email recommended (Toll free number for US & Canada)
Sponsor contactCONTACT
Pompe Registry HelpLine
Sponsor contactCONTACT