Pompe Pregnancy Sub-Registry

NCT ID: NCT00567073

Last Updated: 2026-01-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

20 participants

Study Classification

OBSERVATIONAL

Study Start Date

2007-06-18

Study Completion Date

2034-01-31

Brief Summary

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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.

Detailed Description

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Study Design Time Perspective: Retrospective and Prospective

Conditions

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

Study Design

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Observational Model Type

OTHER

Study Time Perspective

OTHER

Study Groups

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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.

No interventions assigned to this group

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

No interventions assigned to this group

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)

No interventions assigned to this group

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

No interventions assigned to this group

Eligibility Criteria

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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.
Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

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Barrow Neurol Group- Site Number : 840087

Phoenix, Arizona, United States

Site Status RECRUITING

Emory University School Of Medicine- Site Number : 840060

Atlanta, Georgia, United States

Site Status RECRUITING

Indianapolis University School of Medicine- Site Number : 840027

Indianapolis, Indiana, United States

Site Status RECRUITING

Spectrum for Health- Site Number : 840019

Grand Rapids, Michigan, United States

Site Status RECRUITING

New York University School Of Medicine- Site Number : 840040

New York, New York, United States

Site Status RECRUITING

Mt. Sinai School of Medicine- Site Number : 840005

New York, New York, United States

Site Status RECRUITING

Duke University Medical Center Genetics Dept- Site Number : 840037

Durham, North Carolina, United States

Site Status RECRUITING

LSD Data Registry Site LLC- Site Number : 840094

Dublin, Ohio, United States

Site Status RECRUITING

Oregon Health and Science University- Site Number : 840095

Portland, Oregon, United States

Site Status COMPLETED

O&O Alpan, LLC- Site Number : 840025

Fairfax, Virginia, United States

Site Status RECRUITING

Investigational Site Number : 056001

Ghent, , Belgium

Site Status RECRUITING

Investigational Site Number : 1910001

Zagreb, , Croatia

Site Status RECRUITING

Investigational Site Number : 1910002

Zagreb, , Croatia

Site Status RECRUITING

Investigational Site Number : 2030001

Prague, , Czechia

Site Status RECRUITING

Investigational Site Number : 380008

Brescia, , Italy

Site Status RECRUITING

Investigational Site Number : 380006

Cagliari, , Italy

Site Status RECRUITING

Investigational Site Number : 380005

Florence, , Italy

Site Status RECRUITING

Investigational Site Number : 380004

Genova, , Italy

Site Status RECRUITING

Investigational Site Number : 380013

Messina, , Italy

Site Status RECRUITING

Investigational Site Number : 380007

Milan, , Italy

Site Status RECRUITING

Investigational Site Number : 380009

Monza, , Italy

Site Status RECRUITING

Investigational Site Number : 380002

Padua, , Italy

Site Status RECRUITING

Investigational Site Number : 380011

Padua, , Italy

Site Status RECRUITING

Investigational Site Number : 380003

Pavia, , Italy

Site Status RECRUITING

Investigational Site Number : 380015

Roma, , Italy

Site Status RECRUITING

Investigational Site Number : 380012

Roma, , Italy

Site Status RECRUITING

Countries

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United States Belgium Croatia Czechia Italy

Central Contacts

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Trial Transparency email recommended (Toll free number for US & Canada)

Role: CONTACT

800-633-1610 ext. option 6

Pompe Registry HelpLine

Role: CONTACT

617-591-5500

Other Identifiers

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AGLU03506

Identifier Type: -

Identifier Source: org_study_id

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