Clinical trial NCT01031173: Treatment Protocol of Replagal for Patients With Fabry Disease
Official title: An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Fabry Disease
Keywords: α galactosidase A
glycosphingolipid storage disorder
agalsidase alfa
enzyme replacement therapy
Replagal
agalsidase beta
Fabrazyme
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2009-12-14
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT

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®).

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 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.

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