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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

Primary Purpose

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

Status
Recruiting
Phase
Phase 4
Locations
Italy
Study Type
Interventional
Intervention
Aldurazyme (laronidase)
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis I

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)FemaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello SuiluppoRecruiting

Outcomes

Primary Outcome Measures

Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG).

Secondary Outcome Measures

Full Information

First Posted
January 3, 2007
Last Updated
March 21, 2023
Sponsor
Genzyme, a Sanofi Company
Collaborators
BioMarin/Genzyme LLC
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1. Study Identification

Unique Protocol Identification Number
NCT00418821
Brief Title
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 22, 2010 (Actual)
Primary Completion Date
September 24, 2024 (Anticipated)
Study Completion Date
September 24, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company
Collaborators
BioMarin/Genzyme LLC

4. Oversight

Data Monitoring Committee
No

5. 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.
Detailed Description
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
2 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Biological
Intervention Name(s)
Aldurazyme (laronidase)
Other Intervention Name(s)
recombinant human alpha L iduronidase
Intervention Description
dose of 0.58mg/kg body weight intravenously (IV) every week
Primary Outcome Measure Information:
Title
Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG).
Time Frame
Up to 18 months

10. Eligibility

Sex
Female
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
800-745-4447
Email
MedInfo@genzyme.com
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-252-7832
Email
MedInfo@genzyme.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo
City
Rome
Country
Italy
Individual Site Status
Recruiting

12. IPD Sharing Statement

Citations:
PubMed Identifier
25864295
Citation
Castorina M, Antuzzi D, Richards SM, Cox GF, Xue Y. Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement. therapy. Clin Exp Obstet Gynecol. 2015;42(1):108-13.
Results Reference
derived

Learn more about this trial

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

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