Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2006-001910-33 | EudraCT Number | ||
| MSC12868 | Other Identifier | Sanofi |
Not provided
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Per FDA decision, the 2003 Post Marketing commitment has been fulfilled.
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The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full participation of mother and no infant participation was 6 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fabrazyme | Experimental | Mothers with Fabry disease who received commercially available Fabrazyme intravenous infusion as part of the standard of care while lactating/pregnant and their infants who were breastfed while the mothers were receiving Fabrazyme were enrolled in this study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| agalsidase beta | Drug | Pharmaceutical form: powder for reconstitution Route of administration: intravenous |
|
| Measure | Description | Time Frame |
|---|---|---|
| Concentration of Alpha-galactosidase (αGAL) in Plasma | Plasma concentration of αGAL was analyzed. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3, and 6 |
| Concentration of Alpha-galactosidase in Breast Milk | Concentration of αGAL in breast milk was analyzed. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3, and 6 |
| Pharmacokinetics: Observed Maximum Plasma Concentration (Cmax) of Alpha-galactosidase A | Cmax was defined as the maximum observed concentration in plasma. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3, and 6 |
| Pharmacokinetics: Observed Maximum Plasma Concentration of Alpha-galactosidase A in Breast Milk | Cmax was defined as the maximum observed concentration in breast milk. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3 and 6 |
| Pharmacokinetics: Area Under the Plasma Alpha-galactosidase A Concentration Versus Time Curve From Time Zero to Two Hours Post End of Infusion (AUC0-2plasma) of Fabrazyme | AUC0-2plasma was defined as the area under the plasma concentration versus time curve from time zero to 2 hours post end of infusion, calculated using the trapezoidal method. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3 and 6 |
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Inclusion Criteria:
Mothers that met the following criteria were enrolled in this study:
Infants that met the following criteria were enrolled in this study:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational Site Number 840005 | Decatur | Georgia | 30033 | United States | ||
| Investigational Site Number 840006 |
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Mothers/pregnant women enrolled in Fabry Registry, who had received Fabrazyme during lactation/pregnancy and infants born to mother who were receiving Fabrazyme during lactation period were enrolled in this study. This is a rare disease study, with very few participants enrolled. Based on the low enrollment number, the study was terminated by the Sponsor. Overall Arm has been reported in participant flow, Baseline and AE section to protect and maintain participant privacy/confidentiality.
The study was conducted at 5 active sites in 3 countries. A total of 7 participants were enrolled from 28 May 2006 to 04 March 2022 in this study.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Fabrazyme | Mothers with Fabry disease who received commercially available Fabrazyme intravenous infusion as part of the standard of care while lactating/pregnant, and their infants who were breastfed while the mothers were receiving Fabrazyme were enrolled in this study. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Fabrazyme | Mothers with Fabry disease who received commercially available Fabrazyme intravenous infusion as part of the standard of care while lactating/pregnant and their infants who were breastfed while the mothers were receiving Fabrazyme were enrolled in this study. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Concentration of Alpha-galactosidase (αGAL) in Plasma | Plasma concentration of αGAL was analyzed. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3, and 6 |
|
From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
Based on the low enrolment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Fabrazyme | Mothers with Fabry disease who received commercially available Fabrazyme intravenous infusion at the prescribed dose and regimen as part of the standard of care while lactating/pregnant as determined by their treating physician were enrolled in this study. Infants born to the enrolled mothers with Fabry disease who received Fabrazyme during lactation were also enrolled. |
Not provided
Not provided
This is a rare disease study, with very few participants enrolled. The study was terminated by the Sponsor. Based on the low enrolment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi aventis recherche & développement | 800-633-1610 | 6# | Contact-US@sanofi.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 23, 2018 | Mar 12, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 9, 2020 | Mar 12, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| C459420 | agalsidase beta |
Not provided
Not provided
Not provided
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| Pharmacokinetics: Area Under the Milk Alpha-galactosidase A Concentration Versus Time Curve From Time Zero to Two Hours Post End of Infusion (AUC0-2milk) of Fabrazyme | AUC0-2milk was defined as the area under the milk concentration versus time curve from time zero to 2 hours post-end of infusion, calculated using the trapezoidal method. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3 and 6 |
| Pharmacokinetics: Lactation Clearance of Alpha-galactosidase A | Lactation clearance was determined in the 0 to 2 hours interval, according to the following equation: the amount of αGAL excreted over the sampling period divided by the AUC during the sampling period. AUC was defined as area under the plasma αGAL concentration-time curve from time 0 to 2 hours post-end of infusion. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3 and 6 |
| Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to 2 Hours (AUC0-2): Milk to Plasma Ratio | AUC0-2milk was defined as the area under the milk concentration versus time curve from time zero to 2 hours post end of infusion, calculated using the trapezoidal method. AUC0-2plasma was defined as the area under the plasma concentration versus time curve from time zero to 2 hours post end of infusion, calculated using the trapezoidal method. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Month 1, 3 and 6 |
