Pompe Pregnancy Sub-Registry

Conditions

• Glycogen Storage Disease Type II (GSD-II)
• Glycogenesis 2 Acid Maltase Deficiency
• Pompe Disease (Late-onset)

Eligibility Criteria

Sex

FEMALE

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Jun 18, 2007

Status verified

Apr 2026

Primary completion

Jan 31, 2034

Completion

Jan 31, 2034

Study Design

Enrollment

20 participants (estimated)

Arms

• Arm: Pregnant women with confirmed diagnosis of Pompe Disease
  No experimental intervention is given. Pregnant women with confirmed diagnosis of Pompe disease that are participating in the Pompe Registry (NCT00231400) and consented to participate in the Pompe Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglusidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated.
• Arm: Pregnant women receiving no treatment for Pompe disease
  Pregnant women with Pompe disease enrolled in the Pompe disease registry (NCT00231400) who are not receiving treatment
• Arm: Infants born to mothers receiving treatment for Pompe disease
  The infants of mothers with Pompe disease enrolled in the Pompe disease registry (NCT00231400) where the mothers are receiving treatment of alglucosidase alfa (Myozyme/Lumizyme) or avalglucosidase alfa (Nexviadyme/Nexviazyme)
• Arm: Infants born to mothers receiving no treatment for Pompe disease
  The infants of mothers with Pompe disease enrolled in the Pompe Disease Registry (NCT00231400) where the mothers are not receiving Treatment

Primary Outcome Measure

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 [ Time Frame: 10 Months ]

Central Contacts

• Trial Transparency email recommended (Toll free number for US & Canada)
  800-633-1610
  [email protected]
• Pompe Registry HelpLine
  617-591-5500

Locations (15)

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