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A Trial to Investigate the Non-inferiority of Pegloticase Administered Every 4 Weeks (Q4W) With MTX Compared With Every 2 Weeks (Q2W) With MTX in Participants With Uncontrolled Refractory Gout

Phase 4
Active, not recruiting
Conditions
Gout
Interventions
Registration Number
NCT06229145
Lead Sponsor
Amgen
Brief Summary

The study consists of 24-week double-blind trial to evaluate the non-inferiority of the efficacy and safety of pegloticase Q4W with MTX versus pegloticase Q2W with MTX, followed by a 24-week open-label extension of pegloticase Q4W with MTX, in participants with uncontrolled refractory gout.

The main objective of the study is to evaluate the effect of pegloticase 16 mg administered Q4W with MTX versus pegloticase 8 mg administered Q2W with MTX, on the response rate during Month 6, as measured by the sustained normalization of sUA to \< 6 mg/dL for at least 80% of the time.

Detailed Description

Acquired from Horizon in 2024.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
262
Inclusion Criteria
  1. Adult men or women ≥ 18 years of age

  2. Uncontrolled gout, defined as meeting the following criteria:

    • Hyperuricemia during the Screening Period, defined as sUA ≥ 7 mg/dL, and;
    • Failure to maintain normalization of sUA with xanthine oxidase inhibitors at the maximum medically appropriate dose, or with a contraindication to xanthine oxidase inhibitor therapy based on medical record review or participant interview, and;
    • Symptoms of gout
  3. Willing to discontinue any oral ULT for at least 7 days prior to MTX dosing at Week -4 and continue not receiving any oral ULT when receiving pegloticase/placebo for pegloticase infusions

  4. Women of childbearing potential must have negative serum/urine pregnancy tests during screening and Week -4.

  5. Men who are not vasectomized must agree to use appropriate contraception, so as to not impregnate a female partner of reproductive potential during the trial.

Exclusion Criteria
  1. Severe chronic or recurrent bacterial infections, such as recurrent pneumonia or chronic bronchiectasis
  2. 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 (3 months or longer)
  3. History of any transplant surgery requiring maintenance immunosuppressive therapy
  4. Known history of hepatitis B virus surface antigen positivity or hepatitis B DNA positivity
  5. Known history of hepatitis C virus RNA positivity, unless treated and viral load is negative
  6. Known history of human immunodeficiency virus (HIV) positivity
  7. G6PD deficiency (quantitative test at the Screening Visit centrally or locally)
  8. Non-compensated congestive heart failure or hospitalization for congestive heart failure within 3 months of the Screening Visit, uncontrolled arrhythmia, treatment for acute coronary syndrome (myocardial infarction or unstable angina) or uncontrolled blood pressure (> 160/100 mmHg) prior to Week -4
  9. 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
  10. Prior treatment with pegloticase, another recombinant uricase (rasburicase) or concomitant therapy with a PEG-conjugated drug
  11. Unable to tolerate MTX 15 mg orally during the MTX Run-in Period
  12. Chronic liver disease
  13. White blood cell count < 4,000/μL, hematocrit < 32% or platelet count < 75,000/μL
  14. Currently receiving systemic or radiologic treatment for ongoing cancer
  15. History of malignancy within 5 years other than non-melanoma skin cancer or in situ carcinoma of cervix
  16. Diagnosis of osteomyelitis
  17. Known history of hypoxanthine-guanine phosphoribosyl-transferase deficiency, such as Lesch-Nyhan and Kelley-Seegmiller syndrome
  18. A known intolerance to all protocol-standard gout flare prophylaxis regimens (i.e., participant must be able to tolerate at least 1 of the following: colchicine and/or non-steroidal anti-inflammatory drugs and/or low-dose prednisone ≤ 10 mg/day)
  19. Current pulmonary fibrosis, bronchiectasis or interstitial pneumonitis. If deemed necessary by the Investigator, a chest X-ray may be performed during Screening.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Pegloticase + Methotrexate Q2WPegloticase8 mg pegloticase will be administered intravenously every 2 weeks for 24 weeks during double blind treatment and 16 mg pegloticase every 4 weeks for 24 weeks during open label.
Pegloticase + Methotrexate Q2WMethotrexate8 mg pegloticase will be administered intravenously every 2 weeks for 24 weeks during double blind treatment and 16 mg pegloticase every 4 weeks for 24 weeks during open label.
Pegloticase + Methotrexate Q4WPegloticase16 mg pegloticase will be administered intravenously every 4 weeks for 24 weeks during double blind treatment and 24 weeks during open label.
Pegloticase + Methotrexate Q4WMethotrexate16 mg pegloticase will be administered intravenously every 4 weeks for 24 weeks during double blind treatment and 24 weeks during open label.
Primary Outcome Measures
NameTimeMethod
Proportion of Participants achieving and maintaining serum uric acid (sUA) < 6 mg/dL at least 80% of the timeMonth 6
Secondary Outcome Measures
NameTimeMethod
Proportion of Participants with complete resolution of ≥ 1 tophusWeek 24

Trial Locations

Locations (69)

The University of Alabama at Birmingham (UAB)

🇺🇸

Birmingham, Alabama, United States

Orthopedic Physicians Alaska - Rheumatology and Infusion

🇺🇸

Anchorage, Alaska, United States

Arizona Arthritis and Rheumatology

🇺🇸

Tucson, Arizona, United States

Arizona Arthritis and Rheumatology Rese

🇺🇸

Flagstaff, Arizona, United States

Arizona Arthritis & Rheumatology

🇺🇸

Sun City, Arizona, United States

Arizona ArthritisRheumatology Resea

🇺🇸

Mesa, Arizona, United States

Arizona Arthritis and Rheumatology Research

🇺🇸

Phoenix, Arizona, United States

Covina Arthritis Clinic

🇺🇸

Covina, California, United States

Medvin Clinical Research- Riverside

🇺🇸

Hemet, California, United States

TriWest Research Associates

🇺🇸

San Diego, California, United States

PCCRS

🇺🇸

San Leandro, California, United States

Saint John's Health Center

🇺🇸

Santa Monica, California, United States

C.V. Mehta MD Medical Corporation

🇺🇸

Temecula, California, United States

Foothill Arthritis

🇺🇸

Tujunga, California, United States

Medvin Clinical Research

🇺🇸

Whittier, California, United States

University of Colorado Denver

🇺🇸

Aurora, Colorado, United States

Lynn Institute of the Rockies

🇺🇸

Colorado Springs, Colorado, United States

Denver Arthritis Clinic

🇺🇸

Denver, Colorado, United States

Arthritis & Rheumatic Disease

🇺🇸

Aventura, Florida, United States

Prohealth Research Center

🇺🇸

Doral, Florida, United States

Homestead Associates in Research,Inc

🇺🇸

Homestead, Florida, United States

Life Clinical Trials

🇺🇸

Margate, Florida, United States

D&H National Research Centers, Inc.

🇺🇸

Miami, Florida, United States

Well Pharma

🇺🇸

Miami, Florida, United States

Felicidad Medical Research, LLC.

🇺🇸

Miami, Florida, United States

New Generation Of Medical Research

🇺🇸

Naples, Florida, United States

IRIS Research & Development

🇺🇸

Plantation, Florida, United States

D&H Pompano Research Center

🇺🇸

Pompano Beach, Florida, United States

D&H Tamarac Research Center

🇺🇸

Tamarac, Florida, United States

ClinPro Research Solutions, LLC

🇺🇸

Tampa, Florida, United States

GCP Clinical Research

🇺🇸

Tampa, Florida, United States

Conquest Research

🇺🇸

Winter Park, Florida, United States

Arthritis Center of North Georgia

🇺🇸

Gainesville, Georgia, United States

Vista Clinical Research, LLC

🇺🇸

Newnan, Georgia, United States

Velocity Clinical Research - Boise

🇺🇸

Meridian, Idaho, United States

Great Lakes Clin. Trials

🇺🇸

Chicago, Illinois, United States

Great Lakes Clinical Trials - Gurnee

🇺🇸

Gurnee, Illinois, United States

Crowley CORE

🇺🇸

Hinsdale, Illinois, United States

Lake Cumberland Rheumatology, PLLC

🇺🇸

New Albany, Indiana, United States

L-MARC Research Center

🇺🇸

Louisville, Kentucky, United States

Velocity CR - New Orleans

🇺🇸

New Orleans, Louisiana, United States

Michigan Medicine

🇺🇸

Ann Arbor, Michigan, United States

June DO, PC

🇺🇸

Lansing, Michigan, United States

Clinical Research Institute of Michigan, LLC

🇺🇸

Saint Clair Shores, Michigan, United States

Inspire Santa Fe Medical Group

🇺🇸

Santa Fe, New Mexico, United States

Velocity Clinical Research-Vestal

🇺🇸

Vestal, New York, United States

OnSite Clinical Solutions, LLC

🇺🇸

Charlotte, North Carolina, United States

Research Carolina Elite, LLC

🇺🇸

Denver, North Carolina, United States

Triad Clinical Trials

🇺🇸

Greensboro, North Carolina, United States

Accellacare of Hickory

🇺🇸

Hickory, North Carolina, United States

Cape Fear Arthritis Care

🇺🇸

Leland, North Carolina, United States

Shelby Clinical Research

🇺🇸

Shelby, North Carolina, United States

Velocity Clinical Res, Cleveland

🇺🇸

Beachwood, Ohio, United States

Velocity Clinical Research - Cincinnati

🇺🇸

Cincinnati, Ohio, United States

Lynn Health Science Institute

🇺🇸

Oklahoma City, Oklahoma, United States

Altoona Center for Clinical Research - Research

🇺🇸

Duncansville, Pennsylvania, United States

Velocity Clinical Research, Greenville

🇺🇸

Greenville, South Carolina, United States

Premier Clinics Pa

🇺🇸

Rock Hill, South Carolina, United States

Low Country Rheumatology

🇺🇸

Summerville, South Carolina, United States

Amarillo Center for Clinical Research, Ltd.

🇺🇸

Amarillo, Texas, United States

Synergy Groups Medical LLC

🇺🇸

Houston, Texas, United States

Clinical Trial Network

🇺🇸

Houston, Texas, United States

Pioneer Research Solutions, Inc.

🇺🇸

Houston, Texas, United States

AIM Trials - Internal Medicine

🇺🇸

Plano, Texas, United States

Clinical Trials of Texas, Inc.

🇺🇸

San Antonio, Texas, United States

Siena Research Network

🇺🇸

Sugar Land, Texas, United States

Velocity Clinical Research - Salt Lake City

🇺🇸

West Jordan, Utah, United States

Velocity Clin Research-Portsmouth

🇺🇸

Suffolk, Virginia, United States

Arthritis Northwest, PLLC - Research

🇺🇸

Spokane, Washington, United States

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