| Total Volume of Breast Milk | Collected breast milk samples were analyzed for total volume. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Month 2, 6, and 12 |
| Total Fat Content in Breast Milk | Collected breast milk samples were analyzed for total fat content. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Month 2, 6, and 12 |
| Total Protein Content in Breast Milk | Collected breast milk samples were analyzed for total protein content. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Months 2, 6, and 12 |
| Lactation Status of Mothers at Baseline, Months 1, 2, 3, 4, and 6 | Lactation status of mothers was assessed by asking them the question 'Was the infant breastfed?'. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Months 1, 2, 3, 4, and 6 |
| Medical History of Enrolled Mothers | Medical history of enrolled mothers collected from Fabry registry. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline |
| Genotype of the Enrolled Mothers | Genotype of each enrolled mother was collected from the Fabry registry. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline |
| Pregnancy Outcome | Pregnancy outcome of each enrolled mother collected from the Fabry registry. Baseline for mothers was defined as within 1 month prior to delivery. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline |
| Number of Mothers Who Were Seropositive for Anti-Fabrazyme Immunoglobulin G (IgG) Antibodies at Baseline | IgG antibodies data of the enrolled mothers were collected from the Fabry registry. Formation or continued presence of serum IgG antibodies to r-haGAL was considered seropositive. Baseline for mothers was defined as within 1 month prior to delivery. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline |
| Medical History of Infants at Baseline | Medical history of infants included birth difficulties (i.e., normal or caesarian birth). Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline |
| Number of Infant Participants With Abnormal Physical Examination | Physical examination of the infants included: length, weight, head circumference, vital signs, general appearance, skin, head, ears, eyes, nose, throat, lymph nodes, heart, lungs, abdomen, extremities/joints, neurological mental status and external genitalia. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Months 1, 2, 4, 6, 12, 18 and 24 |
| Appearance, Pulse, Grimace, Activity, and Respiration (APGAR) Score of Infants at 1 Minute and 5 Minutes After Birth | The Apgar score is a method to quickly summarize the health of newborn children. The Apgar score is determined by evaluating the newborn baby on five simple criteria: appearance (skin color), pulse (heart rate), grimace (reflex irritability), activity (muscle tone) and respiration on a scale from 0 to 2. Apgar total score (obtained by summing up values from all five items) ranges from 0 to 10 with a score of 0 expressing the worst neonatal status and a score of 10 the best status. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | At 1 minute and 5 minutes after birth |
| Growth Response of Infants | Effects of Fabrazyme on the growth response of infants born to mothers with Fabry disease, who had received fabrazyme during lactation. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Months 1, 2, 3, 6, 12, 18 and 24 |
| Development Response of Infants | Effects of Fabrazyme on the development response of infants born to mothers with Fabry disease, who had received fabrazyme during lactation. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Months 1, 2, 3, 6, 12, 18 and 24 |
| Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin G Antibodies | Formation or continued presence of serum IgG antibodies to r-haGAL was considered as seropositive. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Months 2, 6, and 12 |
| Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin M Antibodies | Formation or continued presence of serum IgM antibodies to r-haGAL was considered as seropositive. Blood samples of umbilical cord collected immediately after birth was used for the Baseline assessment. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline, Months 2, 6, and 12 |
| Genotype of the Infants | Genotype testing was conducted on umbilical cord blood to determine diagnosis of Fabry disease. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Baseline |
| Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs) | Adverse event (AE): any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. Serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24 |
| Number of Participants Who Received Concomitant Medications | Concomitant medication was any medication, prescription or over-the-counter, taken by a participant during their participation in an investigational study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24 |
| Fairfax |
| Virginia |
| 22030 |
| United States |
| investigational site number 01Rhead | Milwaukee | Wisconsin | 53201-1997 | United States |
| investigational site number 04Bodamer | Marl | Austria |
| investigational site number 03Waldek | Salford | M6 8HD | United Kingdom |
| Sex: Female, Male |
|
| Race (NIH/OMB) |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
| Primary | Concentration of Alpha-galactosidase in Breast Milk | Concentration of αGAL in breast milk was analyzed. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3, and 6 |
|
|
| Primary | Pharmacokinetics: Observed Maximum Plasma Concentration (Cmax) of Alpha-galactosidase A | Cmax was defined as the maximum observed concentration in plasma. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3, and 6 |
|
|
| Primary | Pharmacokinetics: Observed Maximum Plasma Concentration of Alpha-galactosidase A in Breast Milk | Cmax was defined as the maximum observed concentration in breast milk. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3 and 6 |
|
|
| Primary | Pharmacokinetics: Area Under the Plasma Alpha-galactosidase A Concentration Versus Time Curve From Time Zero to Two Hours Post End of Infusion (AUC0-2plasma) of Fabrazyme | AUC0-2plasma was defined as the area under the plasma concentration versus time curve from time zero to 2 hours post end of infusion, calculated using the trapezoidal method. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3 and 6 |
|
|
| Primary | Pharmacokinetics: Area Under the Milk Alpha-galactosidase A Concentration Versus Time Curve From Time Zero to Two Hours Post End of Infusion (AUC0-2milk) of Fabrazyme | AUC0-2milk was defined as the area under the milk concentration versus time curve from time zero to 2 hours post-end of infusion, calculated using the trapezoidal method. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3 and 6 |
|
|
| Primary | Pharmacokinetics: Lactation Clearance of Alpha-galactosidase A | Lactation clearance was determined in the 0 to 2 hours interval, according to the following equation: the amount of αGAL excreted over the sampling period divided by the AUC during the sampling period. AUC was defined as area under the plasma αGAL concentration-time curve from time 0 to 2 hours post-end of infusion. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3 and 6 |
|
|
| Primary | Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to 2 Hours (AUC0-2): Milk to Plasma Ratio | AUC0-2milk was defined as the area under the milk concentration versus time curve from time zero to 2 hours post end of infusion, calculated using the trapezoidal method. AUC0-2plasma was defined as the area under the plasma concentration versus time curve from time zero to 2 hours post end of infusion, calculated using the trapezoidal method. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Month 1, 3 and 6 |
|
|
| Primary | Total Volume of Breast Milk | Collected breast milk samples were analyzed for total volume. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Baseline, Month 2, 6, and 12 |
|
|
| Primary | Total Fat Content in Breast Milk | Collected breast milk samples were analyzed for total fat content. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Baseline, Month 2, 6, and 12 |
|
|
| Primary | Total Protein Content in Breast Milk | Collected breast milk samples were analyzed for total protein content. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Baseline, Months 2, 6, and 12 |
|
|
| Primary | Lactation Status of Mothers at Baseline, Months 1, 2, 3, 4, and 6 | Lactation status of mothers was assessed by asking them the question 'Was the infant breastfed?'. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality. | Posted | Baseline, Months 1, 2, 3, 4, and 6 |
|
|
| Primary | Medical History of Enrolled Mothers | Medical history of enrolled mothers collected from Fabry registry. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline |
|
|
| Primary | Genotype of the Enrolled Mothers | Genotype of each enrolled mother was collected from the Fabry registry. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline |
|
|
| Primary | Pregnancy Outcome | Pregnancy outcome of each enrolled mother collected from the Fabry registry. Baseline for mothers was defined as within 1 month prior to delivery. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline |
|
|
| Primary | Number of Mothers Who Were Seropositive for Anti-Fabrazyme Immunoglobulin G (IgG) Antibodies at Baseline | IgG antibodies data of the enrolled mothers were collected from the Fabry registry. Formation or continued presence of serum IgG antibodies to r-haGAL was considered seropositive. Baseline for mothers was defined as within 1 month prior to delivery. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline |
|
|
| Primary | Medical History of Infants at Baseline | Medical history of infants included birth difficulties (i.e., normal or caesarian birth). Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline |
|
|
| Primary | Number of Infant Participants With Abnormal Physical Examination | Physical examination of the infants included: length, weight, head circumference, vital signs, general appearance, skin, head, ears, eyes, nose, throat, lymph nodes, heart, lungs, abdomen, extremities/joints, neurological mental status and external genitalia. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline, Months 1, 2, 4, 6, 12, 18 and 24 |
|
|
| Primary | Appearance, Pulse, Grimace, Activity, and Respiration (APGAR) Score of Infants at 1 Minute and 5 Minutes After Birth | The Apgar score is a method to quickly summarize the health of newborn children. The Apgar score is determined by evaluating the newborn baby on five simple criteria: appearance (skin color), pulse (heart rate), grimace (reflex irritability), activity (muscle tone) and respiration on a scale from 0 to 2. Apgar total score (obtained by summing up values from all five items) ranges from 0 to 10 with a score of 0 expressing the worst neonatal status and a score of 10 the best status. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | At 1 minute and 5 minutes after birth |
|
|
| Primary | Growth Response of Infants | Effects of Fabrazyme on the growth response of infants born to mothers with Fabry disease, who had received fabrazyme during lactation. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Months 1, 2, 3, 6, 12, 18 and 24 |
|
|
| Primary | Development Response of Infants | Effects of Fabrazyme on the development response of infants born to mothers with Fabry disease, who had received fabrazyme during lactation. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Months 1, 2, 3, 6, 12, 18 and 24 |
|
|
| Primary | Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin G Antibodies | Formation or continued presence of serum IgG antibodies to r-haGAL was considered as seropositive. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline, Months 2, 6, and 12 |
|
|
| Primary | Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin M Antibodies | Formation or continued presence of serum IgM antibodies to r-haGAL was considered as seropositive. Blood samples of umbilical cord collected immediately after birth was used for the Baseline assessment. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline, Months 2, 6, and 12 |
|
|
| Primary | Genotype of the Infants | Genotype testing was conducted on umbilical cord blood to determine diagnosis of Fabry disease. Baseline for infants was at birth. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline |
|
|
| Primary | Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs) | Adverse event (AE): any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. Serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (7 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24 |
|
|
| Primary | Number of Participants Who Received Concomitant Medications | Concomitant medication was any medication, prescription or over-the-counter, taken by a participant during their participation in an investigational study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality. | Based on the low enrollment number (7 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality. | Posted | From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24 |
|
|
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |