A Study of Fabrazyme in Pediatric Patients With Fabry Disease
This study has been completed.
Sponsor:
Genzyme
Information provided by:
Genzyme
ClinicalTrials.gov Identifier:
NCT00074958
First received: December 24, 2003
Last updated: August 11, 2009
Last verified: April 2009
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Purpose
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.
| Condition | Intervention | Phase |
|---|---|---|
|
Fabry Disease |
Biological: Fabrazyme (agalsidase beta) |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Fabry disease
Farber lipogranulomatosis
Schindler disease
succinic semialdehyde dehydrogenase deficiency
U.S. FDA Resources
Further study details as provided by Genzyme:
Primary Outcome Measures:
- Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin [ Time Frame: Baseline, Week 24 and Week 48 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Plasma GL-3 [ Time Frame: Baseline, Week 24 and Week 48 ] [ Designated as safety issue: No ]
| Enrollment: | 16 |
| Study Start Date: | October 2002 |
| Study Completion Date: | July 2005 |
| Primary Completion Date: | May 2005 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Fabrazyme
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks
|
Biological: Fabrazyme (agalsidase beta)
1 mg/kg every 2 weeks
Other Name: r-hαGAL
|
Eligibility| Ages Eligible for Study: | 7 Years to 15 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00074958
Locations
| 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 | |
Sponsors and Collaborators
Genzyme
Investigators
| Study Director: | Medical Monitor | Genzyme |
More Information
No publications provided by Genzyme
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Medical Monitor, Genzyme Corporation |
| ClinicalTrials.gov Identifier: | NCT00074958 History of Changes |
| Other Study ID Numbers: | AGAL-016-01 |
| Study First Received: | December 24, 2003 |
| Results First Received: | March 3, 2009 |
| Last Updated: | August 11, 2009 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Genzyme:
|
a-Galactosidase A aGal r-haGAL |
Fabry GL-3 Fabrazyme |
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 June 18, 2013