A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

NCT00140621 | Completed Phase 4 Results

• 1 other identifier: AGAL03204
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 7

Brief Summary

This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme \[recombinant form\]) administered by intravenous drip infusion in participants with cardiac Fabry disease. Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.

Conditions

Fabry Disease

Keywords

cardiac fabry disease

Outcome Measures

Primary Outcomes (4)

• Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156

  Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.

  Baseline to Week 156

• Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156

  Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.

  Baseline to Week 156

• Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156

  Left ventricular mass was assessed by echocardiogram.

  Baseline to Week 156

• Change From Baseline in LVM at Week 156

  Left ventricular mass was assessed by echocardiogram.

  Baseline to Week 156

Secondary Outcomes (3)

• Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test

  Baseline to Week 156

• Percent Change From Baseline in GL-3 Plasma Levels at Week 156

  Baseline to Week 156

• Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156

  Baseline to Week 156

Study Arms (1)

Agalsidase Beta

EXPERIMENTAL

Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.

Drug: Agalsidase beta

Interventions

Agalsidase beta DRUG

Also known as: Fabrazyme®

Agalsidase Beta

Eligibility Criteria

• Age: 20 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria)
• In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants)
• Left ventricular hypertrophy was noted.
• Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed
• Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was related by birth.)
• Without symptoms or signs of Fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign.
• Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent
• Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent.
• Participants classification: inpatients and outpatients
• Participants who had given written informed consent before the study-related baseline tests.

You may not qualify if:

• Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury \[mmHg\] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication)
• Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
• Participants who had undergone kidney transplantation or were currently on dialysis.
• Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\] level exceeded the value five times as high as the upper normal limit).
• Permanent pacemaker or defibrillator implanted participants
• Pregnant or lactating women
• Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent.
• Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.
• Enzyme replacement therapy history, except for agalsidase beta
• Participants who were unwilling to comply with the requirements of the protocol.
• Others judged by the investigator or sub-investigator to be ineligible for the study

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (7)

Fujita Health University Hospital

Aichi, 470-1192, Japan

Location

Sapporo Medical University Hospital

Hokkaido, 060-8543, Japan

Location

Akune Citizen Hospital

Kagoshima, 899-1611, Japan

Location

Tohoku University Hospital

Miyagi, 980-8574, Japan

Location

Nihon University Itabashi Hospital

Tokyo, 173-8610, Japan

Location

Nihon University Nerima Hikarigaoka Hospital

Tokyo, 179-0072, Japan

Location

Yamanashi Prefectural Central Hospital

Yamanashi, 400-8506, Japan

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi

Contact-US@Sanofi.com

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2005
• First Posted: September 1, 2005
• Study Start: July 1, 2005
• Primary Completion: August 1, 2012
• Study Completion: August 1, 2012
• Last Updated: May 12, 2015
• Results First Posted: April 15, 2015

Record last verified: 2015-04

Locations

JP (7)