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

This study has been completed.
Sponsor:
Collaborator:
Savient Pharmaceuticals
Information provided by (Responsible Party):
John Sundy, Duke University Medical Center
ClinicalTrials.gov Identifier:
NCT00111657
First received: May 24, 2005
Last updated: March 21, 2013
Last verified: March 2013
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
  Description
Single Arm - Pegloticase No text entered.

Participant Flow:   Overall Study
    Single Arm - Pegloticase  
STARTED     37 [1]
COMPLETED     21 [2]
NOT COMPLETED     16  
[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.
No text entered.

Reporting Groups
  Description
Single Arm - Pegloticase No text entered.

Baseline Measures
    Single Arm - Pegloticase  
Number of Participants  
[units: participants]
  37  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     25  
>=65 years     12  
Age  
[units: years]
Mean ± Standard Deviation
  57.6  ± 14.8  
Gender  
[units: participants]
 
Female     9  
Male     28  
Region of Enrollment  
[units: participants]
 
United States     37  



  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 Will be Evaluated, Including the Frequency of Gout Flares, Number and Distribution of Swollen/Tender Joints, Change in Size of Tophi, Change in Functional Status   [ Time Frame: weekly through 7 weeks after last infusion ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

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 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   Reduction of the Ratio of Uric Acid:Creatinine in Urine   [ Time Frame: baseline then weekly ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

5.  Secondary:   Development of Antibodies to PEG-uricase   [ Time Frame: baseline, then prior to infusions and 7 wks after last infusion ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

6.  Secondary:   Pharmacokinetics of PEG-uricase   [ Time Frame: weekly ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


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


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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Results Point of Contact:  
Name/Title: John S. Sundy, MD, PhD
Organization: Duke University Medical Center
phone: 919-684-2347
e-mail: john.sundy@duke.edu


Publications of Results:
Other Publications:

Responsible Party: John Sundy, Duke University Medical Center
ClinicalTrials.gov 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: March 21, 2013
Health Authority: United States: Food and Drug Administration
United States: Institutional Reveiw Board