A Study of Fabrazyme in Pediatric Patients With Fabry Disease
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ClinicalTrials.gov Identifier: NCT00074958 |
Recruitment Status :
Completed
First Posted : December 25, 2003
Results First Posted : June 16, 2009
Last Update Posted : April 2, 2015
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Fabry Disease | Biological: Fabrazyme (agalsidase beta) | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 16 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease |
Study Start Date : | October 2002 |
Actual Primary Completion Date : | May 2005 |
Actual Study Completion Date : | July 2005 |

Arm | Intervention/treatment |
---|---|
Experimental: Fabrazyme
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks
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Biological: Fabrazyme (agalsidase beta)
1 mg/kg every 2 weeks
Other Name: r-hαGAL |
- Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin [ Time Frame: Baseline, Week 24 and Week 48 ]Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
- Plasma GL-3 [ Time Frame: Baseline, Week 24 and Week 48 ]Plasma GL-3 values at Baseline, Week 24, and Week 48. Normal plasma GL-3 level is ≤ 7.03 µg/mL.

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Ages Eligible for Study: | 7 Years to 15 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion criteria:
- Patient or legal guardian must provide written informed consent
- Patients must have a clinical diagnosis of Fabry disease and active Fabry disease (clinical signs and symptoms)
- Patients must be at least 7 years of age but no older than 15 years of age at time of enrollment
- Patients must be Tanner Stage ≤ III
- Female patients must have a negative pregnancy test prior to each infusion and use a medically accepted form of contraception throughout the study
Exclusion Criteria:
- Patient has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) that in the opinion of the investigator would preclude participation in the trial
- Patient has participated in a study employing investigational drug within 30 days of the start of this study
- Patient has received prior treatment with enzyme replacement therapy
- Patient is unable to comply with the clinical protocol

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00074958
United States, Arizona | |
University of Arizona | |
Tucson, Arizona, United States, 85724 | |
France | |
Hopital Edouard Herriot | |
Lyon, France, Cedex 03 | |
Hopital de la Timone Enfants | |
Marseille, France, Cedex 05 | |
Hopital Europeen Georges Pompidou | |
Paris, France, Cedex 15 | |
Poland | |
Instytut Pomnik Centrum Zdrowia Dziecka | |
Warsaw, Poland, 04-730 | |
United Kingdom | |
Royal Manchester Children's Hospital | |
Pendlebury, Manchester, United Kingdom, M27 4HA | |
Great Ormond Street Hospital for Sick Children | |
London, United Kingdom, WC1N 3JH |
Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
Responsible Party: | Medical Monitor, Genzyme Corporation |
ClinicalTrials.gov Identifier: | NCT00074958 |
Other Study ID Numbers: |
AGAL-016-01 |
First Posted: | December 25, 2003 Key Record Dates |
Results First Posted: | June 16, 2009 |
Last Update Posted: | April 2, 2015 |
Last Verified: | March 2015 |
a-Galactosidase A aGal r-haGAL |
Fabry GL-3 Fabrazyme |
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders |
Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |