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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

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.

Condition or disease Intervention/treatment
Refractory Chronic Gout Biological: Pegloticase

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.


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Study Type : Observational
Actual Enrollment : 188 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
Actual Study Start Date : November 15, 2011
Actual Primary Completion Date : June 30, 2017
Actual Study Completion Date : June 30, 2017

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Gout
MedlinePlus related topics: Gout
Drug Information available for: Pegloticase

Group/Cohort Intervention/treatment
Pegloticase
Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician.
Biological: Pegloticase
Pegloticase 8 mg intravenous every 2 weeks
Other Name: KRYSTEXXA®




Primary Outcome Measures :
  1. 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.

  2. 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).

  3. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. Number of Swollen Joints Over Time [ Time Frame: Baseline and weeks 24 and 52 ]
  4. Number of Tender Joints Over Time [ Time Frame: Baseline and weeks 24 and 52 ]
  5. 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.


Biospecimen Retention:   Samples Without DNA
Serum samples


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01466166


  Hide Study Locations
Locations
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United States, Alabama
Rheumatology Associates, PC
Birmingham, Alabama, United States, 35205
UAB Rheumatology
Birmingham, Alabama, United States, 35294
Saadat Ansari, MD, LLC
Huntsville, Alabama, United States, 35801
United States, California
Medvin Clinical Research
Covina, California, United States, 91723
Alliance Clinical Research, LLC
Laguna Hills, California, United States, 92653
Advanced Medical Research, LLC
Lakewood, California, United States, 90712
Pacific Arthritis Care Center
Los Angeles, California, United States, 90045
R Srinivasan, MD, Inc
Monterey Park, California, United States, 91754
Brigid Freyne, MD, Inc
Murrieta, California, United States, 92563
Alliance Clinical Research
Poway, California, United States, 92064
United States, Colorado
Denver Nephrologists, PC
Denver, Colorado, United States, 80218
United States, Connecticut
New England Research Associates, LLC
Trumbull, Connecticut, United States, 06611
United States, District of Columbia
Howard University Hospital
Washington, District of Columbia, United States, 20060
Washington DC Veteran's Affairs Medical Center
Washington, District of Columbia, United States, 20422
United States, Florida
Bay Area Arthritis and Osteoporosis
Brandon, Florida, United States, 33511
Countryside Arthritis Center
Clearwater, Florida, United States, 33761
Science and Research Institute, Inc
Jupiter, Florida, United States, 33458
A & O Research Center
Miami, Florida, United States, 33174
Arthritis Research of Florida, Inc.
Palm Harbor, Florida, United States, 34684
Family Clinical Trials, LLC
Pembroke Pines, Florida, United States, 33026
Jedidiah Clinical Research
Tampa, Florida, United States, 33604
Midtown Medical Center
Tampa, Florida, United States, 33614
United States, Georgia
Global Research Partners & Consultants, Inc.
Calhoun, Georgia, United States, 30701
Arthritis Research & Treatment Center
Stockbridge, Georgia, United States, 30281
United States, Illinois
University of Chicago Medical Center
Chicago, Illinois, United States, 60637
United States, Indiana
Diagnostic Rheumatology and Research PC
Indianapolis, Indiana, United States, 46227
United States, Iowa
Physicians' Clinic of Iowa, P.C.
Cedar Rapids, Iowa, United States, 52401
United States, Kentucky
Central Kentucky Research Associates of Kentucky
Mount Sterling, Kentucky, United States, 40353
Research Integrity, LLC
Owensboro, Kentucky, United States, 42303
United States, Louisiana
Horizon Research Group of Opelousas, LLC
Eunice, Louisiana, United States, 70535
Arthritis and Diabetes Clinic, Inc.
Monroe, Louisiana, United States, 71203
United States, Maryland
Walter Reed National Military Medical Center
Bethesda, Maryland, United States, 20889-5630
Klein & Associates MD, PA.
Hagerstown, Maryland, United States, 21740
The Center for Rheumatology and Bone Research
Wheaton, Maryland, United States, 20902
United States, Massachusetts
Clinical Pharmacology Study Groups
Worcester, Massachusetts, United States, 01605
Reliant Medical Group, Inc.
Worcester, Massachusetts, United States, 01605
United States, Michigan
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109-5422
Caro Health Plaza
Caro, Michigan, United States, 48723
Infusion Associates
Grand Rapids, Michigan, United States, 49525
Justus J. Fiechtner, MD, PC
Lansing, Michigan, United States, 48910
Shores Rheumatology, PC
Saint Clair Shores, Michigan, United States, 48081
United States, Minnesota
Saint Paul Rheumatology, PA
Eagan, Minnesota, United States, 55121
United States, Missouri
Kansas City Internal Medicine
Kansas City, Missouri, United States, 64114
United States, Nevada
Arthritis Medical Clinic
Las Vegas, Nevada, United States, 89118
United States, New Jersey
Rheumatology Associates of North Jersey
Teaneck, New Jersey, United States, 07666
United States, New York
Rheumatology Associates of Long Island
Smithtown, New York, United States, 11787
United States, North Carolina
NorthEast Rheumatology
Concord, North Carolina, United States, 28025
Duke University Medical Center
Durham, North Carolina, United States, 27710
Physicians East, PA
Greenville, North Carolina, United States, 27832
Shanahan Rheumatology and Immunotherapy, PLLC
Raleigh, North Carolina, United States, 27617
Specialty Medical Clinic and Research Center
Sanford, North Carolina, United States, 27330
United States, Ohio
Southern Ohio Rheumatology
Portsmouth, Ohio, United States, 45662
United States, Pennsylvania
Keystone Pain Institute, Ilumina Clinical Associates
Altoona, Pennsylvania, United States, 16602
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
United States, South Carolina
Low Country Rheumatology
Charleston, South Carolina, United States, 29406
Acme Research L.L.C.
Orangeburg, South Carolina, United States, 29118
United States, Tennessee
Ramesh C. Gupta, M.D.
Memphis, Tennessee, United States, 38119
United States, Texas
Austin Regional Clinic
Austin, Texas, United States, 78731
Dr. Raj Marwah
El Paso, Texas, United States, 79902
Diagnostic Clinic of Houston
Houston, Texas, United States, 77004
Rheumatic Disease Clinical Research Center
Houston, Texas, United States, 77004
United States, Virginia
Arthritis Clinic of Northern Virginia, P.C.
Arlington, Virginia, United States, 22205
Arthritis & Osteoporosis Center of North Virginia
Manassas, Virginia, United States, 20109
Sentara Rheumatology Specialists
Norfolk, Virginia, United States, 23502
United States, Washington
Apex Clinical Research
Kennewick, Washington, United States, 99336
United States, West Virginia
Mountain State Clinical Research
Clarksburg, West Virginia, United States, 26301
United States, Wisconsin
Rheumatic Disease Center, LLP
Glendale, Wisconsin, United States, 53217
Sponsors and Collaborators
Horizon Pharma Rheumatology LLC
Investigators
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Study Director: Jeffery Nieves, PharmD Horizon Pharma Rheumatology LLC
  Study Documents (Full-Text)

Documents provided by Horizon Pharma Rheumatology LLC:
Study Protocol  [PDF] July 11, 2011
Statistical Analysis Plan  [PDF] March 25, 2018


Additional Information:
Publications:
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Responsible Party: Horizon Pharma Rheumatology LLC
ClinicalTrials.gov Identifier: NCT01466166     History of Changes
Other Study ID Numbers: M0401
First Posted: November 7, 2011    Key Record Dates
Results First Posted: January 11, 2019
Last Update Posted: January 11, 2019
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Additional relevant MeSH terms:
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Gout
Arthritis
Joint Diseases
Musculoskeletal Diseases
Crystal Arthropathies
Rheumatic Diseases
Purine-Pyrimidine Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases