A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Research Study on Laronidase Presence in Breast Milk and Infant Development
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
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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.
This study investigates whether the investigational medication, laronidase, is present in the breast milk of postpartum women who are receiving it as part of their treatment. The purpose is also to understand how this medication might affect the growth, development, and immune response of their breastfed infants. Laronidase is used to treat patients with a deficiency in a specific enzyme that helps break down certain substances in the body.
Participants in the study will undergo procedures to assess the presence of laronidase in breast milk. The study will also monitor the infants for any changes in their growth and development, as well as their immune responses. All participants will need to provide informed consent, and for those under 18, consent will also be required from a legal guardian.
- Who can participate: Mothers must have a documented laronidase deficiency and plan to breastfeed while receiving laronidase therapy. Written informed consent is required, and if under 18, consent from a legal guardian is also necessary.
- Study details: Participants will provide samples for testing laronidase presence in breast milk and will be monitored for effects on infant development.