Pompe Pregnancy Sub-Registry
Key facts
- Where
- Brescia, Cagliari, Florence, Genova, Messina, Milan, Monza, Padova, Padua, Pavia, Roma, and Rome, ItalySee the 12 sites
Where in Italy
- BresciaBrescia, ItalyTaking people
- CagliariCagliari, ItalyTaking people
- FlorenceFlorence, ItalyTaking people
- GenovaGenova, ItalyTaking people
- MessinaMessina, ItalyTaking people
- MilanMilan, ItalyTaking people
- MonzaMonza, ItalyTaking people
- PadovaPadova, ItalyTaking people
- PaduaPadua, ItalyTaking people
- PaviaPavia, ItalyTaking people
- RomaRoma, ItalyTaking people
- RomeRome, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Genzyme, a Sanofi Company
- Trial code
- NCT00567073
- Record updated
- 16 April 2026
- Original description
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.Read full original descriptionShow less
A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
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.
Inclusion: Eligible women must:; be enrolled in the Pompe registry (NCT00231400); be pregnant, or have been pregnant with appropriate medical documentation available; provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed; Note: It is recommended that pregnancy data be collected on eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, participation in this Sub-Registry is encouraged for each individual pregnancy.
Exclusion: There are no exclusion criteria for this Sub-Registry.