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Study of the Effects of Fabrazyme Treatment on Lactation and Infants

This study is currently recruiting participants.
Verified August 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
ClinicalTrials.gov Identifier:
NCT00230607
First Posted: October 3, 2005
Last Update Posted: August 7, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  Purpose
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 Intervention Phase
Fabry Disease Alpha Galactosidase A Deficiency Drug: agalsidase beta Phase 4

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome 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)


Estimated Enrollment: 20
Actual Study Start Date: May 28, 2006
Estimated Study Completion Date: January 2020
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
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.
  Contacts and Locations
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
Contact: Medical Information 800-745-4447 medinfo@genzyme.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

Locations
United States, Massachusetts
Participation in this study is not limited to this facility; facilities not yet active may enroll upon identification of a patient Recruiting
Cambridge, Massachusetts, United States
United States, Texas
Terminated
Dallas, Texas, United States
United States, Wisconsin
Completed
Milwaukee, Wisconsin, United States
Austria
Completed
Salzburg, Austria
United Kingdom
Completed
Salford, United Kingdom
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00230607     History of Changes
Other Study ID Numbers: AGAL02603
2006-001910-33 ( EudraCT Number )
MSC12868 ( Other Identifier: Sanofi )
First Submitted: September 29, 2005
First Posted: October 3, 2005
Last Update Posted: August 7, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Individual participant data (IPD) and supporting clinical documents are available for request at clinicalstudydatarequest.com. While making information available Sanofi continues to protect the privacy of the participants in clinical trials and to remove commercially confidential information (CCI). Details on Data Sharing criteria and process for requesting access can be found at this web address: clinicalstudydatarequest.com

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:
Fabry Disease
Sphingolipidoses
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
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders