Study of the Effects of Fabrazyme Treatment on Lactation and Infants

• Register: ClinicalTrials.gov
• Last refreshed on: 16 May 2022
• Main ID: NCT00230607
• Date of registration: 29/09/2005
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: Study of the Effects of Fabrazyme Treatment on Lactation and Infants
• Scientific title: A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants
• Date of first enrolment: August 30, 2006
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT00230607
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Austria, United Kingdom, United States

Contacts

• Name: Trial Transparency email recommended (Toll free number for US & Canada)

Key inclusion & exclusion criteria

Inclusion Criteria:

Mothers must meet the following criteria to be enrolled in this study:

- provide signed written informed consent to participate in this study,

- be enrolled in the Fabry Registry and receiving Fabrazyme while lactating,

- agree to adhere to the Fabry Registry recommended schedule of assessments for medical
history, pregnancy outcome, genotyping, and antibody testing, and

- agree to adhere to the schedule of evaluations for this study.

Infants must meet the following criteria to be enrolled in this study:

- have the signed written informed consent of the parent(s)/legal guardian(s) to
participate in this study,

- be born to a mother who is receiving Fabrazyme during lactation,

- be receiving breast milk from the mother, and

- have 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 will be excluded from this study if the mother has received an
investigational drug within 30 days prior to study enrollment.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Alpha Galactosidase A Deficiency

Fabry Disease

Intervention(s)

Drug: agalsidase beta

Primary Outcome(s)

Determination of pharmacokinetic parameters of aGAL in breast milk: AUC0-2h [Time Frame: Month 1, 3, and 6]

Determination of pharmacokinetic parameters of aGAL in breast milk: Cmax [Time Frame: Month 1, 3, and 6]

Fat content of breast milk [Time Frame: Baseline, Months 2, 6, and 12]

Growth response of infants [Time Frame: Months 1, 2, 3, 6, 12, 18 and Month 24]

Development response of infants [Time Frame: Months 1, 2, 3, 6, 12, 18 and Month 24]

Immunologic response of infants - IgM [Time Frame: Baseline, Months 2, 6, and 12]

Determination of pharmacokinetic parameters of aGAL in breast milk: lactation clearance [Time Frame: Month 1, 3, and 6]

Immunologic response of infants - IgG [Time Frame: Baseline, Months 2, 6, and 12]

Protein content of breast milk [Time Frame: Baseline, Months 2, 6, and 12]

Volume of breast milk [Time Frame: Baseline, Month 2, 6, and 12]

Secondary ID(s)

2006-001910-33

AGAL02603

MSC12868

Source(s) of Monetary Support

Please refer to primary and secondary sponsors