Trial record 15 of 138 for:
Open Studies | "Brain Diseases, Metabolic, Inborn"
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.
Verified January 2013 by Genzyme
Sponsor:
Genzyme
Information provided by (Responsible Party):
Genzyme
ClinicalTrials.gov Identifier:
NCT00230607
First received: September 29, 2005
Last updated: January 7, 2013
Last verified: January 2013
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
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:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Fabry disease
Farber lipogranulomatosis
Schindler disease
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Breast Feeding
U.S. FDA Resources
Further study details as provided by Genzyme:
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:
|
|
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:
|
|
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:
|
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
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
Investigators
| Study Director: | Medical Monitor | Genzyme |
More Information
No publications provided
| Responsible Party: | Genzyme |
| ClinicalTrials.gov Identifier: | NCT00230607 History of Changes |
| Other Study ID Numbers: | AGAL02603, 2006-001910-33 |
| Study First Received: | September 29, 2005 |
| Last Updated: | January 7, 2013 |
| Health Authority: | United States: Food and Drug Administration United Kingdom: Medicines and Healthcare Products Regulatory Agency |
Keywords provided by Genzyme:
|
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 May 22, 2013