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
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Number of Lactating Women With Serum IgG Antibodies to Laronidase
研究概览
简要总结
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.
详细描述
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •(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.
结局指标
主要结局
Number of Lactating Women With Serum IgG Antibodies to Laronidase
时间窗: Up to 18 months
Number of Women Who Were Successful at Breastfeeding
时间窗: Up to 18 months
Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
时间窗: Up to 18 months
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
时间窗: Up to 18 months
Physical Examination Findings of the Mother
时间窗: Up to 18 months
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.
Heart Rate of the Infant
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Heart rate was measured at specified timepoints.
Number of Women Who Breastfed
时间窗: Up to 18 months
Temperature of the Mother
时间窗: Baseline and Week 12
Respiratory Rate of the Mother
时间窗: Baseline and Week 12
Height of the Mother
时间窗: Baseline
Height of the Infant
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Amount of IgG Antibody Titers to Laronidase in Lactating Women
时间窗: Baseline and Week 12
Number of Women Whose Breast Milk Contains Laronidase
时间窗: Up to 18 months
Blood Pressure of the Mother
时间窗: Baseline and Week 12
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Number of Infants With Abnormal Physical Finding
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
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.
Weight of the Infant
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Head Circumference of the Infant
时间窗: Baseline, 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
时间窗: Baseline, Week 12, Week 24, Week 36, and Week 72
Amount of IgG and IgM Antibody Titers to Laronidase
时间窗: Baseline, Week 12, Week 24, Week 36, and Week 72
Amount of uGAG in the Urine of Women
时间窗: Baseline and Week 12
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.
Heart Rate of the Mother
时间窗: Baseline and Week 12
Weight of the Mother
时间窗: Baseline and Week 12
Blood Pressure of the Infant
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
时间窗: Week 12, Week 24, Week 36, Week 48, and Week 72
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.
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
时间窗: Baseline
Number of Infants With Abnormal uGAG Levels
时间窗: Up to 72 weeks
Amount of uGAG in the Urine of Infants
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
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.
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
时间窗: Baseline
Respiratory Rate of the Infant
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Respiratory rate was measured at specified timepoints.
Temperature of the Infant
时间窗: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Time to Development of IgM and IgG Antibodies to Laronidase
时间窗: up to Week 72
次要结局
未报告次要终点