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

Investigating the Presence of Aldurazyme® (laronidase) in Breast Milk and Its Effects on Infants

Not Recruiting
Female
Phase 4
2 participants needed

Study Overview

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.

Study Details

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

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie
  • Gender: Female

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.

The purpose of this study is to determine if the investigational medication, which is used for treating Mucopolysaccharidosis I (MPS I), is present in the breast milk of post-partum women. This study will also assess the effects of the investigational medication on the growth, development, and immune response of their breastfed infants.

Participants in the study will include mothers who are receiving the investigational medication during breastfeeding. The study will involve collecting breast milk samples from these mothers to test for the presence of the medication. Additionally, the health and development of their infants will be monitored to understand any potential impact of the medication.

  • Who can participate: Women with a documented deficiency in the enzyme laronidase, who are pregnant, planning to breastfeed, and receiving the investigational medication, are eligible. They must provide signed informed consent, and if under 18, consent from a legal guardian is required.
  • Study details: Participants will provide breast milk samples, and their infants' growth and development will be monitored. Mothers must consent to the study, and if under 18, legal guardian consent is needed.
Updated on 20 Feb 2024. Study ID: NCT00418821