A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms

Completed · Phase 3

Conditions studied: Fabry Disease

In brief

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.

Key facts

Study ID

NCT00701415

Run by

Genzyme, a Sanofi Company

People needed

31

Starts

2008-09-01

Expected to finish

2015-06-01

Last updated by the study team

2016-06-29

Who can join

Age: 5 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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 <4 nmol/hr/mg leukocyte (preferred assay; resulted from a central laboratory). If the leukocyte αGAL activity assay was difficult to obtain, the participant might be enrolled based on documented plasma αGAL <1.5 nmol/hr/mL, with the agreement of the Medical Monitor (resulted from a central laboratory).
• The participant must had evidence of globotriaosylceramide (GL-3) accumulation as documented by plasma GL-3 (>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.

You may not qualify if…

• 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 <90 L/min/1.73m\^2. In case of properly documented low protein intake, values as low as 80 mL/min/1.73 m\^2 might be acceptable, after consultation with the Medical Monitor.
• Participant had documented evidence of stroke or transient ischemic attack (TIA), or if a brain magnetic resonance imaging (MRI) had been performed, bright lesions >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.

Where it is running

• Study site — Decatur, Georgia, United States
• Study site — Cincinnati, Ohio, United States
• Study site — Seattle, Washington, United States
• Study site — Buenos Aires, Argentina
• Study site — Passo Fundo, Rio Grande do Sul, Brazil
• Study site — Vancouver, British Columbia, Canada
• Study site — Montreal, QC, Canada
• Study site — Prague, Czechia
• Study site — Amsterdam, Netherlands
• Study site — Bergen, Norway
• Study site — Warsaw, Poland
• Study site — Cambridge, United Kingdom

Full record on ClinicalTrials.gov

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