Treatment Protocol of Replagal for Patients With Fabry Disease

May 19, 2021 updated by: Shire

An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.

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®).

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

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.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Tucson, Arizona, United States, 85718
        • AKDHC Tucson Campbell
    • California
      • La Jolla, California, United States, 92093-0830
        • University of California San Diego Medical Center
      • Los Angeles, California, United States, 90027
        • Kaiser Medical Group Southern CA, Regional Metabolic Services
      • Oakland, California, United States, 94609
        • Children's Hospital & Research Center Oakland
      • Sacramento, California, United States, 95817
        • UC Davis Children's Hospital
      • Salinas, California, United States, 93901
        • Central Coast Nephrology
    • Colorado
      • Denver, Colorado, United States, 80230
        • Denver Nephrologists, PC
    • Florida
      • Coral Springs, Florida, United States, 33065
        • University Research Foundation for Lysosomal Storage Disorders
      • Stuart, Florida, United States, 34994
        • Stuart Oncology Associates
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Emory University
      • Decatur, Georgia, United States, 30033
        • Emory University School of Medicine
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Health Center
    • Massachusetts
      • Springfield, Massachusetts, United States, 01199
        • Baystate Medical Center
    • Michigan
      • Grand Rapids, Michigan, United States, 49525
        • Infusion Associates
    • Missouri
      • Columbia, Missouri, United States, 65212
        • University of Missouri Healthcare
    • New Jersey
      • Paterson, New Jersey, United States, 07503
        • St. Joseph's Regional Medical Center
    • New York
      • Manhasset, New York, United States, 11030
        • North Shore Hematology/Oncology
      • New York, New York, United States, 10016
        • New York University School of Medicine
    • North Carolina
      • Asheville, North Carolina, United States, 28803
        • Fullerton Genetics Center-Mission, St. Joseph's Hospital
    • Ohio
      • Toledo, Ohio, United States, 43606
        • The Toledo Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Texas
      • Dallas, Texas, United States, 75246
        • Baylor University Medical Center
    • Virginia
      • Chesapeake, Virginia, United States, 23320
        • Tidewater Kidney Specialists
      • Christiansburg, Virginia, United States, 24073
        • Carilion New River Valley Medical Center
      • Springfield, Virginia, United States, 22152
        • O & O Alpan LLC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shire

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

December 8, 2009

First Submitted That Met QC Criteria

December 11, 2009

First Posted (Estimate)

December 14, 2009

Study Record Updates

Last Update Posted (Actual)

May 24, 2021

Last Update Submitted That Met QC Criteria

May 19, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HGT-REP-059

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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