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Observational Study of the Use of Pegloticase (KRYSTEXXA®) in Refractory Chronic Gout (EyesOnGOUT)

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ClinicalTrials.gov Identifier: NCT01466166
Recruitment Status : Completed
First Posted : November 7, 2011
Results First Posted : January 11, 2019
Last Update Posted : January 11, 2019
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Rheumatology LLC

Tracking Information
First Submitted Date October 28, 2011
First Posted Date November 7, 2011
Results First Submitted Date June 29, 2018
Results First Posted Date January 11, 2019
Last Update Posted Date January 11, 2019
Actual Study Start Date November 15, 2011
Actual Primary Completion Date June 30, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 29, 2018)
  • Number of Participants With Infusion Reactions [ Time Frame: 52 weeks ]
    Infusion reactions were defined as adverse events (AEs) or clusters of events, not attributable to another cause that occurred during or within 2 hours after the infusion of pegloticase. Any other case that occurred outside of the 2-hour window was categorized per Investigator discretion.
  • Number of Participants With Anaphylaxis [ Time Frame: 52 weeks ]
    Anaphylaxis was defined using the National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network (NIAID/FAAN) criteria: Acute onset of an illness (minutes to several hours) with involvement of the skin, mucosal tissue, or both (e.g., generalized hives; pruritus or flushing; swollen lips, tongue, or uvula), and at least 1 of the following:
    1. Respiratory compromise (e.g., dyspnea, wheeze-bronchospasm, stridor, reduced peak expiratory flow, hypoxemia).
    2. Reduced blood pressure (i.e., systolic blood pressure < 90 mm Hg or greater than 30% decrease from that patient's baseline) or associated symptoms of end-organ failure (e.g., hypotonia [collapse], syncope, incontinence).
  • Number of Participants With Immune Complex-related Events [ Time Frame: From first dose of study drug to the end of the 12-week follow-up period (63 weeks). ]
    Immune complex-related events were defined as any presumptive immune complex-related disorders that were confirmed by an appropriate investigation of the event and of complement markers (C3 and C4 levels). Clinical manifestations could have included skin rash, arthralgia, arthritis, proteinuria, serum sickness, and cryoglobulinemia.
Original Primary Outcome Measures
 (submitted: November 4, 2011)
Number of participants with pre-specified adverse events [ Time Frame: up to 63 weeks ]
The number (percentage) of participants with an infusion reaction, anaphylaxis or immune complex related adverse event at any time in the study will be summarized.
Change History Complete list of historical versions of study NCT01466166 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: June 29, 2018)
  • Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52 [ Time Frame: Week 24 and week 52 ]
    Normalization of serum uric acid was defined as serum uric acid value less than 6 mg/dL.
  • Change From Baseline in Number of Gout Flares [ Time Frame: Baseline, week 24 and week 48 ]
    The number of gout flares occurring in the 2 weeks prior to each visit. Baseline number of flares was calculated as the average number of flares that occurred in the 6-month baseline period divided by 12 weeks.
  • Number of Swollen Joints Over Time [ Time Frame: Baseline and weeks 24 and 52 ]
  • Number of Tender Joints Over Time [ Time Frame: Baseline and weeks 24 and 52 ]
  • Number of Palpable Tophi Over Time [ Time Frame: Baseline and weeks 24 and 52 ]
    Gout tophi are nodular deposits of urate crystals and inflammatory cells in joints, soft tissues, bones, and in some organs.
Original Secondary Outcome Measures
 (submitted: November 4, 2011)
  • Serum uric acid response [ Time Frame: Up to 51 weeks ]
    Treatment responder will be defines as a participant whose serum uric acid values were <6 mg/dL at their final 2 consecutive (on drug) study visits.
  • Change from Baseline in Number of Swollen Joints at 25 weeks [ Time Frame: 25 weeks ]
  • Change from Baseline in Number of Swollen Joints at 51 weeks [ Time Frame: 51 weeks ]
  • Change from Baseline in Number of Tender Joints at 25 weeks [ Time Frame: 25 weeks ]
  • Change from Baseline in Number of Tender Joints at 51 weeks [ Time Frame: 51 weeks ]
  • Change from Baseline in Number of Tophi at 25 weeks [ Time Frame: 25 weeks ]
  • Change from Baseline in Number of Tophi at 51 weeks [ Time Frame: 51 weeks ]
  • Change from Baseline in Number of Swollen Joints at Early Discontinuation [ Time Frame: up to 50 weeks ]
  • Change from Baseline in Number of Tender Joints at Early Discontinuation [ Time Frame: up to 50 weeks ]
  • Change from Baseline in Number of Tophi at Early Discontinuation [ Time Frame: up to 50 weeks ]
  • Change from Baseline in Number of Gout Flares at 6 weeks [ Time Frame: 6 weeks ]
    Baseline refers to participant's reported history upon enrollment.
  • Change from Baseline in Number of Gout Flares in weeks 6-12 [ Time Frame: Weeks 6 to 12 ]
  • Change from Baseline in Number of Gout Flares in Months 3-6 [ Time Frame: Months 3 to 6 ]
  • Change from Baseline in Number of Gout Flares in Months 6-9 [ Time Frame: Months 6 to 9 ]
  • Change from Baseline in Number of Gout Flares in Months 9-12 [ Time Frame: Months 9 to 12 ]
  • Change from Baseline in Number of Gout Flares in Months 12-15 [ Time Frame: Months 12 to 15 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Observational Study of the Use of Pegloticase (KRYSTEXXA®) in Refractory Chronic Gout
Official Title Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
Brief Summary The primary purpose of this study is to observe patients being treated with pegloticase in a standard healthcare setting in order to evaluate the frequency and severity of infusion reactions, anaphylaxis and immune complex related events. Additionally, serious adverse events associated with pegloticase therapy will be identified.
Detailed Description

This was a Phase 4, multicenter, open-label, single-arm observational study of pegloticase 8 mg administered intravenously every 2 weeks in adult hyperuricemic patients with gout refractory to conventional therapy. Study duration is approximately 63 weeks, including 51 weeks of treatment and 12 weeks of follow-up.

The design of this study follows the FDA-approved Full Prescribing Information for the use of pegloticase and allows for capturing additional data related to the safety and efficacy of pegloticase within the standard healthcare setting.

Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples Without DNA
Description:
Serum samples
Sampling Method Non-Probability Sample
Study Population The patient population in this study will be hyperuricemic (serum uric acid (SUA) > 6 mg/dL) adult men and women (age 18 or greater) diagnosed with chronic gout and who are refractory to conventional therapy. Gout refractory to conventional therapy occurs in patients who have failed to normalize SUA and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated. To enter this study, the patient and the physician must have decided to begin treatment with KRYSTEXXA.
Condition Refractory Chronic Gout
Intervention Biological: Pegloticase
Pegloticase 8 mg intravenous every 2 weeks
Other Name: KRYSTEXXA®
Study Groups/Cohorts Pegloticase
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Intervention: Biological: Pegloticase
Publications * Sundy JS, Baraf HS, Yood RA, Edwards NL, Gutierrez-Urena SR, Treadwell EL, Vázquez-Mellado J, White WB, Lipsky PE, Horowitz Z, Huang W, Maroli AN, Waltrip RW 2nd, Hamburger SA, Becker MA. Efficacy and tolerability of pegloticase for the treatment of chronic gout in patients refractory to conventional treatment: two randomized controlled trials. JAMA. 2011 Aug 17;306(7):711-20. doi: 10.1001/jama.2011.1169.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: June 29, 2018)
188
Original Estimated Enrollment
 (submitted: November 4, 2011)
1500
Actual Study Completion Date June 30, 2017
Actual Primary Completion Date June 30, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Adults (age 18 years or more) with chronic gout refractory to conventional therapy, defined as patients who have failed to normalize SUA and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose, or for whom these drugs are contraindicated.
  • Patients who have made the decision, along with their treating physician, to begin treatment with KRYSTEXXA.
  • Patients who are willing and able to give informed consent and adhere to visit/protocol schedules.

Exclusion Criteria:

  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency
  • Non-compensated congestive heart failure
  • Pregnancy or breast feeding
  • Prior treatment with pegloticase or another recombinant uricase
  • Known allergy to urate oxidase
  • Prior treatment or concomitant therapy with a polyethylene glycol (PEG)-conjugated drug
  • Recipient of an investigational drug within 4 weeks prior to study drug administration or plans to take an investigational agent during the study
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
Removed Location Countries  
 
Administrative Information
NCT Number NCT01466166
Other Study ID Numbers M0401
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: Undecided
Responsible Party Horizon Pharma Rheumatology LLC
Study Sponsor Horizon Pharma Rheumatology LLC
Collaborators Not Provided
Investigators
Study Director: Jeffery Nieves, PharmD Horizon Pharma Rheumatology LLC
PRS Account Horizon Pharma Rheumatology LLC
Verification Date June 2018