Treatment Protocol of Replagal for Patients With Fabry Disease

NCT ID: NCT01031173

Last Updated: 2021-05-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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®).

Detailed Description

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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.

Conditions

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Fabry Disease

Interventions

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agalsidase alfa

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

Intervention Type BIOLOGICAL

Other Intervention Names

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Replagal

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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AKDHC Tucson Campbell

Tucson, Arizona, United States

Site Status

University of California San Diego Medical Center

La Jolla, California, United States

Site Status

Kaiser Medical Group Southern CA, Regional Metabolic Services

Los Angeles, California, United States

Site Status

Children's Hospital & Research Center Oakland

Oakland, California, United States

Site Status

UC Davis Children's Hospital

Sacramento, California, United States

Site Status

Central Coast Nephrology

Salinas, California, United States

Site Status

Denver Nephrologists, PC

Denver, Colorado, United States

Site Status

University Research Foundation for Lysosomal Storage Disorders

Coral Springs, Florida, United States

Site Status

Stuart Oncology Associates

Stuart, Florida, United States

Site Status

Emory University School of Medicine

Decatur, Georgia, United States

Site Status

Emory University

Decatur, Georgia, United States

Site Status

University of Iowa Health Center

Iowa City, Iowa, United States

Site Status

Baystate Medical Center

Springfield, Massachusetts, United States

Site Status

Infusion Associates

Grand Rapids, Michigan, United States

Site Status

University of Missouri Healthcare

Columbia, Missouri, United States

Site Status

St. Joseph's Regional Medical Center

Paterson, New Jersey, United States

Site Status

North Shore Hematology/Oncology

Manhasset, New York, United States

Site Status

New York University School of Medicine

New York, New York, United States

Site Status

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

Asheville, North Carolina, United States

Site Status

The Toledo Hospital

Toledo, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Baylor University Medical Center

Dallas, Texas, United States

Site Status

Tidewater Kidney Specialists

Chesapeake, Virginia, United States

Site Status

Carilion New River Valley Medical Center

Christiansburg, Virginia, United States

Site Status

O & O Alpan LLC

Springfield, Virginia, United States

Site Status

Countries

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United States

Other Identifiers

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HGT-REP-059

Identifier Type: -

Identifier Source: org_study_id

NCT00726089

Identifier Type: -

Identifier Source: nct_alias

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