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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pegloticase | Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pegloticase | Biological | Pegloticase 8 mg intravenous every 2 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Infusion Reactions | 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. | 52 weeks |
| Number of Participants With Anaphylaxis | 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:
| 52 weeks |
| Number of Participants With Immune Complex-related Events | 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. | From first dose of study drug to the end of the 12-week follow-up period (63 weeks). |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52 | Normalization of serum uric acid was defined as serum uric acid value less than 6 mg/dL. | Week 24 and week 52 |
| Change From Baseline in Number of Gout Flares |
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Inclusion Criteria:
Exclusion Criteria:
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Jeffery Nieves, PharmD | Horizon Pharma Rheumatology LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rheumatology Associates, PC | Birmingham | Alabama | 35205 | United States | ||
| UAB Rheumatology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21846852 | Background | Sundy JS, Baraf HS, Yood RA, Edwards NL, Gutierrez-Urena SR, Treadwell EL, Vazquez-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. |
| Label | URL |
|---|---|
| Related Info | View source |
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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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Pegloticase | Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 11, 2011 | Jun 29, 2018 |
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Serum samples
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.
| Baseline, week 24 and week 48 |
| Number of Swollen Joints Over Time | Baseline and weeks 24 and 52 |
| Number of Tender Joints Over Time | Baseline and weeks 24 and 52 |
| Number of Palpable Tophi Over Time | Gout tophi are nodular deposits of urate crystals and inflammatory cells in joints, soft tissues, bones, and in some organs. | Baseline and weeks 24 and 52 |
| Birmingham |
| Alabama |
| 35294 |
| United States |
| Saadat Ansari, MD, LLC | Huntsville | Alabama | 35801 | United States |
| Medvin Clinical Research | Covina | California | 91723 | United States |
| Alliance Clinical Research, LLC | Laguna Hills | California | 92653 | United States |
| Advanced Medical Research, LLC | Lakewood | California | 90712 | United States |
| Pacific Arthritis Care Center | Los Angeles | California | 90045 | United States |
| R Srinivasan, MD, Inc | Monterey Park | California | 91754 | United States |
| Brigid Freyne, MD, Inc | Murrieta | California | 92563 | United States |
| Alliance Clinical Research | Poway | California | 92064 | United States |
| Denver Nephrologists, PC | Denver | Colorado | 80218 | United States |
| New England Research Associates, LLC | Trumbull | Connecticut | 06611 | United States |
| Howard University Hospital | Washington D.C. | District of Columbia | 20060 | United States |
| Washington DC Veteran's Affairs Medical Center | Washington D.C. | District of Columbia | 20422 | United States |
| Bay Area Arthritis and Osteoporosis | Brandon | Florida | 33511 | United States |
| Countryside Arthritis Center | Clearwater | Florida | 33761 | United States |
| Science and Research Institute, Inc | Jupiter | Florida | 33458 | United States |
| A & O Research Center | Miami | Florida | 33174 | United States |
| Arthritis Research of Florida, Inc. | Palm Harbor | Florida | 34684 | United States |
| Family Clinical Trials, LLC | Pembroke Pines | Florida | 33026 | United States |
| Jedidiah Clinical Research | Tampa | Florida | 33604 | United States |
| Midtown Medical Center | Tampa | Florida | 33614 | United States |
| Global Research Partners & Consultants, Inc. | Calhoun | Georgia | 30701 | United States |
| Arthritis Research & Treatment Center | Stockbridge | Georgia | 30281 | United States |
| University of Chicago Medical Center | Chicago | Illinois | 60637 | United States |
| Diagnostic Rheumatology and Research PC | Indianapolis | Indiana | 46227 | United States |
| Physicians' Clinic of Iowa, P.C. | Cedar Rapids | Iowa | 52401 | United States |
| Central Kentucky Research Associates of Kentucky | Mount Sterling | Kentucky | 40353 | United States |
| Research Integrity, LLC | Owensboro | Kentucky | 42303 | United States |
| Horizon Research Group of Opelousas, LLC | Eunice | Louisiana | 70535 | United States |
| Arthritis and Diabetes Clinic, Inc. | Monroe | Louisiana | 71203 | United States |
| Walter Reed National Military Medical Center | Bethesda | Maryland | 20889-5630 | United States |
| Klein & Associates MD, PA. | Hagerstown | Maryland | 21740 | United States |
| The Center for Rheumatology and Bone Research | Wheaton | Maryland | 20902 | United States |
| Clinical Pharmacology Study Groups | Worcester | Massachusetts | 01605 | United States |
| Reliant Medical Group, Inc. | Worcester | Massachusetts | 01605 | United States |
| University of Michigan Health System | Ann Arbor | Michigan | 48109-5422 | United States |
| Caro Health Plaza | Caro | Michigan | 48723 | United States |
| Infusion Associates | Grand Rapids | Michigan | 49525 | United States |
| Justus J. Fiechtner, MD, PC | Lansing | Michigan | 48910 | United States |
| Shores Rheumatology, PC | Saint Clair Shores | Michigan | 48081 | United States |
| Saint Paul Rheumatology, PA | Eagan | Minnesota | 55121 | United States |
| Kansas City Internal Medicine | Kansas City | Missouri | 64114 | United States |
| Arthritis Medical Clinic | Las Vegas | Nevada | 89118 | United States |
| Rheumatology Associates of North Jersey | Teaneck | New Jersey | 07666 | United States |
| Rheumatology Associates of Long Island | Smithtown | New York | 11787 | United States |
| NorthEast Rheumatology | Concord | North Carolina | 28025 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Physicians East, PA | Greenville | North Carolina | 27832 | United States |
| Shanahan Rheumatology and Immunotherapy, PLLC | Raleigh | North Carolina | 27617 | United States |
| Specialty Medical Clinic and Research Center | Sanford | North Carolina | 27330 | United States |
| Southern Ohio Rheumatology | Portsmouth | Ohio | 45662 | United States |
| Keystone Pain Institute, Ilumina Clinical Associates | Altoona | Pennsylvania | 16602 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Low Country Rheumatology | Charleston | South Carolina | 29406 | United States |
| Acme Research L.L.C. | Orangeburg | South Carolina | 29118 | United States |
| Ramesh C. Gupta, M.D. | Memphis | Tennessee | 38119 | United States |
| Austin Regional Clinic | Austin | Texas | 78731 | United States |
| Dr. Raj Marwah | El Paso | Texas | 79902 | United States |
| Diagnostic Clinic of Houston | Houston | Texas | 77004 | United States |
| Rheumatic Disease Clinical Research Center | Houston | Texas | 77004 | United States |
| Arthritis Clinic of Northern Virginia, P.C. | Arlington | Virginia | 22205 | United States |
| Arthritis & Osteoporosis Center of North Virginia | Manassas | Virginia | 20109 | United States |
| Sentara Rheumatology Specialists | Norfolk | Virginia | 23502 | United States |
| Apex Clinical Research | Kennewick | Washington | 99336 | United States |
| Mountain State Clinical Research | Clarksburg | West Virginia | 26301 | United States |
| Rheumatic Disease Center, LLP | Glendale | Wisconsin | 53217 | United States |
| Received Treatment |
|
| Completed 24 Weeks of Treatment |
|
| COMPLETED | Patients completed the treatment phase of the study (on study for 52 weeks) |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Pegloticase | Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Baseline Serum Uric Acid | Participants with available data | Mean | Standard Deviation | mg/dL |
| ||||||||||||||||
| Duration Since Initial Gout Diagnosis | Mean | Standard Deviation | years |
| |||||||||||||||||
| Number of Gout Flares in Last 6 Months | Mean | Standard Deviation | flares |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Infusion Reactions | 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. | The intent-to-treat population included all enrolled participants | Posted | Count of Participants | Participants | 52 weeks |
|
|
| ||||||||||||||||||||||||||
| Primary | Number of Participants With Anaphylaxis | 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:
| All enrolled participants | Posted | Count of Participants | Participants | 52 weeks |
|
| |||||||||||||||||||||||||||
| Primary | Number of Participants With Immune Complex-related Events | 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. | All enrolled participants | Posted | Count of Participants | Participants | From first dose of study drug to the end of the 12-week follow-up period (63 weeks). |
|
| |||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52 | Normalization of serum uric acid was defined as serum uric acid value less than 6 mg/dL. | Participants with missing values at week 24 or 52 are counted as not achieving normalization | Posted | Number | percentage of participants | Week 24 and week 52 |
|
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Number of Gout Flares | 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. | Enrolled participants with a value at baseline and each time point | Posted | Mean | Standard Deviation | flares | Baseline, week 24 and week 48 |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Swollen Joints Over Time | Enrolled participants with available data at baseline and each time point. | Posted | Mean | Standard Deviation | swollen joints | Baseline and weeks 24 and 52 |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Tender Joints Over Time | Enrolled participants with available data at baseline and each time point. | Posted | Mean | Standard Deviation | tender joints | Baseline and weeks 24 and 52 |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Palpable Tophi Over Time | Gout tophi are nodular deposits of urate crystals and inflammatory cells in joints, soft tissues, bones, and in some organs. | Enrolled participants with available data at baseline and each time point. | Posted | Mean | Standard Deviation | tophi | Baseline and weeks 24 and 52 |
|
|
From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pegloticase | Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician. | 3 | 188 | 31 | 188 | 130 | 188 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Gastrointestinal arteriovenous malformation | Congenital, familial and genetic disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Large intestinal haemorrhage | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Anaphylaxis | Immune system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
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| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Renal cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 17.1 | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Subcutaneous emphysema | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 17.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Drug hypersensitivity | Immune system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jeffery Nieves, PharmD | Horizon Pharma Rheumatology LLC | 224-383-3000 | clinicaltrials@horizonpharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 25, 2018 | Jun 29, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| C031545 | Pegloticase |
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| Unknown or Not Reported |
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| Black or African American |
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| Native Hawaiian or Pacific Islander |
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| American Indian or Alaskan Native |
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| Other |
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