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

• ClinicalTrials.gov Identifier: NCT01356498
• Recruitment Status: Completed
• First Posted: May 19, 2011
• Results First Posted: December 2, 2011
• Last Update Posted: December 2, 2011
• Sponsor: Savient Pharmaceuticals
• Information provided by (Responsible Party): Savient Pharmaceuticals

Tracking Information

• First Submitted Date: October 27, 2008
• First Posted Date: May 19, 2011
• Results First Submitted Date: August 5, 2011
• Results First Posted Date: December 2, 2011
• Last Update Posted Date: December 2, 2011
• Study Start Date: December 2006
• Actual Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 28, 2011)

Uric Acid (mg/dL) [ Time Frame: Week 13, Week 25, Week 53, Week 101 ]

Uric acid measured at 3 month-intervals

Original Primary Outcome Measures (submitted: May 18, 2011)

Uric Acid (mg/dL) [ Time Frame: Up to 2 years ]

Uric acid measured at 3 month-intervals

Current Secondary Outcome Measures (submitted: October 28, 2011)

• Tophus Response [ Time Frame: Up to 2 years ]
  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.
• Patient Reported Outcome: SF-36 Physical Component Summary Score [ Time Frame: RCT Week 25; OLE Week 25; OLE Week 53, OLE Week 77, OLE Week 101 ]
  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.
• Gout Flare Frequency [ Time Frame: Up to 2 years ]
  The the mean number of flares per subject (flare frequency)was assessed over 3-month periods for up to 2 years of treatment
• Gout Flare Incidence [ Time Frame: Assessed in 3-month intervals up to 2 years ]
  Percentage of participants remaining in the study during the specified interval who experienced a gout flare during this interval.

Original Secondary Outcome Measures (submitted: May 18, 2011)

• Tophus Response [ Time Frame: Up to 2 years ]
  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
• Patient Reported outcome: SF-36 Physical Component Summary Score [ Time Frame: up to 2 years ]
  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 limitations in the dimensions related to physical function and pain.
• Gout Flare Incidence and Frequency [ Time Frame: Up to 2 years ]
  The number of subjects reporting flares (flare frequency) and the mean number of flares per subject (flare frequency)were assessed over 3-month periods for up to 2 years of treatment

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Open-Label Extension Study for Patients Who Completed a Phase 3 Double-blind Study of PEG-uricase for Symptomatic Gout
• Official Title: Multicenter, Open Label Extension Study of 8 mg PEG-uricase in Subjects Who Completed Protocols C0405 or C0406 for Symptomatic Gout
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Gout

Intervention

Biological: pegloticase

8 mg intravenous infusion

Study Arms

• Experimental: q2 RCT
  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
  Intervention: Biological: pegloticase
• Experimental: q4 RCT
  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
  Intervention: Biological: pegloticase
• Experimental: Placebo in RCT
  Placebo arm in Randomized Controlled Trial (RCT), received pegloticase every 2 weeks (q2 wk) or every 4 weeks (q4 wk) in OLE
  Intervention: Biological: pegloticase

Publications *

• Pillinger MH, Fields TR, Yeo AE, Lipsky PE. Dissociation Between Clinical Benefit and Persistent Urate Lowering in Patients with Chronic Refractory Gout Treated with Pegloticase. J Rheumatol. 2020 Apr;47(4):605-612. doi: 10.3899/jrheum.190161. Epub 2019 Jun 15.
• Lipsky PE, Calabrese LH, Kavanaugh A, Sundy JS, Wright D, Wolfson M, Becker MA. Pegloticase immunogenicity: the relationship between efficacy and antibody development in patients treated for refractory chronic gout. Arthritis Res Ther. 2014 Mar 4;16(2):R60. doi: 10.1186/ar4497.
• Baraf HS, Becker MA, Gutierrez-Urena SR, Treadwell EL, Vazquez-Mellado J, Rehrig CD, Ottery FD, Sundy JS, Yood RA. Tophus burden reduction with pegloticase: results from phase 3 randomized trials and open-label extension in patients with chronic gout refractory to conventional therapy. Arthritis Res Ther. 2013 Sep 26;15(5):R137. doi: 10.1186/ar4318.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 18, 2011): 151
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: January 2010
• Actual Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01356498
• Other Study ID Numbers: C0407
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Savient Pharmaceuticals
• Original Responsible Party: Same as current
• Current Study Sponsor: Savient Pharmaceuticals
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Medical Director, M.D.; Savient Pharmaceuticals, Inc.

• PRS Account: Savient Pharmaceuticals
• Verification Date: October 2011