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

This study has been completed.
Savient Pharmaceuticals
Information provided by (Responsible Party):
John Sundy, Duke University Medical Center Identifier:
First received: May 24, 2005
Last updated: September 24, 2014
Last verified: September 2014
Results First Received: December 12, 2012  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Gout
Intervention: Biological: Pegloticase

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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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
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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  
Number of Participants  
[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
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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:   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 ]

4.  Secondary:   Reduction of the Ratio of Uric Acid:Creatinine in Urine   [ Time Frame: baseline then weekly ]

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

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

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

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats
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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
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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:

Responsible Party: John Sundy, Duke University Medical Center Identifier: NCT00111657     History of Changes
Other Study ID Numbers: Pro00006845, FD-R-0002537
Study First Received: May 24, 2005
Results First Received: December 12, 2012
Last Updated: September 24, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Reveiw Board