Study of the Effects of Fabrazyme Treatment on Lactation and Infants

NCT ID: NCT00230607

Last Updated: 2024-04-10

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-05-28
• Study Completion Date: 2024-02-09

Brief Summary

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.

Conditions

Fabry Disease; Alpha Galactosidase A Deficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Fabrazyme

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.

Group Type: EXPERIMENTAL

agalsidase beta

Intervention Type: DRUG

Pharmaceutical form: powder for reconstitution Route of administration: intravenous

Interventions

agalsidase beta

Pharmaceutical form: powder for reconstitution Route of administration: intravenous

Intervention Type: DRUG

Other Intervention Names

r-haGAL; Fabrazyme

Eligibility Criteria

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigational Site Number 840005

Decatur, Georgia, United States

Investigational Site Number 840006

Fairfax, Virginia, United States

investigational site number 01Rhead

Milwaukee, Wisconsin, United States

investigational site number 04Bodamer

Marl, , Austria

investigational site number 03Waldek

Salford, , United Kingdom

Countries

United States; Austria; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2006-001910-33

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MSC12868

Identifier Type: OTHER

Identifier Source: secondary_id

AGAL02603

Identifier Type: -

Identifier Source: org_study_id