A Study of the Effects of Fabrazyme (Agalsidase Beta) on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00230607
First received: September 29, 2005
Last updated: February 10, 2014
Last verified: February 2014
  Purpose

The purpose of this study is to observe the potential effects of Fabrazyme (agalsidase beta) treatment on lactation and on the growth, development, and immunologic response of infants born to mothers with Fabry disease who are treated with Fabrazyme during lactation. There are 3 participation scenarios: mother/infant full participation, mother full participation/infant development assessment only, and mother full participation/infant no participation. Whether or not the mother continues to lactate will be assessed at each visit. If the mother is no longer lactating, the mother will discontinue this study but continue to be followed in the Fabry Registry. The infant (if participating) will be followed for development only for the remainder of this 24 month study.


Condition Intervention Phase
Fabry Disease
Biological: agalsidase beta
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: 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:

Primary Outcome Measures:
  • Presence of alpha-galactosidase (αGAL) activity in breast milk of mothers who are being treated with Fabrazyme (agalsidase beta) during lactation [ Time Frame: Time frame: Month 1, 3, and 6] ] [ Designated as safety issue: No ]
  • Growth and development of infants born to mothers who have received Fabrazyme (agalsidase beta) treatment during lactation [ Time Frame: Up to 24 months ] [ Designated as safety issue: No ]
  • Volume, total fat and protein content in breast milk of mothers who are being treated with Fabrazyme (agalsidase beta) during lactation [ Time Frame: Month 1, 3, and 6 ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Milk αGAL to plasma αGAL ratio [ Time Frame: Months 1, 3, and 6 ] [ Designated as safety issue: No ]
  • Formation or continued presence of immunoglobulin G (IgG) and immunoglobulin M (IgM) antibodies to α-galactosidase A (r-hαGAL) in infants receiving breast milk from mothers treated with Fabrazyme (agalsidase beta) during lactation [ Time Frame: Baseline, Months 2, 6, and 12 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 10
Study Start Date: August 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: Mother and Infant all assessments
Note: A participating mother may choose the level of participation for her infant (i.e., these arms are not assigned randomly)
Biological: agalsidase beta
Mothers receive Fabrazyme (agalsidase beta) treatment at their prescribed dose and regimen as determined by their treating physician.
Other Names:
  • r-hαGAL
  • Fabrazyme
Experimental: Mother all assessments, Infant developmental assessments only
Note: A participating mother may choose the level of participation for her infant (i.e., these arms are not assigned randomly)
Biological: agalsidase beta
Mothers receive Fabrazyme (agalsidase beta) treatment at their prescribed dose and regimen as determined by their treating physician.
Other Names:
  • r-hαGAL
  • Fabrazyme
Experimental: Mother only/ no infant
Note: A participating mother may choose the level of participation for her infant (i.e., these arms are not assigned randomly)
Biological: agalsidase beta
Mothers receive Fabrazyme (agalsidase beta) treatment at their prescribed dose and regimen as determined by their treating physician.
Other Names:
  • r-hαGAL
  • Fabrazyme

Detailed Description:

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

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Mother must provide signed written informed consent to participate in this study.
  • Mother must be enrolled in the Fabry Registry and receiving Fabrazyme (agalsidase beta) while lactating.
  • Mother must agree to adhere to the Fabry Registry recommended schedule of assessments for medical history, pregnancy outcome, genotyping, and antibody testing.
  • Mother must agree to adhere to the schedule of evaluations for this study.
  • Infant must have the signed written informed consent of the parent(s)/legal guardian(s) to participate in this study.
  • Infant must be born to a mother who is receiving Fabrazyme (agalsidase beta) during lactation.
  • Infant must be receiving breast milk from the mother.
  • Infant must 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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00230607     History of Changes
Other Study ID Numbers: AGAL02603, 2006-001910-33
Study First Received: September 29, 2005
Last Updated: February 10, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Sanofi:
alpha Galactosidase A
aGAL
rh aGAL
Fabry
GL3
Fabrazyme
Lysosomal Storage Disorder
Enzyme Replacement Therapy (ERT)

Additional relevant MeSH terms:
Brain Diseases, Metabolic, Inborn
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on July 24, 2014