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

Genzyme

Status and phase: Terminated; Phase 4

Conditions: Hurler-Scheie Syndrome; Hurler's Syndrome; Mucopolysaccharidosis I; Scheie

Treatments: Biological: Laronidase

Study type: Interventional

Funder types: Industry

Identifiers

NCT00418821

OBS12874

2007-007003-33 (EudraCT Number)

ALID01803

Details and patient eligibility

About

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.

Full description

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

Enrollment: 2 patients

Sex: Female

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

2 participants in 1 patient group

Laronidase

Experimental group

Description:

Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase and their infants who were breastfeed while the mothers were receiving laronidase.

Treatment:

Biological: Laronidase