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Study of the Effects of Fabrazyme Treatment on Lactation and Infants

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ClinicalTrials.gov Identifier: NCT00230607
Recruitment Status : Recruiting
First Posted : October 3, 2005
Last Update Posted : May 9, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
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

Condition or disease Intervention/treatment Phase
Fabry Disease Alpha Galactosidase A Deficiency Drug: agalsidase beta Phase 4

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants
Actual Study Start Date : August 30, 2006
Estimated Primary Completion Date : November 2024
Estimated Study Completion Date : November 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Agalsidase beta
Commercially available Fabrazyme treatment at prescribed dose and regimen as determined by their treating physician
Drug: agalsidase beta
Pharmaceutical form: powder for reconstitution Route of administration: intravenous
Other Names:
  • r-hαGAL
  • Fabrazyme




Primary Outcome Measures :
  1. 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

  2. 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)

  3. 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.

  4. Volume of breast milk [ Time Frame: Baseline, Month 2, 6, and 12 ]
    Determination of volume of breast milk sample

  5. Fat content of breast milk [ Time Frame: Baseline, Months 2, 6, and 12 ]
    Determination of total fat content in breast milk sample

  6. Protein content of breast milk [ Time Frame: Baseline, Months 2, 6, and 12 ]
    Determination of total protein content in breast milk sample

  7. Growth response of infants [ Time Frame: Months 1, 2, 3, 6, 12, 18 and Month 24 ]
    Absolute change in growth of infants

  8. Development response of infants [ Time Frame: Months 1, 2, 3, 6, 12, 18 and Month 24 ]
    Absolute change in weight of infants

  9. 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)

  10. 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)



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00230607


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-US@sanofi.com

Locations
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United States, Georgia
Investigational Site Number 840005 Recruiting
Decatur, Georgia, United States, 30033
United States, Virginia
Investigational Site Number 840006 Recruiting
Fairfax, Virginia, United States, 22030
United States, Wisconsin
investigational site number 01Rhead Completed
Milwaukee, Wisconsin, United States, 53201-1997
Austria
investigational site number 04Bodamer Terminated
Marl, Austria
United Kingdom
investigational site number 03Waldek Completed
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
Genzyme, a Sanofi Company
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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00230607    
Other Study ID Numbers: AGAL02603
2006-001910-33 ( EudraCT Number )
MSC12868 ( Other Identifier: Sanofi )
First Posted: October 3, 2005    Key Record Dates
Last Update Posted: May 9, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
alpha Galactosidase A
aGAL
rh aGAL
Fabry
GL3
Fabrazyme
Lysosomal Storage Disorder
Enzyme Replacement Therapy (ERT)
Additional relevant MeSH terms:
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Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors