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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00701415
First received: June 17, 2008
Last updated: March 18, 2014
Last verified: March 2014
  Purpose

The purpose of this study is 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) are effective in treatment-naïve pediatric patients without severe symptoms. Patients will be treated for 5 years.


Condition Intervention Phase
Fabry Disease
Biological: agalsidase beta
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (Agalsidase Beta) in Treatment-Naïve Male Pediatric Patients With Fabry Disease Without Severe Symptoms

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • globotriaosylceramide (GL-3) inclusion in skin vascular endothelium [ Time Frame: Up to Week 260/Year 5 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • GL-3 clearance in Plasma [ Time Frame: Up to month 60 ] [ Designated as safety issue: No ]
  • GL-3 clearance in Urine [ Time Frame: Up to month 60 ] [ Designated as safety issue: No ]

Enrollment: 31
Study Start Date: September 2008
Estimated Study Completion Date: July 2015
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Fabrazyme Dosing Regimen 1 Biological: agalsidase beta
1.0 mg/kg/4 weeks
Other Name: Fabrazyme
Active Comparator: Fabrazyme Dosing Regimen 2 Biological: agalsidase beta
0.5 mg/kg/2 weeks
Other Name: Fabrazyme

  Eligibility

Ages Eligible for Study:   5 Years to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The patient and/or patient's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed.
  • The patient must have a confirmed diagnosis of Fabry disease as documented by leukocyte α-Galactosidase A (αGAL) activity of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory). If the leukocyte αGAL activity assay is difficult to obtain, the patient may be enrolled based on documented plasma αGAL <1.5 nmol/hr/mL, with the agreement of the Medical Monitor (results from a central laboratory).
  • The patient must have 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 patient must be male ≥5 and ≤18 years of age.

Exclusion Criteria:

  • 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).
  • Patient has 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 may be acceptable, after consultation with the Medical Monitor.
  • Patient has documented evidence of stroke or transient ischemic attack (TIA), or if a brain magnetic resonance imaging (MRI) has been performed, bright lesions >2 mm on T2- or fluid attenuated inversion recovery (FLAIR)- weighted images within the white matter or the basal ganglia.
  • 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.
  • 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 from Kampmann, et al 2000) as read at the study site.
  • Patient has received prior treatment specific to Fabry Disease.
  • Patient has participated in a study employing an investigational drug within 30 days of the start of their participation in this study.
  • Patient has any medical condition or extenuating circumstance, which in the opinion of the Study Investigator, could interfere with study compliance.
  • Patient has 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.
  • Patient is on treatment with angiotensin converting enzyme inhibitors/angiotensin receptor blockers (ACEIs/ARBs).
  • Patient has any contra-indication mentioned in the labeling of Fabrazyme and/or iohexol (Omnipaque).
  • Patient or parent(s)/legal guardian(s) is unwilling to comply with the requirements of the protocol.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00701415

Locations
United States, Georgia
Decatur, Georgia, United States
United States, Ohio
Cincinnati, Ohio, United States
United States, Washington
Seattle, Washington, United States
Argentina
Buenos Aires, Argentina
Brazil
Passo Fundo, RS, Brazil
Canada, British Columbia
Vancouver, British Columbia, Canada
Canada
Montreal, QC, Canada
Czech Republic
Prague 2, Czech Republic
Netherlands
Amsterdam, Netherlands
Norway
Bergen, Norway
Poland
Warsaw, Poland
United Kingdom
Cambridge, United Kingdom
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided by Sanofi

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00701415     History of Changes
Other Study ID Numbers: AGAL06207, 2007-005668-28
Study First Received: June 17, 2008
Last Updated: March 18, 2014
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Brazil: National Health Surveillance Agency
Canada: Ministry of Health & Long Term Care, Ontario
Netherlands: Medicines Evaluation Board (MEB)
Norway: Norwegian Medicines Agency (NoMA)
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products (CBEK)
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Sanofi:
α-GAL, α-Galactosidase-A, r-hαGAL

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on April 17, 2014