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

study id #: NCT00418821

condition: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie

status: Recruiting

purpose:

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.

intervention: Aldurazyme (laronidase)

results: https://clinicaltrials.gov/ct2/show/results/NCT00418821

last updated: February 26, 2022

start date: October 22, 2010

estimated completion: September 2023

last updated: November 18, 2021

phase of development: Phase 4

size / enrollment: 10

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

primary outcomes:

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

inclusion 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: 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.

sponsor: Genzyme, a Sanofi Company

contacts: Medical Information, 800-745-4447, [email protected]

investigators: Medical Monitor,Genzyme, A Sanofi Company

trial center locations: Italy

• Italy
  Dipartimento di Scienze Pediatriche Medico – Chirurgiche e Neuro Scienze dello Suiluppo