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

Sponsor
Genzyme, a Sanofi Company (Industry)
Overall Status
Recruiting
CT.gov ID
NCT00418821
Collaborator
BioMarin/Genzyme LLC (Industry)
2
Enrollment
1
Location
154.3
Anticipated Duration (Months)
0
Patients Per Site Per Month

Study Details

Study Description

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
  • 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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
2 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
Actual Study Start Date :
Oct 22, 2010
Anticipated Primary Completion Date :
Sep 1, 2023
Anticipated Study Completion Date :
Sep 1, 2023

Outcome Measures

Primary Outcome Measures

  1. Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG). [Up to 18 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A and Older
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo Rome Italy

Sponsors and Collaborators

  • Genzyme, a Sanofi Company
  • BioMarin/Genzyme LLC

Investigators

  • Study Director: Medical Monitor, Genzyme, a Sanofi Company

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00418821
Other Study ID Numbers:
  • OBS12874
  • 2007-007003-33
  • ALID01803
First Posted:
Jan 5, 2007
Last Update Posted:
Mar 29, 2022
Last Verified:
Mar 1, 2022
Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis I
Syndrome

Study Results

No Results Posted as of Mar 29, 2022