Open-Label Extension Study for Patients Who Completed a Phase 3 Double-blind Study of PEG-uricase for Symptomatic Gout

Study Overview
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Brief Summary
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This is an open-label extension phase of two double-blind, placebo-controled Phase 3 protocols evaluating PEG-uricase in the treatment of hyperuricemic subjects with symptomatic gout. The purpose of this study is to provide a continuation of treatment to subjects completing the double-blind studies and obtain long-term safety and efficacy data.

Official Title
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Multicenter, Open Label Extension Study of 8 mg PEG-uricase in Subjects Who Completed Protocols C0405 or C0406 for Symptomatic Gout

Conditions
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Gout

Intervention / Treatment
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* Biological: pegloticase


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completed treatment in one of two double-blind, placebo controlled studies of PEG-uricase in subjects with hyperuricemia and symptomatic gout Exclusion Criteria: unstable angina uncontrolled arrhythmia uncontrolled hypertension end stage renal disease requiring dialysis anemia

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: q2 RCT<br>Pegloticase every 2 wk arm of Randomized Controlled Trial(RCT), continued to receive pegloticase every 2 weeks (q2 wk) or every 4 weeks (q4 wk) in Open Label Extention (OLE) study | Biological: pegloticase<br>* 8 mg intravenous infusion<br>|
| Experimental: q4 RCT<br>Pegloticase every 4 wk arm of Randomized Controlled Trial (RCT), continued to receive pegloticase every 2 weeks (q2 wk) or every 4 weeks (q4 wk) in Open Label Extention (OLE) study | Biological: pegloticase<br>* 8 mg intravenous infusion<br>|
| Experimental: Placebo in RCT<br>Placebo arm in Randomized Controlled Trial (RCT), received pegloticase every 2 weeks (q2 wk) or every 4 weeks (q4 wk) in OLE | Biological: pegloticase<br>* 8 mg intravenous infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Uric Acid (mg/dL) | Uric acid measured at 3 month-intervals | Week 13, Week 25, Week 53, Week 101 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Tophus Response | Target tophi evaluated during the randomized, controlled study were followed for response at 3, 6, 12, 18 and 24 months in this open-label extention study. Results from each participant's final assessment on drug are reported (as last observation carried forward). Complete response=complete disappearance of at least one tophus with no new or worsening tophus. Partial Response=a 50% or more decrease in at least one tophus with no new or worsening tophus. | Up to 2 years | 
| Patient Reported Outcome: SF-36 Physical Component Summary Score | SF-36 is the Medical Outcomes Survey Short Form-36, a 36-item self-reported questionnaire which assesses health-related limitations in 8 dimensions. The Physical Component Summary Score (PCS) is a composite summary score derived from the dimensions related to physical functioning outcomes: Physical Function, Role Physical, General Health and Bodily Pain (each with a 0 to 100 scale where 0=worst, 100=best). The Summary Score is constructed as a T-score with a mean of 50 and standard deviation of 10, where higher scores indicate a better health status. | RCT Week 25; OLE Week 25; OLE Week 53, OLE Week 77, OLE Week 101 | 
| Gout Flare Frequency | The the mean number of flares per subject (flare frequency)was assessed over 3-month periods for up to 2 years of treatment | Up to 2 years | 
| Gout Flare Incidence | Percentage of participants remaining in the study during the specified interval who experienced a gout flare during this interval. | Assessed in 3-month intervals up to 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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gout, chronic gout