Clinical trial

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

ID

Name

OBS12874

Description

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.

Trial arms

Trial start

2010-10-22

Estimated PCD

2022-12-21

Trial end

2022-12-21

Status

Terminated

Phase

Early phase I

Treatment

Laronidase

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

Arms:

Laronidase

Other names:

Aldurazyme, Recombinant human alpha L iduronidase

Size

2

Primary endpoint

Number of Lactating Women With Serum IgG Antibodies to Laronidase

Up to 18 months

Amount of IgG Antibody Titers to Laronidase in Lactating Women

Baseline and Week 12

Number of Women Who Breastfed

Up to 18 months

Number of Women Who Were Successful at Breastfeeding

Up to 18 months

Number of Women Whose Breast Milk Contains Laronidase

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

Amount of uGAG in the Urine of Women

Baseline and Week 12

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

Baseline

Physical Examination Findings of the Mother

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 Mother

Baseline and Week 12

Blood Pressure of the Mother

Baseline and Week 12

Weight of the Mother

Baseline and Week 12

Height of the Mother

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

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

Baseline

Number of Infants With Abnormal Physical Finding

Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

Heart Rate of the Infant

Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

Respiratory Rate of the Infant

Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

Blood Pressure of the Infant

Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

Weight of the Infant

Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

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

Head Circumference of the Infant

Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

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

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

Time to Development of IgM and IgG Antibodies to Laronidase

up to Week 72

Amount of IgG and IgM Antibody Titers to Laronidase

Baseline, Week 12, Week 24, Week 36, and Week 72

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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 2, 'type': 'ACTUAL'}}

Updated at

2024-02-20

1 organization

1 product

4 indications

Organization

Product

Indication

Mucopolysaccharidosis Type I

Indication

Hurler syndrome

Indication

Mucopolysaccharidosis type I

Indication