Home › Trials › NCT00567073

Tracking pregnancy outcomes in women with Pompe disease

A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease

Observational Sanofi · NCT00567073

This study looks at the pregnancy experiences and outcomes for women with Pompe disease to see how it affects both them and their babies.

Quick facts

Study typeObservational
Enrollment20 (estimated)
SexFemale
SponsorSanofi Industry-sponsored
Locations35 sites (Phoenix, Arizona and 34 other locations)
Trial IDNCT00567073 on ClinicalTrials.gov

What this trial studies

This Sub-registry is a multicenter, international, longitudinal program that observes pregnancy outcomes for women enrolled in the Pompe Registry. It includes women regardless of whether they are receiving disease-specific therapy for Pompe disease. The study collects data on pregnancy complications and infant growth, allowing for both retrospective and prospective assessments. Participants undergo clinical evaluations and receive standard care as determined by their healthcare providers.

Who should consider this trial

Good fit: Ideal candidates for this study are pregnant women who are enrolled in the Pompe Registry.

Not a fit: Patients who are not pregnant or who are not enrolled in the Pompe Registry may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the pregnancy outcomes of women with Pompe disease, potentially improving care and management during pregnancy.

How similar studies have performed: Other studies have successfully tracked pregnancy outcomes in similar patient populations, indicating that this approach is viable.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

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 Criteria:

There are no exclusion criteria for this Sub-Registry

Where this trial is running

Phoenix, Arizona and 34 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Glycogen Storage Disease Type IIPompe DiseaseGlycogenesis 2 Acid Maltase DeficiencyGSD-IIAcid Maltase Deficiency DiseaseGlycogenosis II
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.