Observational Study of the Use of Pegloticase (KRYSTEXXA®) in Refractory Chronic Gout
EyesOnGOUT
1 other identifier
observational
188
1 country
67
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Nov 2011
Longer than P75 for all trials
67 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 28, 2011
CompletedFirst Posted
Study publicly available on registry
November 7, 2011
CompletedStudy Start
First participant enrolled
November 15, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2017
CompletedResults Posted
Study results publicly available
January 11, 2019
CompletedJanuary 11, 2019
June 1, 2018
5.6 years
October 28, 2011
June 29, 2018
June 29, 2018
Conditions
Outcome Measures
Primary Outcomes (3)
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: 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).
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).
Secondary Outcomes (5)
Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52
Week 24 and week 52
Change From Baseline in Number of Gout Flares
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
Baseline and weeks 24 and 52
Study Arms (1)
Pegloticase
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Interventions
Eligibility Criteria
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.
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (67)
Rheumatology Associates, PC
Birmingham, Alabama, 35205, United States
UAB Rheumatology
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
Related Publications (1)
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.
PMID: 21846852BACKGROUND
Related Links
Biospecimen
Serum samples
MeSH Terms
Interventions
Results Point of Contact
- Title
- Jeffery Nieves, PharmD
- Organization
- Horizon Pharma Rheumatology LLC
Study Officials
- STUDY DIRECTOR
Jeffery Nieves, PharmD
Horizon Pharma Rheumatology LLC
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 28, 2011
First Posted
November 7, 2011
Study Start
November 15, 2011
Primary Completion
June 30, 2017
Study Completion
June 30, 2017
Last Updated
January 11, 2019
Results First Posted
January 11, 2019
Record last verified: 2018-06