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Trial record 1 of 1 for:    NCT00081497
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A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

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ClinicalTrials.gov Identifier: NCT00081497
Recruitment Status : Completed
First Posted : April 16, 2004
Results First Posted : August 18, 2010
Last Update Posted : April 2, 2015
Sponsor:
Information provided by:
Sanofi

Study Description
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Brief Summary:
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In 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 (globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: agalsidase beta Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 67 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study
Study Start Date : January 2004
Actual Primary Completion Date : September 2005
Actual Study Completion Date : September 2005

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Fabrazyme 1.0 mg/kg every 2 weeks
This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.
 Biological: agalsidase beta
1.0 mg/kg every 2 weeks
Other Names:
  • Fabrazyme
  • r-hαGAL


Outcome Measures
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Primary Outcome Measures :
  1. Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods [ Time Frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months) ]
    The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.


Secondary Outcome Measures :
  1. Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ]
    Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).

  2. Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ]
    Pre-Fabrazyme=baseline visit of AGAL-00-800 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).

  3. Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ]
    Pre-Fabrazyme=baseline visit of AGAL00800 for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL00800; assessment prior to first Fabrazyme infusion in AGAL02503 for placebo patients who did not transition to Fabrazyme in AGAL00800.

  4. Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ]
    Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).


Eligibility Criteria
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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients must have successfully completed the previous double-blind study AGAL-008-00 (NCT00074984)
  • Patients must provide written informed consent prior to study participation
  • Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study

Exclusion Criteria:

  • The patient was unable to complete AGAL-008-00 (NCT00074984)
  • The patient has undergone kidney transplantation or is currently on dialysis
  • The patient has diabetes mellitus or presence of confounding renal disease
  • The patient has a clinically significant organic disease or an unstable condition that precludes participation
  • The patient is unwilling to comply with the protocol requirements
Contacts and Locations
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Information from the National Library of Medicine

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): NCT00081497


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Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Medical Monitor Genzyme, a Sanofi Company
More Information
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Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00081497     History of Changes
Other Study ID Numbers: AGAL02503
First Posted: April 16, 2004    Key Record Dates
Results First Posted: August 18, 2010
Last Update Posted: April 2, 2015
Last Verified: March 2015

Keywords provided by Sanofi:
alpha-galactosidase A
a-GAL
r-haGAL
 Fabry
GL-3
Fabrazyme

Additional relevant MeSH terms:
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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