Treatment Protocol of Replagal for 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 Type:||Expanded Access What is Expanded Access?|
|Official Title:||An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.|
Biological: agalsidase alfa
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01031173
Show 25 Study Locations
|Principal Investigator:||Alpana Desai, MD||Stuart Oncology Associates|
|Principal Investigator:||Rebecca Mardach, MD||Kaiser Medical Group Southern California|
|Principal Investigator:||Neal Weinreb, MD||University Research Foundation for Lysosomal Storage Disorders|
|Principal Investigator:||Khan Nedd, MD||Infusion Associates|
|Principal Investigator:||Oral Alpan, MD||O & O Alpan LLC|
|Principal Investigator:||Myrl Holida, PA-C||University of Iowa Health Center|
|Principal Investigator:||Jennifer Ibrahim, MD||St. Joseph's Regional Medical Center, Wisconsin|
|Principal Investigator:||Howard Lien||AKDHC Tucson Campbell|
|Principal Investigator:||Bruce Barshop, MD||University of California, San Diego|
|Principal Investigator:||Paul Fernhoff, MD||Emory University|
|Principal Investigator:||Ellen Boyd, MD||Fullerton Genetics Center-Mission, St. Joseph's Hospital|
|Principal Investigator:||Richard Forte, MD||1201 Northern Blvd.|
|Principal Investigator:||Richard Hillman, MD||University of Missouri-Columbia|
|Principal Investigator:||Geoffrey Allan Block, MD||Denver Nephrologists, PC|
|Principal Investigator:||Barbara L. Rever, MD||Central Coast Nephrology|
|Principal Investigator:||Paul R. Harmatz, MD||Children's Hospital & Research Center Oakland|
|Principal Investigator:||Mary-Alice Abbott, MD, PhD||Baystate Medical Center|
|Principal Investigator:||Raphael Schiffmann, MD, MHS||Baylor Health Care System|
|Principal Investigator:||William B. Baker, MD||Carilion New River Valley Medical Center|
|Principal Investigator:||Gregory M. Pastores, MD||New York University School of Medicine|
|Principal Investigator:||Simeon A. Boyd, MD||UC Davis Children's Hospital|
|Principal Investigator:||Robert Zimmerman, MD||The Toledo Hospital|
|Principal Investigator:||Joel T. Bundy, MD||Tidewater Kidney Specialists|
|Principal Investigator:||Ozlem Goker-Alpan, MD||O & O Alpan LLC|
|Principal Investigator:||Suma P. Shankar, MD, PhD||Emory University|
|Principal Investigator:||Robert D. Steiner, MD||Oregon Health and Science University|