Pompe Pregnancy Sub-Registry

  • STATUS
    Recruiting
  • End date
    Jan 31, 2034
  • participants needed
    20
  • sponsor
    Genzyme, a Sanofi Company
Updated on 23 January 2022
alglucosidase alfa

Summary

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) 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.

Description

Study Design Time Perspective: Retrospective and Prospective

Details
Condition Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
Treatment alglucosidase alpha, No Treatment
Clinical Study IdentifierNCT00567073
SponsorGenzyme, a Sanofi Company
Last Modified on23 January 2022

Eligibility

Yes No Not Sure

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
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  • You can expect the study team to contact you via email or phone in the next few days.
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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