Back to resultsStudy of the Effects of Fabrazyme Treatment on Lactation and Infants
Primary Purpose
Fabry Disease, Alpha Galactosidase A Deficiency
Status
Recruiting
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
agalsidase beta
Sponsored by
About this trial
This is an interventional treatment trial for Fabry Disease focused on measuring alpha Galactosidase A, aGAL, rh aGAL, Fabry, GL3, Fabrazyme, Lysosomal Storage Disorder, Enzyme Replacement Therapy (ERT)
Eligibility 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.
Sites / Locations
- Investigational Site Number 840005Recruiting
- Investigational Site Number 840006Recruiting
- investigational site number 01Rhead
- investigational site number 04Bodamer
- investigational site number 03Waldek
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Agalsidase beta
Arm Description
Commercially available Fabrazyme treatment at prescribed dose and regimen as determined by their treating physician
Outcomes
Primary Outcome Measures
Determination of pharmacokinetic parameters of αGAL in breast milk: Cmax
Maximum concentration (Cmax) of αGAL in breast milk
Determination of pharmacokinetic parameters of αGAL in breast milk: AUC0-2h
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
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
Determination of volume of breast milk sample
Fat content of breast milk
Determination of total fat content in breast milk sample
Protein content of breast milk
Determination of total protein content in breast milk sample
Growth response of infants
Absolute change in growth of infants
Development response of infants
Absolute change in weight of infants
Immunologic response of infants - IgG
Formation or presence of immunoglobulin G (IgG) antibodies to recombinant human α galactosidase A (r-hαGAL)
Immunologic response of infants - IgM
Formation or presence of immunoglobulin M (IgM) antibodies to recombinant human α galactosidase A (r-hαGAL)
Secondary Outcome Measures
Full Information
NCT ID
NCT00230607
First Posted
September 29, 2005
Last Updated
May 4, 2022
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT00230607
Brief Title
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
Official Title
A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Recruiting
Study Start Date
August 30, 2006 (Actual)
Primary Completion Date
November 2024 (Anticipated)
Study Completion Date
November 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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
Detailed Description
NOTE: Estimated Enrollment: 10 mothers and up to 10 infants
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease, Alpha Galactosidase A Deficiency
Keywords
alpha Galactosidase A, aGAL, rh aGAL, Fabry, GL3, Fabrazyme, Lysosomal Storage Disorder, Enzyme Replacement Therapy (ERT)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Agalsidase beta
Arm Type
Experimental
Arm Description
Commercially available Fabrazyme treatment at prescribed dose and regimen as determined by their treating physician
Intervention Type
Drug
Intervention Name(s)
agalsidase beta
Other Intervention Name(s)
r-hαGAL, Fabrazyme
Intervention Description
Pharmaceutical form: powder for reconstitution Route of administration: intravenous
Primary Outcome Measure Information:
Title
Determination of pharmacokinetic parameters of αGAL in breast milk: Cmax
Description
Maximum concentration (Cmax) of αGAL in breast milk
Time Frame
Month 1, 3, and 6
Title
Determination of pharmacokinetic parameters of αGAL in breast milk: AUC0-2h
Description
Area under the plasma αGAL concentration-time curve (AUC) from 0 to 2 hours post end of infusion (AUC0-2)
Time Frame
Month 1, 3, and 6
Title
Determination of pharmacokinetic parameters of αGAL in breast milk: lactation clearance
Description
Lactation clearance will be estimated as the amount of αGAL excreted over the sampling period divided by the AUC during the sampling period.
Time Frame
Month 1, 3, and 6
Title
Volume of breast milk
Description
Determination of volume of breast milk sample
Time Frame
Baseline, Month 2, 6, and 12
Title
Fat content of breast milk
Description
Determination of total fat content in breast milk sample
Time Frame
Baseline, Months 2, 6, and 12
Title
Protein content of breast milk
Description
Determination of total protein content in breast milk sample
Time Frame
Baseline, Months 2, 6, and 12
Title
Growth response of infants
Description
Absolute change in growth of infants
Time Frame
Months 1, 2, 3, 6, 12, 18 and Month 24
Title
Development response of infants
Description
Absolute change in weight of infants
Time Frame
Months 1, 2, 3, 6, 12, 18 and Month 24
Title
Immunologic response of infants - IgG
Description
Formation or presence of immunoglobulin G (IgG) antibodies to recombinant human α galactosidase A (r-hαGAL)
Time Frame
Baseline, Months 2, 6, and 12
Title
Immunologic response of infants - IgM
Description
Formation or presence of immunoglobulin M (IgM) antibodies to recombinant human α galactosidase A (r-hαGAL)
Time Frame
Baseline, Months 2, 6, and 12
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Transparency email recommended (Toll free number for US & Canada)
Phone
800-633-1610
Ext
1 then #
Email
Contact-US@sanofi.com
Facility Information:
Facility Name
Investigational Site Number 840005
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Individual Site Status
Recruiting
Facility Name
Investigational Site Number 840006
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Individual Site Status
Recruiting
Facility Name
investigational site number 01Rhead
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53201-1997
Country
United States
Individual Site Status
Completed
Facility Name
investigational site number 04Bodamer
City
Marl
Country
Austria
Individual Site Status
Terminated
Facility Name
investigational site number 03Waldek
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Individual Site Status
Completed
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
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