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Completed

NCT Number: NCT04511702

Infusion Duration Study To Assess Tolerability of Pegloticase Administered With a Shorter Infusion Duration in Subjects With Uncontrolled Gout Receiving Methotrexate

The purpose of this study is to assess the safety, tolerability and efficiency of pegloticase administered with a shorter infusion duration in participants with uncontrolled gout receiving methotrexate.

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Key information

Conditions

Chronic Uncontrolled Gout Arthritis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Crystal Arthropathies Genetic Diseases, Inborn Gout Joint Diseases Metabolic Diseases Metabolism, Inborn Errors Musculoskeletal Diseases Nutritional and Metabolic Diseases Purine-Pyrimidine Metabolism, Inborn Errors Rheumatic Diseases Uncontrolled Gout

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

University of Alabama at Birmingham, Birmingham, Alabama, United States

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About this study

Approximately 180 participants will be enrolled. After a 4-week methotrexate run-in period, participants will be treated for up to 24 weeks with weekly oral methotrexate and biweekly 8mg pegloticase infusions. Up to three pegloticase infusion durations will be assessed in the study: 60-minute infusion, 45-minute infusion and 30-minute infusion. Safety evaluations will be performed regularly throughout the course of the study.

Acquired from Horizon in 2024.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adult men or women ≥18 years of age.
  • Uncontrolled gout, defined as meeting the following criteria:
  • Hyperuricemia during the screening period defined as sUA ≥6 mg/dL
  • Failure to maintain normalization of sUA with xanthine oxidase inhibitors at the maximum medically appropriate dose, or with intolerable side effects or a contraindication to xanthine oxidase inhibitor therapy based on medical record review or subject interview.
  • Symptoms of gout including at least 1 of the following:
  • Presence of at least one tophus
  • Recurrent flares defined as 2 or more flares in the past 12 months prior to screening
  • Presence of chronic gouty arthritis
  • Willing to discontinue all oral urate-lowering therapy at least 7 days prior to MTX dosing at Week -4 and remain off of urate lowering therapy when receiving pegloticase infusions during the study.

Exclusion criteria

  • Severe chronic or recurrent bacterial infections, such as recurrent pneumonia or chronic bronchiectasis.
  • Current or chronic treatment with systemic immunosuppressive agents such as MTX, azathioprine, or mycophenolate mofetil; prednisone ≥10 mg/day or equivalent dose of other corticosteroid on a chronic basis (defined as 3 months or longer) would also meet exclusion criteria.
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency (tested at Screening Visit).
  • Severe chronic renal impairment (estimated glomerular filtration rate <40 mL/min/1.73 m2) at the Screening Visit based on 4 variable-Modification of Diet in Renal Disease [MDRD] formula or currently on dialysis.
  • Non-compensated congestive heart failure or hospitalization for congestive heart failure or treatment for acute coronary syndrome (myocardial infarction or unstable angina) within 3 months of the Screening Visit, or current uncontrolled arrhythmia, or current uncontrolled blood pressure (BP) (>160/100 mmHg) prior to Week -4.
  • Pregnant, planning to become pregnant, breastfeeding, planning to impregnate female partner, or not on an effective form of birth control, as determined by the Investigator.
  • Prior treatment with pegloticase (KRYSTEXXA), another recombinant uricase (rasburicase), or concomitant therapy with a polyethylene glycol-conjugated drug.
  • Currently receiving systemic or radiologic treatment for ongoing cancer.
  • History of malignancy within 5 years other than non-melanoma skin cancer or in situ carcinoma of cervix.

Treatment and study plan

Pegloticase with MTX

Biological

Participants will receive MTX during the run-in period then pegloticase with MTX for up to 24 weeks during the treatment period

Other names: Methotrexate

Primary outcomes

  1. Percentage of Participants Who Experienced an Infusion Reaction (IR), Including Anaphylaxis, Related to Pegloticase From Day 1 to Week 24

    Time frame: Day 1 to Week 24

    IRs, including anaphylaxis, were assessed after each infusion for a period of 24 weeks. IRs were defined as any infusion-related adverse event (AE) or cluster of temporally-related AEs, not attributable to another cause, which occurred during the pegloticase infusion and up to 2 hours post-infusion. Additional AEs occurring outside of the 2-hour window following infusion could also be categorized as an IR at the Principal Investigator's discretion.

Secondary outcomes

  1. Percentage of Serum Uric Acid (sUA) Responders at Month 6

    Time frame: Up to 6 months

    Responders were defined as participants who achieved and maintained sUA levels below 6 mg/dL for at least 80% of the time during month 6.

    Responders who met sUA discontinuation criteria (2 consecutive sUA > 6 mg/dL), regardless of treatment status were considered non-responders.

  2. Percentage of Participants Who Experienced an IR Leading to Discontinuation of Treatment, Anaphylaxis or Met sUA Discontinuation Criteria

    Time frame: Day 1 to Week 24

    IRs were defined as any infusion-related AE or cluster of temporally-related AEs, not attributable to another cause, which occurred during the pegloticase infusion and for 2 hours post-infusion. Other AEs that occurred outside of the 2 hour window following the infusion may have also been categorized as an IR at the Principal Investigator's discretion.

    Anaphylaxis was defined using the National Institute of Allergy and Infectious Diseases/Food Allergy and Anaphylaxis Network criteria.

    Meeting individual sUA discontinuation criteria was defined as two consecutive pre-infusion sUAs > 6 mg/dL after Day 1.

  3. Time to First Event of IR Leading to Discontinuation of Treatment, Anaphylaxis or Meeting sUA Discontinuation Criteria in All Participants

    Time frame: Day 1 to Week 24

    Infusion reactions were defined as any infusion-related adverse event or cluster of temporally-related adverse events, not attributable to another cause, which occurred during the infusion and for 2 hours post-infusion. Other adverse events that occurred outside of the 2-hour window following the infusion may have also been categorized as an infusion reaction at the Principal Investigator's discretion.

    Anaphylaxis was defined using the National Institute of Allergy and Infectious Diseases/Food Allergy and Anaphylaxis Network criteria.

    Meeting individual serum uric acid discontinuation criteria was defined as two consecutive pre-infusion serum uric acid levels greater than 6 mg/dL after Day 1.

    Time to event was derived as the date of the event minus the date of the first dose of the treatment plus 1. All participants were censored at the date of their last infusion.

  4. Time to First Event of IR Leading to Discontinuation of Treatment, Anaphylaxis or Meeting sUA Discontinuation Criteria in Participants With an Event

    Time frame: Day 1 to Week 24

    IRs were defined as any infusion-related adverse event or cluster of temporally-related adverse events, not attributable to another cause, which occurred during the infusion and for 2 hours post-infusion. Other adverse events that occurred outside of the 2-hour window following the infusion may have also been categorized as an infusion reaction at the Principal Investigator's discretion.

    Anaphylaxis was defined using the National Institute of Allergy and Infectious Diseases/Food Allergy and Anaphylaxis Network criteria.

    Meeting individual serum uric acid discontinuation criteria was defined as two consecutive pre-infusion serum uric acid levels greater than 6 mg/dL after Day 1.

    Time to event was derived as the date of the event minus the date of the first dose of the treatment plus 1. Only participants who experienced an event were included in this analysis.

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

A Phase 4, Multicenter, Open-Label, Infusion Duration Study To Assess Safety, Tolerability and Efficacy of Pegloticase Administered With a Shorter Infusion Duration in Subjects With Uncontrolled Gout Receiving Methotrexate

Important dates

Study start
2020
Primary completion
2024
Study completion
2024
First posted
Aug 13, 2020
Registry last updated
Oct 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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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.

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