A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Overview

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.

Full Title of Study: “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 Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 2005

Interventions

  • Biological: agalsidase beta
    • 1.0 mg/kg every 2 weeks

Arms, Groups and Cohorts

  • 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.

Clinical Trial Outcome Measures

Primary Measures

  • 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 Measures

  • 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).
  • 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).
  • 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.
  • 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).

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 16 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Genzyme, a Sanofi Company
  • Provider of Information About this Clinical Study
    • Medical Monitor, Genzyme Corporation
  • Overall Official(s)
    • Medical Monitor, Study Director, Genzyme, a Sanofi Company

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