Treatment Protocol of Replagal for Patients With Fabry Disease

NCT01031173 | Unknown

• 1 other identifier: HGT-REP-059
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 25

Brief Summary

The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week. The study will monitor the course of disease in males and females with Fabry disease who are naive to treatment or were previously treated with agalsidase beta (Fabrazyme®).

Conditions

Fabry Disease

Keywords

α galactosidase A; glycosphingolipid storage disorder; agalsidase alfa; enzyme replacement therapy; Replagal; agalsidase beta; Fabrazyme

Interventions

agalsidase alfa BIOLOGICAL

0.2 mg/kg body weight, administered by an intravenous infusion over 40 minutes, every other week.

Also known as: Replagal

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of Fabry disease.
• Patient is willing and able to provide written informed consent, and assent if applicable.
• Females of childbearing potential must agree to use a method of birth control throughout the study and for at least 30 days after the final infusion. The method of contraception must be considered adequate and appropriate in the opinion of the investigator.

You may not qualify if:

• Hypersensitivity to Replagal, the active substance, or any of the excipients.
• The patient is pregnant or breast feeding.
• Concomitant use of agalsidase beta (Fabrazyme).
• Has received treatment with any investigation drug or device within the 30 days prior to study entry.
• Otherwise unsuitable for the study, in the opinion of the Investigator.

Sponsors & Collaborators

• Shire: lead

Study Sites (25)

AKDHC Tucson Campbell

Tucson, Arizona, 85718, United States

Location

University of California San Diego Medical Center

La Jolla, California, 92093-0830, United States

Location

Kaiser Medical Group Southern CA, Regional Metabolic Services

Los Angeles, California, 90027, United States

Location

Children's Hospital & Research Center Oakland

Oakland, California, 94609, United States

Location

UC Davis Children's Hospital

Sacramento, California, 95817, United States

Location

Central Coast Nephrology

Salinas, California, 93901, United States

Location

Denver Nephrologists, PC

Denver, Colorado, 80230, United States

Location

University Research Foundation for Lysosomal Storage Disorders

Coral Springs, Florida, 33065, United States

Location

Stuart Oncology Associates

Stuart, Florida, 34994, United States

Location

Emory University School of Medicine

Decatur, Georgia, 30033, United States

Location

Emory University

Decatur, Georgia, 30033, United States

Location

University of Iowa Health Center

Iowa City, Iowa, 52242, United States

Location

Baystate Medical Center

Springfield, Massachusetts, 01199, United States

Location

Infusion Associates

Grand Rapids, Michigan, 49525, United States

Location

University of Missouri Healthcare

Columbia, Missouri, 65212, United States

Location

St. Joseph's Regional Medical Center

Paterson, New Jersey, 07503, United States

Location

North Shore Hematology/Oncology

Manhasset, New York, 11030, United States

Location

New York University School of Medicine

New York, New York, 10016, United States

Location

Fullerton Genetics Center-Mission, St. Joseph's Hospital

Asheville, North Carolina, 28803, United States

Location

The Toledo Hospital

Toledo, Ohio, 43606, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Tidewater Kidney Specialists

Chesapeake, Virginia, 23320, United States

Location

Carilion New River Valley Medical Center

Christiansburg, Virginia, 24073, United States

Location

O & O Alpan LLC

Springfield, Virginia, 22152, United States

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase alfa

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

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2009
• First Posted: December 14, 2009
• Last Updated: May 24, 2021

Record last verified: 2021-05

Locations

US (25)