Pompe Pregnancy Sub-Registry
This study is a registry for women with Pompe disease (Glycogen Storage Disease Type II, a rare genetic disorder) who are pregnant or have recently been pregnant. It aims to understand pregnancy outcomes, including any complications and how babies grow, for women with Pompe disease. This includes women who are receiving enzyme replacement therapy (ERT) with alglucosidase alfa or avalglucosidase alfa, as well as those who are not. Researchers will follow the mothers' pregnancies for about 10 months and then follow their babies for 3 years after birth. To join, you must already be enrolled in the Pompe registry (NCT00231400) and be pregnant or have documentation of a past pregnancy. The study is currently unclear on its recruitment status.
- Study design
- This is an observational study, meaning no experimental treatment is given. It is designed to track pregnancy outcomes for women with Pompe disease.
- What's involved
- You would undergo clinical assessments and receive standard of care treatment as determined by your physician. Data collection would be performed after you provide signed consent.
- Compensation
- Not stated in the trial record.
- Follow-up
- Pregnancy outcomes will be measured at 10 months, and infants will be followed for 3 years post-partum.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Pompe Pregnancy Sub-Registry
At a glance
Conditions
NCT00567073
Where you'd take part
This study runs at 35 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Barrow Neurol Group- Site Number : 840087
Phoenix, Arizonano site contact published
Recruiting
Columbia University Irving Medical Center- Site Number : 157199
New York, New Yorkno site contact published
Recruiting
Duke University Medical Center Genetics Dept- Site Number : 840037
Durham, North Carolinano site contact published
Recruiting
Emory University School Of Medicine- Site Number : 840060
Atlanta, Georgiano site contact published
Recruiting
Greenwood Genetic Center - Greenville- Site Number : 151184
Greenville, South Carolinano site contact published
Recruiting
Indianapolis University School of Medicine- Site Number : 840027
Indianapolis, Indianano site contact published
Recruiting
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 150978
São Paulo, Brazilno site contact published
Recruiting
Investigational Site Number : 056001
Ghent, Belgiumno site contact published
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Study Director · STUDY_DIRECTOR · Genzyme, a Sanofi Company
Who to contact
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Inclusion
What this trial measures
- 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 alfa10 Months
- Follow-up of infants born to women with Pompe disease for 3 years post-partum3 years