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

Phase 4

Terminated

• Conditions: Mucopolysaccharidosis I; Hurler's Syndrome; Scheie; Hurler-Scheie Syndrome
• Interventions: Biological: Laronidase

• Registration Number: NCT00418821
• Lead Sponsor: Genzyme, a Sanofi Company

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 Timeline

• Study First Submitted: 2007-01-03
• Study First Submitted QC: 2007-01-04
• Study First Posted: 2007-01-05
• Study Start: 2010-10-22
• Primary Completion: 2022-12-21
• Study Completion: 2022-12-21
• Results First Submitted: 2023-12-11
• Status Verified: 2024-02
• Results First Submitted QC: 2024-02-16
• Last Update Submitted: 2024-02-16
• Results First Posted: 2024-02-20
• Last Update Posted: 2024-02-20

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Female
• Target Recruitment: 2

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Laronidase	Laronidase	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.

Primary Outcome Measures

Name	Time	Method
Number of Women Who Breastfed	Up to 18 months
Temperature of the Mother	Baseline and Week 12
Heart Rate of the Mother	Baseline and Week 12
Respiratory Rate of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72	Respiratory rate was measured at specified timepoints.
Number of Lactating Women With Serum IgG Antibodies to Laronidase	Up to 18 months
Number of Women Who Were Successful at Breastfeeding	Up to 18 months
Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease	Up to 18 months
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels	Up to 18 months
Physical Examination Findings of the Mother	Up to 18 months	Physical examination was performed at specified intervals. Physical examination included the following physical observations: general appearance, skin, head, ears, eyes, nose, and throat, lymph nodes, abdomen, extremities/joints, neurological, mental status, and the following, if appropriate, breasts, external genitalia, pelvic, and rectal.
Heart Rate of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72	Heart rate was measured at specified timepoints.
Respiratory Rate of the Mother	Baseline and Week 12
Height of the Mother	Baseline
Amount of IgG Antibody Titers to Laronidase in Lactating Women	Baseline and Week 12
Number of Women Whose Breast Milk Contains Laronidase	Up to 18 months
Blood Pressure of the Mother	Baseline and Week 12	Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Number of Infants With Abnormal Physical Finding	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72	Physical examination included the following physical observations: general appearance, skin, lymph nodes, heart, lungs, abdomen, extremities/joints, neurological, mental status, breasts, external genitalia, pelvic, rectal, and Heent.
Weight of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Head Circumference of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point	Baseline, Week 12, Week 24, Week 36, and Week 72
Amount of IgG and IgM Antibody Titers to Laronidase	Baseline, Week 12, Week 24, Week 36, and Week 72
Amount of uGAG in the Urine of Women	Baseline and Week 12	Urine samples were collected at specified intervals to measure uGAG in the urine of women. Reference range of uGAG between 2.64 - 37.65 was considered as normal. mg/g creatinine = milligram per gram of creatinine.
Weight of the Mother	Baseline and Week 12
Blood Pressure of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72	Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores	Week 12, Week 24, Week 36, Week 48, and Week 72	Infant development was assessed with the Denver II Developmental Screening Test. It consisted of 5 areas/subscore i.e., test behavior, personal-social, fine motor, language, and gross motor. The number of participants with normal overall assessment measured using Denver II Developmental Screening Scores have been reported by visit.
Number of Participants With Medical History of the Mother: Pre-Existing Conditions	Baseline
Number of Infants With Abnormal uGAG Levels	Up to 72 weeks
Amount of uGAG in the Urine of Infants	Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72	Urine samples were collected at specified intervals to measure uGAG in the urine of infant. Reference range of uGAG between 30 - 300 was considered as normal.
Number of Participants With Medical History of the Infant:Pre-Existing Conditions	Baseline
Height of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Temperature of the Infant	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Time to Development of IgM and IgG Antibodies to Laronidase	up to Week 72

Trial Locations

Locations (1)

Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo; 🇮🇹 Rome, Italy