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
|Condition or disease||Intervention/treatment|
|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.
|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.|
- Biological: agalsidase alfa
0.2 mg/kg body weight, administered by an intravenous infusion over 40 minutes, every other week.Other Name: Replagal
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
|Study Director:||Study Director||Takeda|