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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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27 February 2024 |
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Main ID: |
NCT00418821 |
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Date of registration:
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03/01/2007 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
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Scientific title:
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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 |
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Date of first enrolment:
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October 22, 2010 |
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Target sample size:
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2 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT00418821 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 4
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Countries of recruitment
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Italy
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United States
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Contacts
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Name:
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Clinical Sciences & Operations |
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Address:
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Telephone:
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Email:
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Affiliation:
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Sanofi |
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Key inclusion & exclusion criteria
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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.
Age minimum:
N/A
Age maximum:
N/A
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis I
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Hurler's Syndrome
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Hurler-Scheie Syndrome
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Scheie
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Intervention(s)
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Biological: Laronidase
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Primary Outcome(s)
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Height of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Number of Women Whose Breast Milk Contains Laronidase
[Time Frame: Up to 18 months]
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Height of the Mother
[Time Frame: Baseline]
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Number of Infants With Abnormal Physical Finding
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
[Time Frame: Baseline, Week 12, Week 24, Week 36, and Week 72]
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Number of Participants With Medical History of the Infant:Pre-Existing Conditions
[Time Frame: Baseline]
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Number of Infants With Abnormal uGAG Levels
[Time Frame: Up to 72 weeks]
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Number of Lactating Women With Serum IgG Antibodies to Laronidase
[Time Frame: Up to 18 months]
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Weight of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
[Time Frame: Week 12, Week 24, Week 36, Week 48, and Week 72]
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Amount of IgG and IgM Antibody Titers to Laronidase
[Time Frame: Baseline, Week 12, Week 24, Week 36, and Week 72]
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Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
[Time Frame: Up to 18 months]
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Amount of IgG Antibody Titers to Laronidase in Lactating Women
[Time Frame: Baseline and Week 12]
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Blood Pressure of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Number of Women Who Were Successful at Breastfeeding
[Time Frame: Up to 18 months]
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Blood Pressure of the Mother
[Time Frame: Baseline and Week 12]
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Number of Women Who Breastfed
[Time Frame: Up to 18 months]
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Heart Rate of the Mother
[Time Frame: Baseline and Week 12]
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Temperature of the Mother
[Time Frame: Baseline and Week 12]
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Respiratory Rate of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Temperature of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Physical Examination Findings of the Mother
[Time Frame: Up to 18 months]
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Amount of uGAG in the Urine of Women
[Time Frame: Baseline and Week 12]
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Time to Development of IgM and IgG Antibodies to Laronidase
[Time Frame: up to Week 72]
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Weight of the Mother
[Time Frame: Baseline and Week 12]
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Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
[Time Frame: Up to 18 months]
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Amount of uGAG in the Urine of Infants
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72]
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Head Circumference of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Heart Rate of the Infant
[Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72]
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Number of Participants With Medical History of the Mother: Pre-Existing Conditions
[Time Frame: Baseline]
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Respiratory Rate of the Mother
[Time Frame: Baseline and Week 12]
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Secondary ID(s)
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ALID01803
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2007-007003-33
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OBS12874
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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