Pegloticase
BiologicalPegloticase 8 mg intravenous every 2 weeks
Other names: KRYSTEXXA®
Completed
NCT Number: NCT01466166
Observational Study of the Use of Pegloticase (KRYSTEXXA®) in Refractory Chronic Gout
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.
Completed
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Conditions
Age range
18 year and older
Sex eligibility
All sexes
Study type
Observational
Primary location
Rheumatology Associates, PC, Birmingham, Alabama, United States
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About this study
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.
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
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
Treatment and study plan
Primary outcomes
-
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.
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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:
- Respiratory compromise (e.g., dyspnea, wheeze-bronchospasm, stridor, reduced peak expiratory flow, hypoxemia).
- 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).
-
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 outcomes
-
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.
-
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.
-
Number of Swollen Joints Over Time
Time frame: Baseline and weeks 24 and 52
-
Number of Tender Joints Over Time
Time frame: Baseline and weeks 24 and 52
-
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.
Sponsors and collaborators
Lead sponsor
Horizon Pharma Rheumatology LLC
Industry
Registry information
Official study title
Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
Acronym: EyesOnGOUT
Important dates
- Study start
- 2011
- Primary completion
- 2017
- Study completion
- 2017
- First posted
- Nov 7, 2011
- Registry last updated
- Jan 11, 2019
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.