Trial record 7 of 33 for:
Open Studies | "Mucopolysaccharidoses"
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
This study is currently recruiting participants.
Verified June 2013 by Genzyme
Sponsor:
Genzyme
Collaborator:
BioMarin/Genzyme LLC
Information provided by (Responsible Party):
Genzyme
ClinicalTrials.gov Identifier:
NCT00418821
First received: January 3, 2007
Last updated: June 10, 2013
Last verified: June 2013
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Purpose
The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic response of their breastfed infants.
| Condition | Intervention | Phase |
|---|---|---|
|
Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie |
Biological: Aldurazyme (laronidase) |
Phase 4 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants |
Resource links provided by NLM:
MedlinePlus related topics:
Breast Feeding
Drug Information available for:
Laronidase
U.S. FDA Resources
Further study details as provided by Genzyme:
Primary Outcome Measures:
- Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG). [ Time Frame: Up to 18 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 10 |
| Study Start Date: | October 2010 |
| Estimated Study Completion Date: | January 2020 |
| Estimated Primary Completion Date: | January 2020 (Final data collection date for primary outcome measure) |
Intervention Details:
Detailed Description:
-
Biological: Aldurazyme (laronidase)
dose of 0.58mg/kg body weight intravenously (IV) every week
Other Name: recombinant human alpha L iduronidase
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.
Eligibility| Genders Eligible for Study: | Female |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria (Mothers):
- The patient must have a documented laronidase deficiency with a fibroblast, plasma, serum, leukocyte, or dried blood spot laronidase enzyme activity assay.
- Be pregnant, planning to breastfeed post-partum, and receiving Aldurazyme (laronidase) therapy while breastfeeding.
- Provide signed, written informed consent prior to any protocol-related procedures. Consent of a legally authorized guardian(s) is (are) required for mothers younger than 18 years of age. If a mother is under 18 years old and can understand the consent, written informed consent is required from both the mother and the authorized guardian(s).
- Provide signed, written informed consent for their infants to participate as study patients. If a mother is younger than 18 years of age, consent for mother and infant will be obtained from the legal guardian.
Exclusion Criteria (Mothers and Infants):
- Have a medical condition, serious intercurrent illness, or other extenuating circumstance that may interfere with study compliance, including all prescribed evaluations and follow-up activities.
- Have received an investigational drug within 30 days prior to study enrollment.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00418821
Contacts
| Contact: Medical Information | 800-745-4447 | MedInfo@genzyme.com |
| Contact: Medical Information | 617-252-7832 | MedInfo@genzyme.com |
Locations
| Italy | |
| Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo | Recruiting |
| Rome, Italy | |
Sponsors and Collaborators
Genzyme
BioMarin/Genzyme LLC
Investigators
| Study Director: | Medical Monitor | Genzyme |
More Information
No publications provided
| Responsible Party: | Genzyme |
| ClinicalTrials.gov Identifier: | NCT00418821 History of Changes |
| Other Study ID Numbers: | ALID01803, 2007-007003-33 |
| Study First Received: | January 3, 2007 |
| Last Updated: | June 10, 2013 |
| Health Authority: | United States: Food and Drug Administration Italy: The Italian Medicines Agency |
Additional relevant MeSH terms:
|
Mucopolysaccharidosis I Mucopolysaccharidoses Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |
ClinicalTrials.gov processed this record on June 18, 2013