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. (see Contacts and Locations)
Verified October 2015 by Sanofi
BioMarin/Genzyme LLC
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
First received: January 3, 2007
Last updated: October 26, 2015
Last verified: October 2015
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
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:

Further study details as provided by Sanofi:

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:
    Biological: Aldurazyme (laronidase)
    dose of 0.58mg/kg body weight intravenously (IV) every week
    Other Name: recombinant human alpha L iduronidase
Detailed Description:
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.

Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No

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
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

Contact: Medical Information 800-745-4447 MedInfo@genzyme.com
Contact: Medical Information 617-252-7832 MedInfo@genzyme.com

Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo Recruiting
Rome, Italy
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00418821     History of Changes
Other Study ID Numbers: ALID01803  2007-007003-33 
Study First Received: January 3, 2007
Last Updated: October 26, 2015
Health Authority: United States: Food and Drug Administration
Italy: The Italian Medicines Agency

Additional relevant MeSH terms:
Mucopolysaccharidosis I
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Pathologic Processes

ClinicalTrials.gov processed this record on May 23, 2016