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

Mucopolysaccharidosis I Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00418821
• Other study ID #: OBS12874
• Secondary ID: 2007-007003-33AL
• Status: Terminated
• Phase: Phase 4
• Start date: October 22, 2010
• Est. completion date: December 21, 2022

Study information

• Verified date: February 2024
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: December 21, 2022
• Est. primary completion date: December 21, 2022
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hurler's Syndrome
• Hurler-Scheie Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Scheie
• Syndrome

Intervention

Biological:
• Laronidase
dose of 0.58mg/kg body weight intravenously (IV) every week

Locations

Country	Name	City	State
Italy	Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo	Rome

Sponsors (2)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company	BioMarin/Genzyme LLC

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Lactating Women With Serum IgG Antibodies to Laronidase		Up to 18 months
Primary	Amount of IgG Antibody Titers to Laronidase in Lactating Women		Baseline and Week 12
Primary	Number of Women Who Breastfed		Up to 18 months
Primary	Number of Women Who Were Successful at Breastfeeding		Up to 18 months
Primary	Number of Women Whose Breast Milk Contains Laronidase		Up to 18 months
Primary	Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease		Up to 18 months
Primary	Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels		Up to 18 months
Primary	Amount of uGAG in the Urine of Women	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.	Baseline and Week 12
Primary	Number of Participants With Medical History of the Mother: Pre-Existing Conditions		Baseline
Primary	Physical Examination Findings of the Mother	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.	Up to 18 months
Primary	Temperature of the Mother		Baseline and Week 12
Primary	Heart Rate of the Mother		Baseline and Week 12
Primary	Respiratory Rate of the Mother		Baseline and Week 12
Primary	Blood Pressure of the Mother	Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.	Baseline and Week 12
Primary	Weight of the Mother		Baseline and Week 12
Primary	Height of the Mother		Baseline
Primary	Number of Infants With Abnormal uGAG Levels		Up to 72 weeks
Primary	Amount of uGAG in the Urine of Infants	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.	Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
Primary	Number of Participants With Medical History of the Infant:Pre-Existing Conditions		Baseline
Primary	Number of Infants With Abnormal Physical Finding	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.	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Heart Rate of the Infant	Heart rate was measured at specified timepoints.	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Respiratory Rate of the Infant	Respiratory rate was measured at specified timepoints.	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Blood Pressure of the Infant	Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.	Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Weight of the Infant		Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Height of the Infant		Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Temperature of the Infant		Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Head Circumference of the Infant		Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores	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.	Week 12, Week 24, Week 36, Week 48, and Week 72
Primary	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
Primary	Time to Development of IgM and IgG Antibodies to Laronidase		up to Week 72
Primary	Amount of IgG and IgM Antibody Titers to Laronidase		Baseline, Week 12, Week 24, Week 36, and Week 72

External Links

•   Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement therapy