Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Genzyme

Status and phase: Terminated; Phase 4

Conditions: Fabry Disease; Alpha Galactosidase A Deficiency

Treatments: Drug: agalsidase beta

Study type: Interventional

Funder types: Industry

Identifiers

NCT00230607

2006-001910-33 (EudraCT Number)

AGAL02603

MSC12868 (Other Identifier)

Details and patient eligibility

About

The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full participation of mother and no infant participation was 6 months.

Enrollment: 7 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

Mothers that met the following criteria were enrolled in this study:

• provided signed written informed consent to participate in this study,
• be enrolled in the Fabry Registry and received Fabrazyme while lactating,
• agreed to adhere to the Fabry Registry recommended schedule of assessments for medical history, pregnancy outcome, genotyping, and antibody testing, and
• agreed to adhere to the schedule of evaluations for this study.

Infants that met the following criteria were enrolled in this study:

• had the signed written informed consent of the parent(s)/legal guardian(s) to participate in this study,
• born to a mother who was receiving Fabrazyme during lactation,
• received breast milk from the mother, and
• had the agreement of the parent(s)/legal guardian(s) to adhere to the schedule of evaluations for this study.

Exclusion criteria

• The mother and infant were excluded from this study if the mother received an investigational drug within 30 days prior to study enrollment.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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7 participants in 1 patient group

Fabrazyme

Experimental group

Description:

Mothers with Fabry disease who received commercially available Fabrazyme intravenous infusion as part of the standard of care while lactating/pregnant and their infants who were breastfed while the mothers were receiving Fabrazyme were enrolled in this study.

Treatment:

Drug: agalsidase beta