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 January 2014 by Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
First received: January 3, 2007
Last updated: January 23, 2014
Last verified: January 2014
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.
Biological: Aldurazyme (laronidase)
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||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
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:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||January 2020 (Final data collection date for primary outcome measure)
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.
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00418821
|Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo
|Rome, Italy |
Genzyme, a Sanofi Company
||Genzyme, a Sanofi Company
No publications provided
||Sanofi ( Genzyme, a Sanofi Company )
History of Changes
|Other Study ID Numbers:
|Study First Received:
||January 3, 2007
||January 23, 2014
||United States: Food and Drug Administration
Italy: The Italian Medicines Agency
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on April 15, 2014
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases