Treatment Protocol of Replagal for Patients With Fabry Disease

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT01031173
• Other study ID #: HGT-REP-059
• Status: No longer available

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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®).

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.

Other known NCT identifiers

• NCT00726089

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Biological:
• agalsidase alfa
0.2 mg/kg body weight, administered by an intravenous infusion over 40 minutes, every other week.

Locations

Country	Name	City	State
United States	Fullerton Genetics Center-Mission, St. Joseph's Hospital	Asheville	North Carolina
United States	Tidewater Kidney Specialists	Chesapeake	Virginia
United States	Carilion New River Valley Medical Center	Christiansburg	Virginia
United States	University of Missouri Healthcare	Columbia	Missouri
United States	University Research Foundation for Lysosomal Storage Disorders	Coral Springs	Florida
United States	Baylor University Medical Center	Dallas	Texas
United States	Emory University	Decatur	Georgia
United States	Emory University School of Medicine	Decatur	Georgia
United States	Denver Nephrologists, PC	Denver	Colorado
United States	Infusion Associates	Grand Rapids	Michigan
United States	University of Iowa Health Center	Iowa City	Iowa
United States	University of California San Diego Medical Center	La Jolla	California
United States	Kaiser Medical Group Southern CA, Regional Metabolic Services	Los Angeles	California
United States	North Shore Hematology/Oncology	Manhasset	New York
United States	New York University School of Medicine	New York	New York
United States	Children's Hospital & Research Center Oakland	Oakland	California
United States	St. Joseph's Regional Medical Center	Paterson	New Jersey
United States	Oregon Health and Science University	Portland	Oregon
United States	UC Davis Children's Hospital	Sacramento	California
United States	Central Coast Nephrology	Salinas	California
United States	Baystate Medical Center	Springfield	Massachusetts
United States	O & O Alpan LLC	Springfield	Virginia
United States	Stuart Oncology Associates	Stuart	Florida
United States	The Toledo Hospital	Toledo	Ohio
United States	AKDHC Tucson Campbell	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States,