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Treatment Protocol of Replagal for Patients With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01031173
Expanded Access Status : No longer available
First Posted : December 14, 2009
Last Update Posted : May 24, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
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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®).

Condition or disease Intervention/treatment
Fabry Disease Biological: agalsidase alfa

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

Resource links provided by the National Library of Medicine


Interventions
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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
Eligibility Criteria
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Information from the National Library of Medicine

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, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01031173


Locations
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Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Takeda
More Information
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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01031173    
Obsolete Identifiers: NCT00726089
Other Study ID Numbers: HGT-REP-059
First Posted: December 14, 2009    Key Record Dates
Last Update Posted: May 24, 2021
Last Verified: May 2021
Keywords provided by Takeda ( Shire ):
α galactosidase A
glycosphingolipid storage disorder
agalsidase alfa
enzyme replacement therapy
 Replagal
agalsidase beta
Fabrazyme
Additional relevant MeSH terms:
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Fabry Disease
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
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders