Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie
Conditions
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.
Interventions
BIOLOGICALLaronidase
dose of 0.58mg/kg body weight intravenously (IV) every week
Sponsors
BioMarin/Genzyme LLC
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 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 | 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. |
| Number of Participants With Medical History of the Mother: Pre-Existing Conditions | Baseline | — |
| Physical Examination Findings of the Mother | 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. |
| 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 | Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints. |
| 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 | 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 | Baseline | — |
| Number of Infants With Abnormal Physical Finding | 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. |
| Heart Rate of the Infant | Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72 | Heart rate was measured at specified timepoints. |
| Respiratory Rate of the Infant | Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72 | Respiratory rate was measured at specified timepoints. |
| Blood Pressure of the Infant | 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. |
| Weight 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 | 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 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 | — |
Countries
Italy
Participant flow
Pre-assignment details
Study was conducted to fulfill a post marketing commitment (PMC). Upon acknowledgement of closure of PMC by FDA, the study was terminated by the Sponsor.
Participants by arm
| Arm | Count |
|---|---|
| Laronidase Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase and their infants who were breastfeed while the mothers were receiving laronidase. | 2 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Fulfils one of the exclusion criteria | 1 |
Baseline characteristics
| Characteristic | Laronidase |
|---|---|
| Age, Customized Adults (between 18 and 64 years) | 1 participants |
| Age, Customized Newborns (0-27 days) | 1 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 1 | 0 / 1 |
| other Total, other adverse events | 1 / 1 | 1 / 1 |
| serious Total, serious adverse events | 0 / 1 | 0 / 1 |
Outcome results
Primary
Amount of IgG and IgM Antibody Titers to Laronidase
Time frame: Baseline, Week 12, Week 24, Week 36, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase. No IgG antibody was detected at Week 12, Week 24, Week 36, and Week 72. No IgM was detected at Baseline, Week 12, Week 24, Week 36, and Week 72.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Amount of IgG and IgM Antibody Titers to Laronidase | IgG at Baseline | 6400 antibody titer |
Primary
Amount of IgG Antibody Titers to Laronidase in Lactating Women
Time frame: Baseline and Week 12
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Amount of IgG Antibody Titers to Laronidase in Lactating Women | Baseline | 25600 antibody titer |
| Laronidase | Amount of IgG Antibody Titers to Laronidase in Lactating Women | Week 12 | 51200 antibody titer |
Primary
Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
Time frame: Up to 18 months
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. There were no women whose breast milk contained laronidase hence overall number of participants analyzed is zero.
Primary
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
Population: Infants who were breastfeed while the mothers were receiving laronidase. No sample was collected at Week 60.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Amount of uGAG in the Urine of Infants | Baseline | 76 mg/g creatinine |
| Laronidase | Amount of uGAG in the Urine of Infants | Week 36 | 55 mg/g creatinine |
| Laronidase | Amount of uGAG in the Urine of Infants | Week 12 | 194 mg/g creatinine |
| Laronidase | Amount of uGAG in the Urine of Infants | Week 24 | 29 mg/g creatinine |
| Laronidase | Amount of uGAG in the Urine of Infants | Week 48 | 45 mg/g creatinine |
| Laronidase | Amount of uGAG in the Urine of Infants | Week 72 | 44 mg/g creatinine |
Primary
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
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Amount of uGAG in the Urine of Women | Baseline | 28.13 mg/g creatinine |
| Laronidase | Amount of uGAG in the Urine of Women | Week 12 | 26.98 mg/g creatinine |
Primary
Blood Pressure of the Infant
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase. Blood pressure was not measured at Baseline, Week 12, and Week 24.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Blood Pressure of the Infant | Week 72 DBP | 54 millimeters of mercury |
| Laronidase | Blood Pressure of the Infant | Week 36 SBP | 90 millimeters of mercury |
| Laronidase | Blood Pressure of the Infant | Week 48 SBP | 87 millimeters of mercury |
| Laronidase | Blood Pressure of the Infant | Week 72 SBP | 90 millimeters of mercury |
| Laronidase | Blood Pressure of the Infant | Week 36 DBP | 48 millimeters of mercury |
| Laronidase | Blood Pressure of the Infant | Week 48 DBP | 52 millimeters of mercury |
| Unknown | Blood Pressure of the Infant | Baseline SBP | — millimeters of mercury |
| Unknown | Blood Pressure of the Infant | Week 12 SBP | — millimeters of mercury |
| Unknown | Blood Pressure of the Infant | Week 24 SBP | — millimeters of mercury |
| Unknown | Blood Pressure of the Infant | Baseline DBP | — millimeters of mercury |
| Unknown | Blood Pressure of the Infant | Week 12 DBP | — millimeters of mercury |
| Unknown | Blood Pressure of the Infant | Week 24 DBP | — millimeters of mercury |
Primary
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
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Blood pressure was not measured at Week 12.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Blood Pressure of the Mother | SBP at Baseline | 125 millimeters of mercury |
| Laronidase | Blood Pressure of the Mother | DBP at Baseline | 80 millimeters of mercury |
Primary
Head Circumference of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Head Circumference of the Infant | Week 48 | 47 centimeter |
| Laronidase | Head Circumference of the Infant | Baseline | 33 centimeter |
| Laronidase | Head Circumference of the Infant | Week 12 | 39 centimeter |
| Laronidase | Head Circumference of the Infant | Week 24 | 42 centimeter |
| Laronidase | Head Circumference of the Infant | Week 36 | 43.2 centimeter |
| Laronidase | Head Circumference of the Infant | Week 72 | 45 centimeter |
Primary
Heart Rate of the Infant
Heart rate was measured at specified timepoints.
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase. Heart rate was not measured at Baseline and Week 24.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Heart Rate of the Infant | Week 12 | 121 beats per minute |
| Laronidase | Heart Rate of the Infant | Week 36 | 102 beats per minute |
| Laronidase | Heart Rate of the Infant | Week 48 | 120 beats per minute |
| Laronidase | Heart Rate of the Infant | Week 72 | 119 beats per minute |
| Unknown | Heart Rate of the Infant | Baseline | — beats per minute |
| Unknown | Heart Rate of the Infant | Week 24 | — beats per minute |
Primary
Heart Rate of the Mother
Time frame: Baseline and Week 12
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Heart rate was not measured at Week 12.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Heart Rate of the Mother | Baseline | 90 beats per minute |
Primary
Height of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Height of the Infant | Week 12 | 64.0 centimeter |
| Laronidase | Height of the Infant | Baseline | 50.0 centimeter |
| Laronidase | Height of the Infant | Week 24 | 66.5 centimeter |
| Laronidase | Height of the Infant | Week 36 | 72.0 centimeter |
| Laronidase | Height of the Infant | Week 48 | 76.5 centimeter |
| Laronidase | Height of the Infant | Week 72 | 78.0 centimeter |
Primary
Height of the Mother
Time frame: Baseline
Population: Based on the low enrolment numbers, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Primary
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.
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Laronidase | Number of Infants With Abnormal Physical Finding | Extremities/Joints at Baseline (clinodactily hands) | 1 participants |
| Laronidase | Number of Infants With Abnormal Physical Finding | Skin at Baseline (bilateral transverse palmar crease) | 1 participants |
| Laronidase | Number of Infants With Abnormal Physical Finding | Skin at Week 12 (bilateral transverse palmar crease) | 1 participants |
| Laronidase | Number of Infants With Abnormal Physical Finding | Skin at Week 24 (bilateral transverse palmar crease) | 1 participants |
| Laronidase | Number of Infants With Abnormal Physical Finding | Skin at Week 36 (bilateral transverse palmar crease almost disappeared) | 1 participants |
| Laronidase | Number of Infants With Abnormal Physical Finding | Lungs at Week 48 (grunting) | 1 participants |
| Laronidase | Number of Infants With Abnormal Physical Finding | Skin at Week 72 (bilateral transverse palmar crease) | 1 participants |
Primary
Number of Infants With Abnormal uGAG Levels
Time frame: Up to 72 weeks
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Laronidase | Number of Infants With Abnormal uGAG Levels | 1 Participants |
Primary
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
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgG at Baseline | 1 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgG at Week 12 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgG at Week 24 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgG at Week 36 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgG at Week 72 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgM at Baseline | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgM at Week 12 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgM at Week 24 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgM at Week 36 | 0 Participants |
| Laronidase | Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point | IgM at Week 72 | 0 Participants |
Primary
Number of Lactating Women With Serum IgG Antibodies to Laronidase
Time frame: Up to 18 months
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Laronidase | Number of Lactating Women With Serum IgG Antibodies to Laronidase | 1 Participants |
Primary
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
Time frame: Baseline
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Laronidase | Number of Participants With Medical History of the Infant:Pre-Existing Conditions | Extremities/Joints (clinodactily hands) | 1 Participants |
| Laronidase | Number of Participants With Medical History of the Infant:Pre-Existing Conditions | Skin (bilateral transverse palmar crease) | 1 Participants |
Primary
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Time frame: Baseline
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Laronidase | Number of Participants With Medical History of the Mother: Pre-Existing Conditions | Mucopolysaccharidosis type I | 1 Participants |
| Laronidase | Number of Participants With Medical History of the Mother: Pre-Existing Conditions | Aortic valvular insufficiency | 1 Participants |
| Laronidase | Number of Participants With Medical History of the Mother: Pre-Existing Conditions | Stipsi | 1 Participants |
| Laronidase | Number of Participants With Medical History of the Mother: Pre-Existing Conditions | Carpal tunnel syndrome | 1 Participants |
Primary
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
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.
Time frame: Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Laronidase | Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores | Week 48 | 1 Participants |
| Laronidase | Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores | Week 72 | 1 Participants |
| Laronidase | Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores | Week 12 | 1 Participants |
| Laronidase | Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores | Week 24 | 1 Participants |
| Laronidase | Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores | Week 36 | 1 Participants |
Primary
Number of Women Who Breastfed
Time frame: Up to 18 months
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Laronidase | Number of Women Who Breastfed | 1 Participants |
Primary
Number of Women Whose Breast Milk Contains Laronidase
Time frame: Up to 18 months
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Laronidase | Number of Women Whose Breast Milk Contains Laronidase | 0 Participants |
Primary
Number of Women Who Were Successful at Breastfeeding
Time frame: Up to 18 months
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Laronidase | Number of Women Who Were Successful at Breastfeeding | 1 Participants |
Primary
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
Time frame: Up to 18 months
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Laronidase | Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels | 0 Participants |
Primary
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.
Time frame: Up to 18 months
Population: Based on the low enrolment numbers, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Primary
Respiratory Rate of the Infant
Respiratory rate was measured at specified timepoints.
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase. Respiratory rate was not measured at Baseline and Week 24.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Respiratory Rate of the Infant | Week 12 | 18 breaths/min |
| Laronidase | Respiratory Rate of the Infant | Week 36 | 13 breaths/min |
| Laronidase | Respiratory Rate of the Infant | Week 48 | 16 breaths/min |
| Laronidase | Respiratory Rate of the Infant | Week 72 | 23 breaths/min |
| Unknown | Respiratory Rate of the Infant | Baseline | — breaths/min |
| Unknown | Respiratory Rate of the Infant | Week 24 | — breaths/min |
Primary
Respiratory Rate of the Mother
Time frame: Baseline and Week 12
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Respiratory rate was not measured at Baseline and Week 12.
| Arm | Measure | Group | Value |
|---|---|---|---|
| Unknown | Respiratory Rate of the Mother | Baseline | — |
| Unknown | Respiratory Rate of the Mother | Week 12 | — |
Primary
Temperature of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase. Temperature was not measured at Baseline and Week 12.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Temperature of the Infant | Week 24 | 36.5 Celcius |
| Laronidase | Temperature of the Infant | Week 36 | 36.2 Celcius |
| Laronidase | Temperature of the Infant | Week 48 | 35.9 Celcius |
| Laronidase | Temperature of the Infant | Week 72 | 36.3 Celcius |
| Unknown | Temperature of the Infant | Baseline | — Celcius |
| Unknown | Temperature of the Infant | Week 12 | — Celcius |
Primary
Temperature of the Mother
Time frame: Baseline and Week 12
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Temperature was not measured at Week 12.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Temperature of the Mother | Baseline | 35.8 Celsius |
Primary
Time to Development of IgM and IgG Antibodies to Laronidase
Time frame: up to Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase. No IgM and IgG antibody was detected during the study, hence time is not-evaluable.
Primary
Weight of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Population: Infants who were breastfeed while the mothers were receiving laronidase.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Weight of the Infant | Baseline | 2.5 kilograms |
| Laronidase | Weight of the Infant | Week 12 | 5.8 kilograms |
| Laronidase | Weight of the Infant | Week 24 | 7.8 kilograms |
| Laronidase | Weight of the Infant | Week 36 | 9.2 kilograms |
| Laronidase | Weight of the Infant | Week 48 | 9.9 kilograms |
| Laronidase | Weight of the Infant | Week 72 | 10.8 kilograms |
Primary
Weight of the Mother
Time frame: Baseline and Week 12
Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Laronidase | Weight of the Mother | Baseline | 58.5 kilograms |
| Laronidase | Weight of the Mother | Week 12 | 51 kilograms |