Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00418821
Recruitment Status : Recruiting
First Posted : January 5, 2007
Last Update Posted : August 22, 2017
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date  ICMJE January 3, 2007
First Posted Date  ICMJE January 5, 2007
Last Update Posted Date August 22, 2017
Study Start Date  ICMJE October 2010
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 2, 2012)
Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG). [ Time Frame: Up to 18 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 4, 2007)
To determine the effect of Aldurazyme® (laronidase) treatment in lactating MPS I patients and the growth, development, and immunologic response of their breastfed infants.
Change History Complete list of historical versions of study NCT00418821 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mucopolysaccharidosis I
  • Hurler's Syndrome
  • Hurler-Scheie Syndrome
  • Scheie
Intervention  ICMJE Biological: Aldurazyme (laronidase)
dose of 0.58mg/kg body weight intravenously (IV) every week
Other Name: recombinant human alpha L iduronidase
Study Arms  ICMJE Not Provided
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 4, 2007)
10
Original Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2020
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information 800-745-4447 MedInfo@genzyme.com
Contact: Medical Information 617-252-7832 MedInfo@genzyme.com
Listed Location Countries  ICMJE Italy
Removed Location Countries United States
 
Administrative Information
NCT Number  ICMJE NCT00418821
Other Study ID Numbers  ICMJE ALID01803
2007-007003-33 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Study Sponsor  ICMJE Genzyme, a Sanofi Company
Collaborators  ICMJE BioMarin/Genzyme LLC
Investigators  ICMJE
Study Director: Medical Monitor Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date August 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP