NCT04511702

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
191

participants targeted

Target at P50-P75 for phase_4

Timeline
Completed

Started Oct 2020

Longer than P75 for phase_4

Geographic Reach
1 country

39 active sites

Status
completed

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

August 11, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 13, 2020

Completed
2 months until next milestone

Study Start

First participant enrolled

October 2, 2020

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 18, 2024

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 25, 2024

Completed
10 months until next milestone

Results Posted

Study results publicly available

January 7, 2025

Completed
Last Updated

October 20, 2025

Status Verified

October 1, 2025

Enrollment Period

3.3 years

First QC Date

August 11, 2020

Results QC Date

December 12, 2024

Last Update Submit

October 7, 2025

Conditions

Keywords

goutuncontrolled gout

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants Who Experienced an Infusion Reaction (IR), Including Anaphylaxis, Related to Pegloticase From 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.

    Day 1 to Week 24

Secondary Outcomes (4)

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

    Up to 6 months

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

    Day 1 to Week 24

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

    Day 1 to Week 24

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

    Day 1 to Week 24

Study Arms (3)

Pegloticase 60 Minute Infusion with methotrexate (MTX)

EXPERIMENTAL

Pegloticase 60 Minute Infusion with methotrexate (MTX). Participants will receive MTX (15 mg) (weekly) during the Run-in Period, then pegloticase (every 2 weeks) with MTX (weekly) for 24 weeks

Biological: Pegloticase with MTX

Pegloticase 45 Minute Infusion with methotrexate (MTX)

EXPERIMENTAL

Pegloticase 45 Minute Infusion with methotrexate (MTX). Participants will receive MTX (15 mg) (weekly) during the Run-in Period, then pegloticase (every 2 weeks) with MTX (weekly) for 24 weeks

Biological: Pegloticase with MTX

Pegloticase 30 Minute Infusion with methotrexate (MTX)

EXPERIMENTAL

Pegloticase 30 Minute Infusion with methotrexate (MTX). Participants will receive MTX (15 mg) (weekly) during the Run-in Period, then pegloticase (every 2 weeks) with MTX (weekly) for 24 weeks

Biological: Pegloticase with MTX

Interventions

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

Also known as: Methotrexate
Pegloticase 30 Minute Infusion with methotrexate (MTX)Pegloticase 45 Minute Infusion with methotrexate (MTX)Pegloticase 60 Minute Infusion with methotrexate (MTX)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • Severe chronic or recurrent bacterial infections, such as recurrent pneumonia or chronic bronchiectasis.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (39)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Orthopedic Physicians Alaska

Anchorage, Alaska, 99508, United States

Location

Arizona Arthritis & Rheumatology Research

Gilbert, Arizona, 85297, United States

Location

Arthritis & Rheumatology Research

Mesa, Arizona, 85210, United States

Location

Arizona Arthritis & Rheumatology Research

Phoenix, Arizona, 85037, United States

Location

Medvin Clinical Research

Covina, California, 91722, United States

Location

Amicis Research Center

Northridge, California, 91324, United States

Location

Providence Saint John's Health Center

Santa Monica, California, 90404, United States

Location

Medvin Clinical Research

Thousand Oaks, California, 91360, United States

Location

Medvin Clinical Research

Tujunga, California, 91042, United States

Location

University of Colorado Division of Rheumatology

Aurora, Colorado, 80045, United States

Location

Denver Arthritis Clinic

Denver, Colorado, 80230, United States

Location

Bradenton Research Center

Bradenton, Florida, 34205, United States

Location

Prohealth Research Center

Doral, Florida, 33166, United States

Location

LIFE Clinical Trials

Margate, Florida, 33063, United States

Location

D&H National Research Centers

Miami, Florida, 33155, United States

Location

IRIS Research and Development, LLC

Plantation, Florida, 33324, United States

Location

Napa Research Center

Pompano Beach, Florida, 33064, United States

Location

D&H Tamarac Research Centers

Tamarac, Florida, 33321, United States

Location

GCP Clinical Research

Tampa, Florida, 33064, United States

Location

ClinPro Research Solutions

Tampa, Florida, 33609, United States

Location

ForCare Clinical Research

Tampa, Florida, 33613, United States

Location

MedPharmics, LLC

Metairie, Louisiana, 70006, United States

Location

MD Medical Research

Oxon Hill, Maryland, 20745, United States

Location

Santa Fe Rheumatology

Santa Fe, New Mexico, 87505, United States

Location

Long Island Arthritis & Osteoporosis Care

Babylon, New York, 11702, United States

Location

Research Carolina Elite

Denver, North Carolina, 28037, United States

Location

PMG Research of Hickory, LLC

Hickory, North Carolina, 28602, United States

Location

Cape Fear Arthritis Care

Leland, North Carolina, 28451, United States

Location

PMG Research of Salisbury, LLC

Salisbury, North Carolina, 28144, United States

Location

Shelby Clinical Research, LLC

Shelby, North Carolina, 28150, United States

Location

Velocity Clinical Research Cincinnati

Cincinnati, Ohio, 45242, United States

Location

Altoona Center for Clinical Research

Duncansville, Pennsylvania, 16635, United States

Location

Chattanooga Research & Medicine PLLC (CHARM)

Chattanooga, Tennessee, 37404, United States

Location

Abigail Rebecca Neiman, MD, PA

Houston, Texas, 77024, United States

Location

Pioneer Research Solutions Inc - Houston

Houston, Texas, 77099, United States

Location

Velocity Clinical Research, Salt Lake City

West Jordan, Utah, 84088, United States

Location

Western Washington Medical Group

Bothell, Washington, 98021, United States

Location

Arthritis Northwest

Spokane, Washington, 99204, United States

Location

Related Links

MeSH Terms

Conditions

Gout

Interventions

PegloticaseMethotrexate

Condition Hierarchy (Ancestors)

ArthritisJoint DiseasesMusculoskeletal DiseasesCrystal ArthropathiesRheumatic DiseasesPurine-Pyrimidine Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

AminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds

Results Point of Contact

Title
Study Director
Organization
Amgen Inc.

Study Officials

  • MD

    Amgen

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Three different infusion durations may be used. 60-minute infusion, 45-minute infusion and 30-minute.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 11, 2020

First Posted

August 13, 2020

Study Start

October 2, 2020

Primary Completion

January 18, 2024

Study Completion

March 25, 2024

Last Updated

October 20, 2025

Results First Posted

January 7, 2025

Record last verified: 2025-10

Locations