Trial record 4 of 39 for:
"Mucopolysaccharidosis type I"
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Verified August 2014 by Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
First received: January 3, 2007
Last updated: August 11, 2014
Last verified: August 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.
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: 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
||August 11, 2014
||United States: Food and Drug Administration
Italy: The Italian Medicines Agency
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on August 25, 2014
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases