Pompe Pregnancy Sub-Registry

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. 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.

Conditions

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

Study Overview

Study Details

Study overview

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.

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

Pompe Pregnancy Sub-Registry

Condition
Glycogen Storage Disease Type II (GSD-II)
Intervention / Treatment

-

Contacts and Locations

Phoenix

Barrow Neurol Group- Site Number : 840087, Phoenix, Arizona, United States, 85013

Atlanta

Emory University School Of Medicine- Site Number : 840060, Atlanta, Georgia, United States, 30322

Indianapolis

Indianapolis University School of Medicine- Site Number : 840027, Indianapolis, Indiana, United States, 46202

Grand Rapids

Spectrum for Health- Site Number : 840019, Grand Rapids, Michigan, United States, 49503

New York

New York University School Of Medicine- Site Number : 840040, New York, New York, United States, 10016

New York

Mt. Sinai School of Medicine- Site Number : 840005, New York, New York, United States, 10029

Durham

Duke University Medical Center Genetics Dept- Site Number : 840037, Durham, North Carolina, United States, 27710

Dublin

LSD Data Registry Site LLC- Site Number : 840094, Dublin, Ohio, United States, 43017

Portland

Oregon Health and Science University- Site Number : 840095, Portland, Oregon, United States, 97239

Fairfax

O&O Alpan, LLC- Site Number : 840025, Fairfax, Virginia, United States, 22030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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.

Ages Eligible for Study

to

Sexes Eligible for Study

FEMALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Genzyme, a Sanofi Company,

Study Director, STUDY_DIRECTOR, Genzyme, a Sanofi Company

Study Record Dates

2034-01-31