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

Phase 4TerminatedNCT00418821

Genzyme, a Sanofi Company2 enrolled

Overview

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.

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie

Interventions

LaronidaseBIOLOGICAL

dose of 0.58mg/kg body weight intravenously (IV) every week

Eligibility

Sex: FEMALE

Medical Language ↔ Plain English

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.

Locations (1)

Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo

Rome, Italy

Outcomes

Primary Outcomes

Number of Lactating Women With Serum IgG Antibodies to Laronidase

Time frame: Up to 18 months

Amount of IgG Antibody Titers to Laronidase in Lactating Women

Time frame: Baseline and Week 12

Number of Women Who Breastfed

Time frame: Up to 18 months

Number of Women Who Were Successful at Breastfeeding

Time frame: Up to 18 months

Number of Women Whose Breast Milk Contains Laronidase

Time frame: Up to 18 months

Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease

Time frame: Up to 18 months

Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels

Time frame: Up to 18 months

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.

Time frame: Baseline and Week 12

Number of Participants With Medical History of the Mother: Pre-Existing Conditions

Time frame: Baseline

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.

Data from ClinicalTrials.gov

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Time frame: Up to 18 months

Temperature of the Mother

Time frame: Baseline and Week 12

Heart Rate of the Mother

Time frame: Baseline and Week 12

Respiratory Rate of the Mother

Time frame: Baseline and Week 12

Blood Pressure of the Mother

Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.

Time frame: Baseline and Week 12

Weight of the Mother

Time frame: Baseline and Week 12

Height of the Mother

Time frame: Baseline

Number of Infants With Abnormal uGAG Levels

Time frame: Up to 72 weeks

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.

Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72

Number of Participants With Medical History of the Infant:Pre-Existing Conditions

Time frame: Baseline

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.