COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00418821
Recruitment Status : Unknown
Verified August 2017 by Sanofi ( Genzyme, a Sanofi Company ).
Recruitment status was:  Recruiting
First Posted : January 5, 2007
Last Update Posted : August 22, 2017
BioMarin/Genzyme LLC
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
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 or disease Intervention/treatment Phase
Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie Biological: Aldurazyme (laronidase) Phase 4

Detailed Description:
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : October 2010
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2020

Intervention Details:
  • Biological: Aldurazyme (laronidase)
    dose of 0.58mg/kg body weight intravenously (IV) every week
    Other Name: recombinant human alpha L iduronidase

Primary Outcome Measures :
  1. Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG). [ Time Frame: Up to 18 months ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00418821

Layout table for location contacts
Contact: Medical Information 800-745-4447
Contact: Medical Information 617-252-7832

Layout table for location information
Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo Recruiting
Rome, Italy
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Layout table for investigator information
Study Director: Medical Monitor Genzyme, a Sanofi Company

Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Genzyme, a Sanofi Company Identifier: NCT00418821    
Other Study ID Numbers: ALID01803
2007-007003-33 ( EudraCT Number )
First Posted: January 5, 2007    Key Record Dates
Last Update Posted: August 22, 2017
Last Verified: August 2017
Additional relevant MeSH terms:
Layout table for MeSH terms
Mucopolysaccharidosis I
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases