Clinical trial NCT00230607: Study of the Effects of Fabrazyme Treatment on Lactation and Infants
Official title: A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Fabry Disease
Alpha Galactosidase A Deficiency
Keywords: alpha Galactosidase A
aGAL
r-haGAL
Fabry
GL3
Fabrazyme
Lysosomal Storage Disorder
Enzyme Replacement Therapy (ERT)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2005-10-03
Enrollment: 7
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

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:

* 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.

Exclusion Criteria:

* The mother and infant were excluded from this study if the mother received an investigational drug within 30 days prior to study enrollment.

Primary outcomes:
measure: Concentration of Alpha-galactosidase (αGAL) in Plasma
timeFrame: Month 1, 3, and 6
measure: Concentration of Alpha-galactosidase in Breast Milk
timeFrame: Month 1, 3, and 6
measure: Pharmacokinetics: Observed Maximum Plasma Concentration (Cmax) of Alpha-galactosidase A
timeFrame: Month 1, 3, and 6
measure: Pharmacokinetics: Observed Maximum Plasma Concentration of Alpha-galactosidase A in Breast Milk
timeFrame: Month 1, 3 and 6
measure: 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
timeFrame: Month 1, 3 and 6
measure: 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
timeFrame: Month 1, 3 and 6
measure: Pharmacokinetics: Lactation Clearance of Alpha-galactosidase A
timeFrame: Month 1, 3 and 6
measure: Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to 2 Hours (AUC0-2): Milk to Plasma Ratio
timeFrame: Month 1, 3 and 6
measure: Total Volume of Breast Milk
timeFrame: Baseline, Month 2, 6, and 12
measure: Total Fat Content in Breast Milk
timeFrame: Baseline, Month 2, 6, and 12
measure: Total Protein Content in Breast Milk
timeFrame: Baseline, Months 2, 6, and 12
measure: Lactation Status of Mothers at Baseline, Months 1, 2, 3, 4, and 6
timeFrame: Baseline, Months 1, 2, 3, 4, and 6
measure: Medical History of Enrolled Mothers
timeFrame: Baseline
measure: Genotype of the Enrolled Mothers
timeFrame: Baseline
measure: Pregnancy Outcome
timeFrame: Baseline
measure: Number of Mothers Who Were Seropositive for Anti-Fabrazyme Immunoglobulin G (IgG) Antibodies at Baseline
timeFrame: Baseline
measure: Medical History of Infants at Baseline
timeFrame: Baseline
measure: Number of Infant Participants With Abnormal Physical Examination
timeFrame: Baseline, Months 1, 2, 4, 6, 12, 18 and 24
measure: Appearance, Pulse, Grimace, Activity, and Respiration (APGAR) Score of Infants at 1 Minute and 5 Minutes After Birth
timeFrame: At 1 minute and 5 minutes after birth
measure: Growth Response of Infants
timeFrame: Months 1, 2, 3, 6, 12, 18 and 24
measure: Development Response of Infants
timeFrame: Months 1, 2, 3, 6, 12, 18 and 24
measure: Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin G Antibodies
timeFrame: Baseline, Months 2, 6, and 12
measure: Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin M Antibodies
timeFrame: Baseline, Months 2, 6, and 12
measure: Genotype of the Infants
timeFrame: Baseline
measure: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs)
timeFrame: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
measure: Number of Participants Who Received Concomitant Medications
timeFrame: From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
Secondary outcomes:
Other outcomes:
Locations:
city: Decatur
country: United States
facility: Investigational Site Number 840005
state: Georgia
city: Fairfax
country: United States
facility: Investigational Site Number 840006
state: Virginia
city: Milwaukee
country: United States
facility: investigational site number 01Rhead
state: Wisconsin
city: Marl
country: Austria
facility: investigational site number 04Bodamer
city: Salford
country: United Kingdom
facility: investigational site number 03Waldek
Study officials:
References: