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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.


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


  Show 67 Study Locations
Sponsors and Collaborators
Horizon Pharma Rheumatology LLC
Investigators
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:
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:
Gout
Arthritis
Joint Diseases
Musculoskeletal Diseases
Crystal Arthropathies
Rheumatic Diseases
Purine-Pyrimidine Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases