Fabry DiseasePhase 4NCT00230607
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
A Phase 4 study testing agalsidase beta in people with Fabry Disease and Alpha Galactosidase A Deficiency, run by Genzyme, a Sanofi Company at 5 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fabry Disease, Alpha Galactosidase A Deficiency
- What is being tested?
- agalsidase beta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesin 3 countries
- Study dates
- 28 May 2006to 9 Feb 2024
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Mothers that met the following criteria were enrolled in this study:
- provided signed written informed consent to participate in this study,
- be enrolled in the Fabry Registry and received Fabrazyme while lactating,
- agreed to adhere to the Fabry Registry recommended schedule of assessments for medical history, pregnancy outcome, genotyping, and antibody testing, and
- agreed to adhere to the schedule of evaluations for this study.
- Infants that met the following criteria were enrolled in this study:
Show all 10Show fewer
- had the signed written informed consent of the parent(s)/legal guardian(s) to participate in this study,
- born to a mother who was receiving Fabrazyme during lactation,
- received breast milk from the mother, and
- had the agreement of the parent(s)/legal guardian(s) to adhere to the schedule of evaluations for this study.
Who cannot join
- The mother and infant were excluded from this study if the mother received an investigational drug within 30 days prior to study enrollment.
Trial locations and contacts
5 sites in 3 countries.
5 sites in 3 countries
United States3 sites
Investigational Site Number 840005
Decatur, Georgia, 30033
Investigational Site Number 840006
Fairfax, Virginia, 22030
investigational site number 01Rhead
Milwaukee, Wisconsin, 53201-1997
United Kingdom1 site
investigational site number 03Waldek
Salford, M6 8HD
About this study
Official title: A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants
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.
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Being tested
- agalsidase beta
- Keywords
- alpha Galactosidase A, aGAL, r-haGAL, Fabry, GL3, Fabrazyme, Lysosomal Storage Disorder, Enzyme Replacement Therapy (ERT)
- Medical subject headings
- Fabry Disease, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
agalsidase betaDrug
Also called r-haGAL, Fabrazyme
Pharmaceutical form: powder for reconstitution Route of administration: intravenous
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: agalsidase beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Month 1, 3 and 6
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2005 (estimated)
- Study start28 May 2006
- Primary completion9 Feb 2024
- Study completion9 Feb 2024
Why it stopped: Per FDA decision, the 2003 Post Marketing commitment has been fulfilled.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00230607
- Sponsor's protocol ID
- AGAL02603
- Other IDs
- 2006-001910-33, MSC12868
- Status last verified
- Mar 2024
- First submitted
- 29 Sep 2005
- Last updated
- 10 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants
- ClinicalTrials.gov ID
- NCT00230607
- Acronym / sponsor ID
- AGAL02603
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Keywords
- alpha Galactosidase A, aGAL, r-haGAL, Fabry, GL3, Fabrazyme, Lysosomal Storage Disorder, Enzyme Replacement Therapy (ERT)
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Per FDA decision, the 2003 Post Marketing commitment has been fulfilled.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 28 May 2006 (actual)
- Primary completion
- 9 Feb 2024 (actual)
- Study completion
- 9 Feb 2024 (actual)
- First posted
- 3 Oct 2005 (estimated)
- Results
- Posted 10 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fabrazyme | Experimental | Drug: agalsidase betaMothers 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- provided signed written informed consent to participate in this study,
- be enrolled in the Fabry Registry and received Fabrazyme while lactating,
- agreed to adhere to the Fabry Registry recommended schedule of assessments for medical history, pregnancy outcome, genotyping, and antibody testing, and
- agreed to adhere to the schedule of evaluations for this study.
- had the signed written informed consent of the parent(s)/legal guardian(s) to participate in this study,
Key exclusion criteria (1 listed)
- The mother and infant were excluded from this study if the mother received an investigational drug within 30 days prior to study enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| agalsidase betaAlso: r-haGAL, FabrazymePharmaceutical form: powder for reconstitution Route of administration: intravenous | Drug | Fabrazyme |
Primary outcome
Concentration of Alpha-galactosidase (αGAL) in Plasma
Time frame: Month 1, 3, and 6
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.
Concentration of Alpha-galactosidase in Breast Milk
Time frame: Month 1, 3, and 6
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.
Pharmacokinetics: Observed Maximum Plasma Concentration (Cmax) of Alpha-galactosidase A
Time frame: Month 1, 3, and 6
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.
Pharmacokinetics: Observed Maximum Plasma Concentration of Alpha-galactosidase A in Breast Milk
Time frame: Month 1, 3 and 6
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.
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
Time frame: Month 1, 3 and 6
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.
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
Time frame: Month 1, 3 and 6
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.
Pharmacokinetics: Lactation Clearance of Alpha-galactosidase A
Time frame: Month 1, 3 and 6
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.
Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to 2 Hours (AUC0-2): Milk to Plasma Ratio
Time frame: Month 1, 3 and 6
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.
Total Volume of Breast Milk
Time frame: Baseline, Month 2, 6, and 12
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.
Total Fat Content in Breast Milk
Time frame: Baseline, Month 2, 6, and 12
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.
Total Protein Content in Breast Milk
Time frame: Baseline, Months 2, 6, and 12
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.
Lactation Status of Mothers at Baseline, Months 1, 2, 3, 4, and 6
Time frame: 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.
Medical History of Enrolled Mothers
Time frame: Baseline
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.
Genotype of the Enrolled Mothers
Time frame: Baseline
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.
Pregnancy Outcome
Time frame: Baseline
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.
Number of Mothers Who Were Seropositive for Anti-Fabrazyme Immunoglobulin G (IgG) Antibodies at Baseline
Time frame: 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.
Medical History of Infants at Baseline
Time frame: 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.
Number of Infant Participants With Abnormal Physical Examination
Time frame: Baseline, Months 1, 2, 4, 6, 12, 18 and 24
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.
Appearance, Pulse, Grimace, Activity, and Respiration (APGAR) Score of Infants at 1 Minute and 5 Minutes After Birth
Time frame: 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.
Growth Response of Infants
Time frame: Months 1, 2, 3, 6, 12, 18 and 24
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.
Development Response of Infants
Time frame: Months 1, 2, 3, 6, 12, 18 and 24
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.
Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin G Antibodies
Time frame: Baseline, Months 2, 6, and 12
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.
Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin M Antibodies
Time frame: Baseline, Months 2, 6, and 12
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.
Genotype of the Infants
Time frame: Baseline
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.
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs)
Time frame: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
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.
Number of Participants Who Received Concomitant Medications
Time frame: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing agalsidase beta in people with Fabry Disease and Alpha Galactosidase A Deficiency, run by Genzyme, a Sanofi Company at 5 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 10 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Austria, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Concentration of Alpha-galactosidase (αGAL) in Plasma (time frame: Month 1, 3, and 6). The study lists 26 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2024.