Clinical trial NCT00418821: 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: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: BioMarin/Genzyme LLC
First posted: 2007-01-05
Enrollment: 2
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of Lactating Women With Serum IgG Antibodies to Laronidase
timeFrame: Up to 18 months
measure: Amount of IgG Antibody Titers to Laronidase in Lactating Women
timeFrame: Baseline and Week 12
measure: Number of Women Who Breastfed
timeFrame: Up to 18 months
measure: Number of Women Who Were Successful at Breastfeeding
timeFrame: Up to 18 months
measure: Number of Women Whose Breast Milk Contains Laronidase
timeFrame: Up to 18 months
measure: Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
timeFrame: Up to 18 months
measure: Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
timeFrame: Up to 18 months
measure: Amount of uGAG in the Urine of Women
timeFrame: Baseline and Week 12
measure: Number of Participants With Medical History of the Mother: Pre-Existing Conditions
timeFrame: Baseline
measure: Physical Examination Findings of the Mother
timeFrame: Up to 18 months
measure: Temperature of the Mother
timeFrame: Baseline and Week 12
measure: Heart Rate of the Mother
timeFrame: Baseline and Week 12
measure: Respiratory Rate of the Mother
timeFrame: Baseline and Week 12
measure: Blood Pressure of the Mother
timeFrame: Baseline and Week 12
measure: Weight of the Mother
timeFrame: Baseline and Week 12
measure: Height of the Mother
timeFrame: Baseline
measure: Number of Infants With Abnormal uGAG Levels
timeFrame: Up to 72 weeks
measure: Amount of uGAG in the Urine of Infants
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
measure: Number of Participants With Medical History of the Infant:Pre-Existing Conditions
timeFrame: Baseline
measure: Number of Infants With Abnormal Physical Finding
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Heart Rate of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Respiratory Rate of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Blood Pressure of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Weight of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Height of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Temperature of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Head Circumference of the Infant
timeFrame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
timeFrame: Week 12, Week 24, Week 36, Week 48, and Week 72
measure: Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
timeFrame: Baseline, Week 12, Week 24, Week 36, and Week 72
measure: Time to Development of IgM and IgG Antibodies to Laronidase
timeFrame: up to Week 72
measure: Amount of IgG and IgM Antibody Titers to Laronidase
timeFrame: Baseline, Week 12, Week 24, Week 36, and Week 72
Secondary outcomes:
Other outcomes:
Locations:
city: Rome
country: Italy
facility: Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References:
citation: 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.