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Treatment Protocol of Replagal for Patients With Fabry Disease

Expanded access is no longer available for this treatment.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01031173
First received: December 8, 2009
Last updated: February 19, 2014
Last verified: February 2014
  Purpose

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®).


Condition Intervention
Fabry Disease
Biological: agalsidase alfa

Study Type: Expanded Access     What is Expanded Access?
Official Title: An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.

Resource links provided by NLM:


Further study details as provided by Shire:

Intervention Details:
    Biological: agalsidase alfa
    0.2 mg/kg body weight, administered by an intravenous infusion over 40 minutes, every other week.
    Other Name: Replagal
Detailed Description:

This study will evaluate the safety and efficacy of Replagal in patients with Fabry disease who are either naive to treatment, who were previously treated with agalsidase beta, or who had previously received Replagal.

Patients diagnosed with Fabry disease who have not previously received treatment, who have received agalsidase beta, or who had previously received Replagal will be eligible to enroll in the study and will receive Replagal at a dose of 0.2 mg/kg body weight administered by an IV infusion over 40 minutes every other week.

This study will be conducted in the United States.

Study visits will occur in 3 phases:

  • Screening/Baseline Phase: A Screening/Baseline period (Day -30 to Day -1) to determine eligibility and obtain baseline measurements. Patients who have previously received agalsidase beta will be tested for the presence of anti-agalsidase beta antibodies.
  • Treatment Phase: A 12-month treatment phase during which all patients will receive Replagal administered IV every other week. Clinical assessments will occur at Months 1, 3, 6, 9, and 12. The study may be extended to continue giving patients access to treatment.
  • End-of-Study Phase: An end-of-study contact either as an office visit or follow-up telephone call will occur one month after the last infusion.
  Eligibility

Genders Eligible for Study:   Both
Criteria

Inclusion Criteria:

  1. Confirmed diagnosis of Fabry disease.
  2. Patient is willing and able to provide written informed consent, and assent if applicable.
  3. 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.

Exclusion Criteria:

  1. Hypersensitivity to Replagal, the active substance, or any of the excipients.
  2. The patient is pregnant or breast feeding.
  3. Concomitant use of agalsidase beta (Fabrazyme).
  4. Has received treatment with any investigation drug or device within the 30 days prior to study entry.
  5. Otherwise unsuitable for the study, in the opinion of the Investigator.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01031173

  Show 25 Study Locations
Sponsors and Collaborators
Shire
Investigators
Principal Investigator: Alpana Desai, MD Stuart Oncology Associates
Principal Investigator: Rebecca Mardach, MD Kaiser Medical Group Southern California
Principal Investigator: Neal Weinreb, MD University Research Foundation for Lysosomal Storage Disorders
Principal Investigator: Khan Nedd, MD Infusion Associates
Principal Investigator: Oral Alpan, MD O & O Alpan LLC
Principal Investigator: Myrl Holida, PA-C University of Iowa Health Center
Principal Investigator: Jennifer Ibrahim, MD St. Joseph's Regional Medical Center, Wisconsin
Principal Investigator: Howard Lien AKDHC Tucson Campbell
Principal Investigator: Bruce Barshop, MD University of California, San Diego
Principal Investigator: Paul Fernhoff, MD Emory University
Principal Investigator: Ellen Boyd, MD Fullerton Genetics Center-Mission, St. Joseph's Hospital
Principal Investigator: Richard Forte, MD 1201 Northern Blvd.
Principal Investigator: Richard Hillman, MD University of Missouri-Columbia
Principal Investigator: Geoffrey Allan Block, MD Denver Nephrologists, PC
Principal Investigator: Barbara L. Rever, MD Central Coast Nephrology
Principal Investigator: Paul R. Harmatz, MD Children's Hospital & Research Center Oakland
Principal Investigator: Mary-Alice Abbott, MD, PhD Baystate Medical Center
Principal Investigator: Raphael Schiffmann, MD, MHS Baylor Health Care System
Principal Investigator: William B. Baker, MD Carilion New River Valley Medical Center
Principal Investigator: Gregory M. Pastores, MD New York University School of Medicine
Principal Investigator: Simeon A. Boyd, MD UC Davis Children's Hospital
Principal Investigator: Robert Zimmerman, MD The Toledo Hospital
Principal Investigator: Joel T. Bundy, MD Tidewater Kidney Specialists
Principal Investigator: Ozlem Goker-Alpan, MD O & O Alpan LLC
Principal Investigator: Suma P. Shankar, MD, PhD Emory University
Principal Investigator: Robert D. Steiner, MD Oregon Health and Science University
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01031173     History of Changes
Obsolete Identifiers: NCT00726089
Other Study ID Numbers: HGT-REP-059
Study First Received: December 8, 2009
Last Updated: February 19, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Shire:
α galactosidase A
glycosphingolipid storage disorder
agalsidase alfa
enzyme replacement therapy
Replagal
agalsidase beta
Fabrazyme

Additional relevant MeSH terms:
Fabry Disease
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Cardiovascular Diseases
Central Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lipidoses
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Sphingolipidoses
Vascular Diseases

ClinicalTrials.gov processed this record on November 20, 2014