Pegylated Recombinant Mammalian Uricase (PEG-uricase) as Treatment for Refractory Gout

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. Identifier: NCT00111657
Recruitment Status : Completed
First Posted : May 25, 2005
Results First Posted : January 18, 2013
Last Update Posted : October 3, 2014
Savient Pharmaceuticals
Information provided by (Responsible Party):
John Sundy, Duke University

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Gout
Intervention: Biological: Pegloticase

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
Single Arm - Pegloticase No text entered.

Participant Flow:   Overall Study
    Single Arm - Pegloticase
STARTED   37 [1] 
COMPLETED   21 [2] 
[1] 37 consented. 30 initiated treatment. 29 completed at least 1 dose.
[2] Completed all intended doses.

  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
Single Arm - Pegloticase No text entered.

Baseline Measures
   Single Arm - Pegloticase 
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
<=18 years   0 
Between 18 and 65 years   25 
>=65 years   12 
[Units: Years]
Mean (Standard Deviation)
 57.6  (14.8) 
[Units: Participants]
Female   9 
Male   28 
Region of Enrollment 
[Units: Participants]
United States   37 
Baseline urate concentration (pUA) 
[Units: mg/dL]
Mean (Standard Deviation)
 10.8  (1.2) 

  Outcome Measures

1.  Primary:   Reduction in Plasma Uric Acid to Less Than 6 mg/dL.   [ Time Frame: Baseline to Day 105 ]

2.  Secondary:   Clinical Response: Number of Swollen and Tender Joints   [ Time Frame: Basline and day 134 ]

3.  Secondary:   Development of Antibodies to PEG-uricase   [ Time Frame: baseline, then prior to infusions and 7 wks after last infusion ]

4.  Secondary:   Infusion 1: Maximum Concentration (Cmax) Value   [ Time Frame: 2 hours ]

5.  Secondary:   Infusion 1: Minimum Concentration (Cmin)   [ Time Frame: 21 days after the infusion ]

6.  Secondary:   In a Subset of Subjects Who Volunteer Separately, Change in Uric Acid Pool Size Will be Assessed by a Method That Involves Infusion of Uric Acid Labeled With N15, a Stable (Nonradioactive) Isotope of Nitrogen.   [ Time Frame: baseline and 7 weeks after last infusion ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

7.  Secondary:   Reduction of the Ratio of Uric Acid:Creatinine in Urine   [ Time Frame: baseline then weekly ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact:  
Name/Title: John S. Sundy, MD, PhD
Organization: Duke University Medical Center
phone: 919-684-2347

Publications of Results:
Other Publications:
Publications automatically indexed to this study by Identifier (NCT Number):

Responsible Party: John Sundy, Duke University Identifier: NCT00111657     History of Changes
Other Study ID Numbers: Pro00006845
FD-R-0002537 ( Other Identifier: FDA OOPD )
First Submitted: May 24, 2005
First Posted: May 25, 2005
Results First Submitted: December 12, 2012
Results First Posted: January 18, 2013
Last Update Posted: October 3, 2014