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Clinical trial

A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants

ID
Name
AGAL02603
Description
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.
Trial arms
Trial start
2006-05-28
Estimated PCD
2024-02-09
Trial end
2024-02-09
Status
Terminated
Phase
Early phase I
Treatment
agalsidase beta
Pharmaceutical form: powder for reconstitution Route of administration: intravenous
Arms:
Fabrazyme
Other names:
r-haGAL, Fabrazyme
Size
7
Primary endpoint
Concentration of Alpha-galactosidase (αGAL) in Plasma
Month 1, 3, and 6
Concentration of Alpha-galactosidase in Breast Milk
Month 1, 3, and 6
Pharmacokinetics: Observed Maximum Plasma Concentration (Cmax) of Alpha-galactosidase A
Month 1, 3, and 6
Pharmacokinetics: Observed Maximum Plasma Concentration of Alpha-galactosidase A in Breast Milk
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
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
Month 1, 3 and 6
Pharmacokinetics: Lactation Clearance of Alpha-galactosidase A
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
Month 1, 3 and 6
Total Volume of Breast Milk
Baseline, Month 2, 6, and 12
Total Fat Content in Breast Milk
Baseline, Month 2, 6, and 12
Total Protein Content in Breast Milk
Baseline, Months 2, 6, and 12
Lactation Status of Mothers at Baseline, Months 1, 2, 3, 4, and 6
Baseline, Months 1, 2, 3, 4, and 6
Medical History of Enrolled Mothers
Baseline
Genotype of the Enrolled Mothers
Baseline
Pregnancy Outcome
Baseline
Number of Mothers Who Were Seropositive for Anti-Fabrazyme Immunoglobulin G (IgG) Antibodies at Baseline
Baseline
Medical History of Infants at Baseline
Baseline
Number of Infant Participants With Abnormal Physical Examination
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
At 1 minute and 5 minutes after birth
Growth Response of Infants
Months 1, 2, 3, 6, 12, 18 and 24
Development Response of Infants
Months 1, 2, 3, 6, 12, 18 and 24
Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin G Antibodies
Baseline, Months 2, 6, and 12
Number of Infants Seropositive for Anti-Fabrazyme Immunoglobulin M Antibodies
Baseline, Months 2, 6, and 12
Genotype of the Infants
Baseline
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs)
From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
Number of Participants Who Received Concomitant Medications
From Baseline (Mother: within 1 month prior to delivery; Infant: at birth) up to Month 24
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 7, 'type': 'ACTUAL'}}
Updated at
2024-04-10

1 organization

1 product

2 indications

Organization
Genzyme
Product
Agalsidase beta
Indication
Fabry Disease
Indication
Alpha-galactosidase A deficiency