Trial Outcomes & Findings for A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants (NCT NCT00418821)
NCT ID: NCT00418821
Last Updated: 2024-02-20
Results Overview
TERMINATED
PHASE4
2 participants
Baseline, Week 12, Week 24, Week 36, and Week 72
2024-02-20
Participant Flow
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.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
Fulfils one of the exclusion criteria
|
1
|
Baseline Characteristics
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Baseline characteristics by cohort
| Measure |
Laronidase
n=2 Participants
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.
|
|---|---|
|
Age, Customized
Newborns (0-27 days)
|
1 participants
n=2 Participants
|
|
Age, Customized
Adults (between 18 and 64 years)
|
1 participants
n=2 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=2 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=2 Participants
|
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Lactating Women With Serum IgG Antibodies to Laronidase
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Amount of IgG Antibody Titers to Laronidase in Lactating Women
Baseline
|
25600 antibody titer
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of IgG Antibody Titers to Laronidase in Lactating Women
Week 12
|
51200 antibody titer
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Women Who Breastfed
|
1 Participants
|
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Women Who Were Successful at Breastfeeding
|
1 Participants
|
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Women Whose Breast Milk Contains Laronidase
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: 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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
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.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Amount of uGAG in the Urine of Women
Baseline
|
28.13 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of uGAG in the Urine of Women
Week 12
|
26.98 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: BaselinePopulation: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Mucopolysaccharidosis type I
|
1 Participants
|
|
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Aortic valvular insufficiency
|
1 Participants
|
|
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Stipsi
|
1 Participants
|
|
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Carpal tunnel syndrome
|
1 Participants
|
PRIMARY outcome
Timeframe: Up to 18 monthsPopulation: Based on the low enrolment numbers, no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Temperature was not measured at Week 12.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Temperature of the Mother
Baseline
|
35.8 Celsius
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Heart rate was not measured at Week 12.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Heart Rate of the Mother
Baseline
|
90 beats per minute
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Respiratory rate was not measured at Baseline and Week 12.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Blood pressure was not measured at Week 12.
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Blood Pressure of the Mother
SBP at Baseline
|
125 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Blood Pressure of the Mother
DBP at Baseline
|
80 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Weight of the Mother
Baseline
|
58.5 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Weight of the Mother
Week 12
|
51 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrolment numbers, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Up to 72 weeksPopulation: Infants who were breastfeed while the mothers were receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Infants With Abnormal uGAG Levels
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase. No sample was collected at Week 60.
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.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Amount of uGAG in the Urine of Infants
Baseline
|
76 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of uGAG in the Urine of Infants
Week 12
|
194 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of uGAG in the Urine of Infants
Week 24
|
29 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of uGAG in the Urine of Infants
Week 36
|
55 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of uGAG in the Urine of Infants
Week 48
|
45 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Amount of uGAG in the Urine of Infants
Week 72
|
44 mg/g creatinine
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: BaselinePopulation: Infants who were breastfeed while the mothers were receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
Extremities/Joints (clinodactily hands)
|
1 Participants
|
|
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
Skin (bilateral transverse palmar crease)
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase.
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.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Infants With Abnormal Physical Finding
Extremities/Joints at Baseline (clinodactily hands)
|
1 participants
|
|
Number of Infants With Abnormal Physical Finding
Skin at Baseline (bilateral transverse palmar crease)
|
1 participants
|
|
Number of Infants With Abnormal Physical Finding
Skin at Week 12 (bilateral transverse palmar crease)
|
1 participants
|
|
Number of Infants With Abnormal Physical Finding
Skin at Week 24 (bilateral transverse palmar crease)
|
1 participants
|
|
Number of Infants With Abnormal Physical Finding
Skin at Week 36 (bilateral transverse palmar crease almost disappeared)
|
1 participants
|
|
Number of Infants With Abnormal Physical Finding
Lungs at Week 48 (grunting)
|
1 participants
|
|
Number of Infants With Abnormal Physical Finding
Skin at Week 72 (bilateral transverse palmar crease)
|
1 participants
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase. Heart rate was not measured at Baseline and Week 24.
Heart rate was measured at specified timepoints.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Heart Rate of the Infant
Week 12
|
121 beats per minute
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Heart Rate of the Infant
Week 36
|
102 beats per minute
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Heart Rate of the Infant
Week 48
|
120 beats per minute
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Heart Rate of the Infant
Week 72
|
119 beats per minute
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase. Respiratory rate was not measured at Baseline and Week 24.
Respiratory rate was measured at specified timepoints.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Respiratory Rate of the Infant
Week 12
|
18 breaths/min
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Respiratory Rate of the Infant
Week 36
|
13 breaths/min
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Respiratory Rate of the Infant
Week 48
|
16 breaths/min
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Respiratory Rate of the Infant
Week 72
|
23 breaths/min
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase. Blood pressure was not measured at Baseline, Week 12, and Week 24.
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Blood Pressure of the Infant
Week 36 DBP
|
48 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Blood Pressure of the Infant
Week 36 SBP
|
90 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Blood Pressure of the Infant
Week 48 SBP
|
87 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Blood Pressure of the Infant
Week 72 SBP
|
90 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Blood Pressure of the Infant
Week 48 DBP
|
52 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Blood Pressure of the Infant
Week 72 DBP
|
54 millimeters of mercury
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Weight of the Infant
Baseline
|
2.5 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Weight of the Infant
Week 12
|
5.8 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Weight of the Infant
Week 24
|
7.8 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Weight of the Infant
Week 36
|
9.2 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Weight of the Infant
Week 48
|
9.9 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Weight of the Infant
Week 72
|
10.8 kilograms
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Height of the Infant
Baseline
|
50.0 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Height of the Infant
Week 12
|
64.0 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Height of the Infant
Week 24
|
66.5 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Height of the Infant
Week 36
|
72.0 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Height of the Infant
Week 48
|
76.5 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Height of the Infant
Week 72
|
78.0 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase. Temperature was not measured at Baseline and Week 12.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Temperature of the Infant
Week 24
|
36.5 Celcius
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Temperature of the Infant
Week 36
|
36.2 Celcius
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Temperature of the Infant
Week 48
|
35.9 Celcius
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Temperature of the Infant
Week 72
|
36.3 Celcius
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Head Circumference of the Infant
Baseline
|
33 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Head Circumference of the Infant
Week 12
|
39 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Head Circumference of the Infant
Week 24
|
42 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Head Circumference of the Infant
Week 36
|
43.2 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Head Circumference of the Infant
Week 48
|
47 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
|
Head Circumference of the Infant
Week 72
|
45 centimeter
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
PRIMARY outcome
Timeframe: Week 12, Week 24, Week 36, Week 48, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase.
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.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Week 12
|
1 Participants
|
|
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Week 24
|
1 Participants
|
|
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Week 36
|
1 Participants
|
|
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Week 48
|
1 Participants
|
|
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Week 72
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, and Week 72Population: Infants who were breastfeed while the mothers were receiving laronidase.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgG at Baseline
|
1 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgG at Week 12
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgG at Week 24
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgG at Week 36
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgG at Week 72
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgM at Baseline
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgM at Week 12
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgM at Week 24
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgM at Week 36
|
0 Participants
|
|
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
IgM at Week 72
|
0 Participants
|
PRIMARY outcome
Timeframe: up to Week 72Population: 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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Week 12, Week 24, Week 36, and Week 72Population: 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.
Outcome measures
| Measure |
Laronidase
n=1 Participants
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase. Mothers received commercially available laronidase as per the country labeling information at the study site.
|
|---|---|
|
Amount of IgG and IgM Antibody Titers to Laronidase
IgG at Baseline
|
6400 antibody titer
Standard Deviation NA
Standard deviation cannot be derived for a single participant.
|
Adverse Events
Mothers Treated With Laronidase
Infants Who Were Breastfeed
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Mothers Treated With Laronidase
n=1 participants at risk
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.
|
Infants Who Were Breastfeed
n=1 participants at risk
Infants who were breastfeed while the mothers were receiving laronidase.
|
|---|---|---|
|
Cardiac disorders
Tachycardia
|
100.0%
1/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
0.00%
0/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
|
Infections and infestations
Influenza
|
100.0%
1/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
0.00%
0/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
|
Psychiatric disorders
Perinatal depression
|
100.0%
1/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
0.00%
0/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
100.0%
1/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
0.00%
0/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
100.0%
1/1 • Up to approximately 18 months for mother and 72 weeks for the infant.
|
Additional Information
Trial Transparency Team
Sanofi aventis recherche & développement
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER