Pompe Lactation Sub-Registry

March 15, 2024 updated by: Genzyme, a Sanofi Company

A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.

The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease who receive alglucosidase alfa.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Biological: alglucosidase alfa

Study Type

Observational

Enrollment (Actual)

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

United States
- Massachusetts
  - Cambridge, Massachusetts, United States, 02142
    - Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient
- Michigan
  - Grand Rapids, Michigan, United States, 49503

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.

Eligibility Criteria

Ages Eligible for Study

Child
Adult
Older Adult

Accepts Healthy Volunteers

Sampling Method

Non-Probability Sample

Study Population

Females diagnosed with Pompe Disease and Lactating

Description

Inclusion Criteria:

Must be enrolled in Pompe Registry (NCT00231400)
Must be pregnant and intend to breast-feed or be currently lactating and receive at least one infusion of alglucosidase alfa while lactating
Provide a signed Patient Information and Authorization form to participate in the sub-registry prior to any sub-registry-related assessments are performed
Agree to adhere to the sub-registry guidelines for antibody testing and recommended schedule of assessments.

Exclusion Criteria:

Patients will be excluded from this sub-registry if they have received an investigational drug (excluding alglucosidase alfa in regions where alglucosidase alfa is not commercially available) within 30 days prior to Visit 1 breast milk collection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Observational Models: Other
Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
alglucosidase alfa accumulation in immature (1-3 months post partum) or mature (4-6 months post partum) breast milk sample from mothers with Pompe disease who are being treated with alglucosidase alfa. Time Frame: 6 Months	6 Months
breast milk production and composition in women with Pompe disease who receive alglucosidase alfa Time Frame: 6 Months	6 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Investigators

Study Director: Medical Monitor, Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 31, 2012

Primary Completion (Actual)

February 9, 2024

Study Completion (Actual)

February 9, 2024

Study Registration Dates

First Submitted

December 1, 2007

First Submitted That Met QC Criteria