Phase 3
Completed N=31
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
Source: ClinicalTrials.gov NCT00701415 ↗Enrolled (actual)
31
Serious AEs
35.5%
Results posted
Jun 2016
Primary outcomePrimary: Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium — 18.8; 33.3; 75; 80 Percentage of participants
◆ Published Evidence
Highly cited
167citations · ~13 / year
Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.
Summary
The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without severe symptoms. Participants were to be treated for 5 years.
Linked Publications (2)
-
Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.
-
Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium |
18.8; 33.3; 75; 80; 56.3; 80 | — |
| SECONDARY Percent Change From Baseline in GL-3 Clearance From Plasma |
-52.37; -52.74; -49.06; -47.55; -52.01; -50.82 | — |
| SECONDARY Percent Change From Baseline in GL-3 Clearance From Urine |
-50.77; -63.39; -50.84; -52.55; -44.22; -63.87 | — |
Eligibility Criteria
Inclusion Criteria
- The participant and/or participant's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed.
- The participant must had a confirmed diagnosis of Fabry disease as documented by leukocyte α-Galactosidase A (αGAL) activity of 7.0 µg/mL) or urinary GL-3 (>0.3 mg GL-3/mmol creatinine) levels (results from a central laboratory).
- The participant must be male ≥5 and ≤18 years of age.
Exclusion Criteria
- Participant had 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).
- Participant had a Glomerular Filtration Rate (GFR) by iohexol 2 mm on T2- or fluid attenuated inversion recovery (FLAIR)- weighted images within the white matter or the basal ganglia.
- Participant had severe and recurrent acroparesthesia, judged by the physician as frequent (more than once a week) pain episodes for at least 3 months that influenced daily activities, irrespective of medication.
- Participant had 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 from Kampmann, et al 2000) as read at the study site.
- Participant had received prior treatment specific to Fabry Disease.
- Participant had participated in a study employing an investigational drug within 30 days of the start of their participation in this study.
- Participant had any medical condition or extenuating circumstance, which in the opinion of the Study Investigator, could interfere with study compliance.
- Participant had any medical condition or extenuating circumstance, for example diabetes mellitus, which in the opinion of the Study Investigator, could interfere with the interpretation of study results.
- Participant was on treatment with angiotensin converting enzyme inhibitors/angiotensin receptor blockers (ACEIs/ARBs).
- Participant had any contra-indication mentioned in the labeling of Fabrazyme and/or iohexol (Omnipaque).
- Participant or parent(s)/legal guardian(s) was unwilling to comply with the requirements of the protocol.
Data sourced from ClinicalTrials.gov (NCT00701415) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.