Pompe Lactation Sub-Registry

Pompe Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00566878
• Other study ID #: AGLU03406
• Secondary ID: LTS13972
• Status: Completed
• Start date: March 31, 2012
• Est. completion date: February 9, 2024

Study information

• Verified date: March 2024
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Est. completion date: February 9, 2024
• Est. primary completion date: February 9, 2024
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type II
• Pompe Disease

Intervention

Biological:
• alglucosidase alfa
At least one infusion of alglucosidase alfa post partum

Locations

Country	Name	City	State
United States	Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient	Cambridge	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		6 Months
Primary	breast milk production and composition in women with Pompe disease who receive alglucosidase alfa		6 Months