Trial Outcomes & Findings for Study of the Effects of Fabrazyme Treatment on Lactation and Infants (NCT NCT00230607)
NCT ID: NCT00230607
Last Updated: 2024-04-10
Results Overview
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.
TERMINATED
PHASE4
7 participants
Month 1, 3, and 6
2024-04-10
Participant Flow
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.
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.
Participant milestones
| Measure |
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.
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|---|---|
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Overall Study
STARTED
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7
|
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Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
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.
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|---|---|
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Overall Study
Other
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3
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Baseline Characteristics
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: Month 1, 3, and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3, and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3, and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3 and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3 and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3 and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3 and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Month 1, 3 and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Month 2, 6, and 12Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Month 2, 6, and 12Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Months 2, 6, and 12Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Months 1, 2, 3, 4, and 6Population: Evaluable data was collected for 3 participants only; and thus, was not presented to protect participant confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrollment number (5 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Months 1, 2, 4, 6, 12, 18 and 24Population: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: At 1 minute and 5 minutes after birthPopulation: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Months 1, 2, 3, 6, 12, 18 and 24Population: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Months 1, 2, 3, 6, 12, 18 and 24Population: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Months 2, 6, and 12Population: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Months 2, 6, and 12Population: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: BaselinePopulation: Based on the low enrollment number (2 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24Population: Based on the low enrollment number (7 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24Population: Based on the low enrollment number (7 participants), no data is reported here in order to protect and maintain participant privacy/confidentiality.
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.
Outcome measures
Outcome data not reported
Adverse Events
Fabrazyme
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Trial Transparency Team
Sanofi aventis recherche & développement
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER