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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

Study Type Observational
Study Design Observational Model: Case-Only;   Time Perspective: Prospective
Condition Refractory Chronic Gout
Intervention Biological: Pegloticase
Enrollment 188
Recruitment Details A total of 249 patients were screened at 66 of 112 activated clinical sites in the United States; 61 (24.5%) of the 249 patients were screen failures and 188 were enrolled.
Pre-assignment Details  
Arm/Group Title Pegloticase
Hide Arm/Group Description Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Period Title: Overall Study
Started 188
Received Treatment 188
Completed 24 Weeks of Treatment 55
Completed [1] 25
Not Completed 163
Reason Not Completed
Death             1
Lost to Follow-up             7
Infusion Reaction (IR)             27
Adverse Event (Other than IR)             11
Elevated Serum Uric Acid             78
Withdrawal by Subject             13
Sponsor Decision             4
Protocol Violation/Noncompliance             2
Other             20
[1]
Patients completed the treatment phase of the study (on study for 52 weeks)
Arm/Group Title Pegloticase
Hide Arm/Group Description Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Baseline Participants 188
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 188 participants
55.32  (13.15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 188 participants
Female
19
  10.1%
Male
169
  89.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 188 participants
Hispanic or Latino
20
  10.6%
Not Hispanic or Latino
168
  89.4%
Unknown or Not Reported
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 188 participants
White
131
  69.7%
Asian
16
   8.5%
Black or African American
30
  16.0%
Native Hawaiian or Pacific Islander
1
   0.5%
American Indian or Alaskan Native
2
   1.1%
Other
8
   4.3%
Baseline Serum Uric Acid   [1] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 185 participants
9.03  (2.29)
[1]
Measure Analysis Population Description: Participants with available data
Duration Since Initial Gout Diagnosis  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 188 participants
15.54  (10.36)
Number of Gout Flares in Last 6 Months  
Mean (Standard Deviation)
Unit of measure:  Flares
Number Analyzed 188 participants
7.60  (12.17)
1.Primary Outcome
Title Number of Participants With Infusion Reactions
Hide Description 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.
Time Frame 52 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The intent-to-treat population included all enrolled participants
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Measure Type: Count of Participants
Unit of Measure: Participants
42
  22.3%
2.Primary Outcome
Title Number of Participants With Anaphylaxis
Hide Description

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).
Time Frame 52 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Measure Type: Count of Participants
Unit of Measure: Participants
5
   2.7%
3.Primary Outcome
Title Number of Participants With Immune Complex-related Events
Hide Description 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.
Time Frame From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Measure Type: Count of Participants
Unit of Measure: Participants
3
   1.6%
4.Secondary Outcome
Title Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52
Hide Description Normalization of serum uric acid was defined as serum uric acid value less than 6 mg/dL.
Time Frame Week 24 and week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with missing values at week 24 or 52 are counted as not achieving normalization
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Measure Type: Number
Unit of Measure: percentage of participants
Week 24 27.7
Week 52 12.2
5.Secondary Outcome
Title Change From Baseline in Number of Gout Flares
Hide Description 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.
Time Frame Baseline, week 24 and week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants with a value at baseline and each time point
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Mean (Standard Deviation)
Unit of Measure: flares
Week 24 Number Analyzed 46 participants
-0.83  (0.88)
Week 48 Number Analyzed 26 participants
-1.00  (0.89)
6.Secondary Outcome
Title Number of Swollen Joints Over Time
Hide Description [Not Specified]
Time Frame Baseline and weeks 24 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants with available data at baseline and each time point.
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Mean (Standard Deviation)
Unit of Measure: swollen joints
Baseline Number Analyzed 186 participants
8.60  (10.847)
Week 24 Number Analyzed 42 participants
4.05  (7.241)
Week 52 Number Analyzed 24 participants
1.46  (3.336)
7.Secondary Outcome
Title Number of Tender Joints Over Time
Hide Description [Not Specified]
Time Frame Baseline and weeks 24 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants with available data at baseline and each time point.
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Mean (Standard Deviation)
Unit of Measure: tender joints
Baseline Number Analyzed 186 participants
9.33  (11.715)
Week 24 Number Analyzed 42 participants
2.38  (5.539)
Week 52 Number Analyzed 24 participants
0.79  (1.318)
8.Secondary Outcome
Title Number of Palpable Tophi Over Time
Hide Description Gout tophi are nodular deposits of urate crystals and inflammatory cells in joints, soft tissues, bones, and in some organs.
Time Frame Baseline and weeks 24 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants with available data at baseline and each time point.
Arm/Group Title Pegloticase
Hide Arm/Group Description:
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Overall Number of Participants Analyzed 188
Mean (Standard Deviation)
Unit of Measure: tophi
Baseline Number Analyzed 161 participants
13.58  (18.999)
Week 24 Number Analyzed 33 participants
5.70  (5.643)
Week 52 Number Analyzed 19 participants
3.68  (3.038)
Time Frame From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Pegloticase
Hide Arm/Group Description Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
All-Cause Mortality
Pegloticase
Affected / at Risk (%)
Total   3/188 (1.60%) 
Show Serious Adverse Events Hide Serious Adverse Events
Pegloticase
Affected / at Risk (%)
Total   31/188 (16.49%) 
Blood and lymphatic system disorders   
Haemolytic anaemia  1  1/188 (0.53%) 
Cardiac disorders   
Atrial fibrillation  1  1/188 (0.53%) 
Cardiac failure  1  1/188 (0.53%) 
Cardiac failure congestive  1  2/188 (1.06%) 
Coronary artery disease  1  1/188 (0.53%) 
Congenital, familial and genetic disorders   
Gastrointestinal arteriovenous malformation  1  1/188 (0.53%) 
Gastrointestinal disorders   
Large intestinal haemorrhage  1  1/188 (0.53%) 
Pancreatitis  1  1/188 (0.53%) 
Upper gastrointestinal haemorrhage  1  1/188 (0.53%) 
Immune system disorders   
Anaphylaxis  1  5/188 (2.66%) 
Drug hypersensitivity  1  3/188 (1.60%) 
Infections and infestations   
Cellulitis  1  2/188 (1.06%) 
Pneumonia  1  1/188 (0.53%) 
Septic shock  1  1/188 (0.53%) 
Urinary tract infection  1  1/188 (0.53%) 
Metabolism and nutrition disorders   
Gout  1  2/188 (1.06%) 
Hyperglycaemia  1  1/188 (0.53%) 
Hypoglycaemia  1  1/188 (0.53%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/188 (0.53%) 
Osteoarthritis  1  1/188 (0.53%) 
Pain in extremity  1  1/188 (0.53%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Renal cancer  1  1/188 (0.53%) 
Nervous system disorders   
Cerebral haemorrhage  1  1/188 (0.53%) 
Transient ischaemic attack  1  1/188 (0.53%) 
Psychiatric disorders   
Mental status changes  1  2/188 (1.06%) 
Renal and urinary disorders   
Renal failure  1  1/188 (0.53%) 
Renal failure acute  1  4/188 (2.13%) 
Respiratory, thoracic and mediastinal disorders   
Acute respiratory failure  1  2/188 (1.06%) 
Pneumothorax spontaneous  1  1/188 (0.53%) 
Skin and subcutaneous tissue disorders   
Subcutaneous emphysema  1  1/188 (0.53%) 
Vascular disorders   
Hypertension  1  1/188 (0.53%) 
Hypotension  1  1/188 (0.53%) 
1
Term from vocabulary, MedDRA 17.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Pegloticase
Affected / at Risk (%)
Total   130/188 (69.15%) 
Immune system disorders   
Drug hypersensitivity  1  11/188 (5.85%) 
Metabolism and nutrition disorders   
Gout  1  126/188 (67.02%) 
1
Term from vocabulary, MedDRA 17.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jeffery Nieves, PharmD
Organization: Horizon Pharma Rheumatology LLC
Phone: 224-383-3000
EMail: clinicaltrials@horizonpharma.com
Layout table for additonal information
Responsible Party: Horizon Pharma Rheumatology LLC
ClinicalTrials.gov Identifier: NCT01466166     History of Changes
Other Study ID Numbers: M0401
First Submitted: October 28, 2011
First Posted: November 7, 2011
Results First Submitted: June 29, 2018
Results First Posted: January 11, 2019
Last Update Posted: January 11, 2019