Fabry disease MedDRA version: 14.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Fabrazyme
Product Name: Fabrazyme
Product Code: Agalsidase beta
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: agalsidase beta
CAS Number: 10413
Sponsors
Genzyme Europe BV
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Patients who meet the following inclusion criteria will be eligible for enrollment in this study: 1. The patient and/or patient’s parent(s)/legal guardian(s) must provide written informed consent prior to any protocol-related procedures being performed. 2. The patient must have a confirmed diagnosis of Fabry disease as documented by leukocyte a-Galactosidase A (alpha-GAL) activity of 7.0 microg/mL) and/or urinary GL-3 (>0.03 mg GL-3/mmol creatinine) levels (by central analysis laboratories). 4. The patient must be male =5 and =18 years of age. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients who meet any of the following exclusion criteria will not be eligible for enrollment in this study: 1. Patient has albuminuria (first morning void urinary albumin/creatinine ratio >30 mg/g on at least 2 out of 3 consecutive samples, each at least 1 week apart. 2. Patient has a GFRiohexol 2 mm on T2- or fluid attenuated inversion recovery (FLAIR)-weighted images within the white matter or the basal ganglia. 4. Patient has severe and recurrent acroparesthesia, judged by the physician as frequent (more than once a week) pain episodes for at least 3 months that influence daily activities, irrespective of medication. 5. Patient has an end-diastolic left ventricular posterior wall thickness (LVPWTd) and/or an end-diastolic interventricular septum thickness (IVSTd) =2 standard deviations (SD) compared to normal based on body surface area (BSA) normal ranges as read at the study site. 6. Patient has received prior treatment specific to Fabry Disease. 7. Patient has participated in a study employing an investigational drug within 30 days of the start of their participation in this study. 8. Patient has any medical condition or extenuating circumstance, which, in the opinion of the Study Investigator, could interfere with study compliance. 9. Patient has any medical condition or extenuating circumstance, (eg, diabetes mellitus) which, in the opinion of the Study Investigator, could interfere with the interpretation of study results. 10. Patient is on treatment with angiotensin converting enzyme inhibitors/angiotensin receptor blockers (ACEIs/ARBs). 11. The presence of any contra-indication mentioned in the labeling of Fabrazyme and/or iohexol (Omnipaque). 12. Patient or parent(s)/legal guardian(s) is unwilling to comply with the requirements of the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg every 4 weeks [q4w]) in treatment-naive male pediatric patients (5 years to 18 years of age) with Fabry disease without severe symptoms.;Secondary Objective: ;Primary end point(s): Primary Efficacy Endpoint: The primary efficacy endpoint will be histological evaluation of GL-3 inclusions in the superficial skin vascular endothelium conducted using light microscopy (LM) histochemistry during Screening or Day 1, Week 52/Year 1, Week 156/Year 3, and Week 260/Year 5. Secondary Efficacy Endpoints: The secondary efficacy endpoints will be the effect of Fabrazyme treatment on GL-3 clearance in plasma and urine collection measured at Screening and every 3 months for the first year (through Week 52/Year 1) and every 6 months thereafter. ;Timepoint(s) of evaluation of this end point: During Screening or Day 1, Week 52/Year 1, Week 156/Year 3, and Week 260/Year 5. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy Endpoints: The secondary efficacy endpoints will be the effect of Fabrazyme treatment on GL-3 clearance in plasma and urine collection.;Timepoint(s) of evaluation of this end point: At Screening and every 3 months for the first year (through Week 52/Year 1) and every 6 months thereafter. | — |
Countries
Argentina, Brazil, Canada, Czech Republic, France, Germany, Netherlands, Norway, Poland, Portugal, United Kingdom, United States
Contacts
Public ContactMedical Information Genzyme Europe
Genzyme Europe B.V.
Outcome results
None listed