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.

Eligibility criteria

Qualifiers

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.

Disqualifiers

None

Trial design

Treatments tested in this trial

  • Not listed

Trial groups

20 Participants
are grouped into 4 trial groups

Locations

35
Australia
Investigational Site Number : 1510692145, WestmeadNew South Wales, Australia
Belgium
Investigational Site Number : 0560019000, Ghent Belgium
Brazil
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 15097804020-041, São Paulo Brazil
Croatia
Investigational Site Number : 191000110000, Zagreb Croatia

Sponsors and collaborators

Genzyme, a Sanofi Company

Lead sponsor