Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Overview

The study will last for up to 2 years (24 months). Full participation for both mother and infant is 24 months, full participation of mother and development of infant is 24 months, while full participation of mother and no infant participation is 6 months

Full Title of Study: “A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: November 2024

Detailed Description

NOTE: Estimated Enrollment: 10 mothers and up to 10 infants

Interventions

  • Drug: agalsidase beta
    • Pharmaceutical form: powder for reconstitution Route of administration: intravenous

Arms, Groups and Cohorts

  • Experimental: Agalsidase beta
    • Commercially available Fabrazyme treatment at prescribed dose and regimen as determined by their treating physician

Clinical Trial Outcome Measures

Primary Measures

  • Determination of pharmacokinetic parameters of αGAL in breast milk: Cmax
    • Time Frame: Month 1, 3, and 6
    • Maximum concentration (Cmax) of αGAL in breast milk
  • Determination of pharmacokinetic parameters of αGAL in breast milk: AUC0-2h
    • Time Frame: Month 1, 3, and 6
    • Area under the plasma αGAL concentration-time curve (AUC) from 0 to 2 hours post end of infusion (AUC0-2)
  • Determination of pharmacokinetic parameters of αGAL in breast milk: lactation clearance
    • Time Frame: Month 1, 3, and 6
    • Lactation clearance will be estimated as the amount of αGAL excreted over the sampling period divided by the AUC during the sampling period.
  • Volume of breast milk
    • Time Frame: Baseline, Month 2, 6, and 12
    • Determination of volume of breast milk sample
  • Fat content of breast milk
    • Time Frame: Baseline, Months 2, 6, and 12
    • Determination of total fat content in breast milk sample
  • Protein content of breast milk
    • Time Frame: Baseline, Months 2, 6, and 12
    • Determination of total protein content in breast milk sample
  • Growth response of infants
    • Time Frame: Months 1, 2, 3, 6, 12, 18 and Month 24
    • Absolute change in growth of infants
  • Development response of infants
    • Time Frame: Months 1, 2, 3, 6, 12, 18 and Month 24
    • Absolute change in weight of infants
  • Immunologic response of infants – IgG
    • Time Frame: Baseline, Months 2, 6, and 12
    • Formation or presence of immunoglobulin G (IgG) antibodies to recombinant human α galactosidase A (r-hαGAL)
  • Immunologic response of infants – IgM
    • Time Frame: Baseline, Months 2, 6, and 12
    • Formation or presence of immunoglobulin M (IgM) antibodies to recombinant human α galactosidase A (r-hαGAL)

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Genzyme, a Sanofi Company
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Contact(s)
    • Trial Transparency email recommended (Toll free number for US & Canada), 800-633-1610, Contact-US@sanofi.com

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