CSP594 Comparative Effectiveness in Gout: Allopurinol vs. Febuxostat

NCT ID: NCT02579096

Last Updated: 2024-03-29

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

950 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-06

Study Completion Date

2021-04-15

Brief Summary

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This trial will compare two effective therapies, allopurinol and febuxostat, to lower serum uric acid and therefore prevent further gout attacks. These therapies have never been compared at appropriate doses. Further, they will be studied in patients with kidney disease for the first time.

Detailed Description

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Gout is the most common form of inflammatory arthritis affecting adults (1), with a disease frequency that continues to increase dramatically (2). Gout is associated with substantial morbidity and mortality which are concentrated in older men and magnified in patients with chronic kidney disease (CKD) (3-6), demographics common to the Veterans Affairs (VA) Health System. Effective gout therapies are readily available and are centered primarily on the use of approved urate lowering therapy (ULT). Despite having excellent ULT options available to patients (7), gout is extremely poorly managed especially in patients with CKD (8-10).

The two most widely used ULTs in clinical practice, allopurinol and febuxostat, have recently been endorsed as the two acceptable first-line treatment strategies in chronic gout (7). Although both agents appear to be efficacious and generally well-tolerated, allopurinol and febuxostat have significantly different costs and have never been compared to each other at appropriate doses. Randomized controlled trials completed to date comparing allopurinol with febuxostat in gout have used 'fixed' and, in many cases, insufficient doses of allopurinol (11-13), an approach that is contrary to current guideline recommendations (7). Furthermore, these studies have included only very small proportions of gout patients with CKD even though CKD is present in approximately 1 of every 2 gout sufferers (14).

To test the hypothesis that allopurinol is non-inferior to febuxostat in the treatment of gout, the investigators propose a randomized open-label non-inferiority trial, which for the first time compares allopurinol with febuxostat using appropriately titrated doses and a "treat-to-target" approach. Further, the investigators will assess the comparative effectiveness of these agents in a significant number of gout patients with co-morbid CKD.

The investigators plan to enroll 950 participants with a diagnosis of gout, including participants with stage 3 CKD, who are hyperuricemic defined as a serum uric acid concentration (sUA) above 6.8 mg/dl. Participants will be recruited from 18 Veteran Affairs and 5 Rheumatology and Arthritis Investigational Network (RAIN) sites. The total duration of the trial will be 4 years. Recruitment will occur over 24 months. Participants will be followed for 72 weeks. This will include a 24 week Dose Titration Phase (Phase 1) followed by a 24 week Maintenance and Optimization Phase (Phase 2) and then a 24 week Steady State Flare Observation Phase (Phase 3). The investigators will use a "treat-to-target" approach with specified titration of ULT dosing to obtain goal sUA. Maximal daily drug doses will be 800 mg/day for allopurinol or 120 mg/day for febuxostat.

The primary outcome will be the proportion of participants who have at least one gout flare in the allopurinol group compared to the febuxostat group during Phase 3. This primary outcome was endorsed by the patient and VA provider groups that were surveyed (see below). All participants will be followed during Phase 3 regardless of the achievement of sUA goal. The primary hypothesis will test the non-inferiority of allopurinol with regards to proportions of flares. The investigators anticipate that approximately 15 to 20% of patients will flare during Phase 3.

Conditions

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Gout Chronic Kidney Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Allopurinol / Sham Comparator (Febuxostat)

Patients will be titrated up to the dose that will lower to target uric acid levels. A placebo resembling Febuxostat will be given with allopurinol

Group Type ACTIVE_COMPARATOR

allopurinol capsule, 100-800 mg by mouth once daily

Intervention Type DRUG

Patients will be up-titrated up to the dose required to reach target uric acid levels.

Placebo, vehicle control (febuxostat-shaped)

Intervention Type DRUG

Placebo tablets resembling febuxostat will be given with allopurinol.

Febuxostat / Sham Comparator (Allopurinol)

Febuxostat will be titrated up to the dose that will lower to target uric acid levels. A placebo resembling Allopurinol will be given with Febuxostat

Group Type ACTIVE_COMPARATOR

febuxostat tablet 40-120 mg by mouth once daily

Intervention Type DRUG

Patients will be up-titrated to the dose required to reach target uric acid levels.

Placebo, vehicle control (allopurinol-shaped)

Intervention Type DRUG

Placebo capsules resembling allopurinol will be given with febuxostat.

Interventions

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allopurinol capsule, 100-800 mg by mouth once daily

Patients will be up-titrated up to the dose required to reach target uric acid levels.

Intervention Type DRUG

febuxostat tablet 40-120 mg by mouth once daily

Patients will be up-titrated to the dose required to reach target uric acid levels.

Intervention Type DRUG

Placebo, vehicle control (febuxostat-shaped)

Placebo tablets resembling febuxostat will be given with allopurinol.

Intervention Type DRUG

Placebo, vehicle control (allopurinol-shaped)

Placebo capsules resembling allopurinol will be given with febuxostat.

Intervention Type DRUG

Other Intervention Names

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Zyloprim; CAS: 315-30-0 Uloric; CAS: 144060-53-7; NDCs: 64764-677-11, 64764-677-13, 64764-677-19, 64764-677-30, 64764-918-11, 64764-918-18, 64764-918-30, 64764-918-90

Eligibility Criteria

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Inclusion Criteria

* Age 18 years
* History of gout - crystal proven or historical as defined by ACR criteria listed above
* Serum urate level \> 6.8 mg/dl

Exclusion Criteria

* Stage 4 or 5 Chronic Kidney Disease (CKD) - defined as eGFR \< 30 ml/min/1.73 m2
* Women less than 50 years of age
* Patients with a history of prior solid organ / hematopoietic transplantation
* Previous allergy or intolerance to allopurinol or febuxostat
* Patients who are not candidates for any of the recommended prophylactic medications (colchicine, naprosyn or glucocorticoids)
* Patients who in the opinion of the investigator have a high genetic risk for allopurinol hypersensitivity syndrome (AHS\*) unless they have been found to be negative for HLA B5801.
* Previous history of failure to reach target uric acid levels despite therapy with allopurinol at dose \> 300 mg/day
* Prior febuxostat use
* Patients with malignancies that are currently active with exception of non-melanoma skin cancer
* Patients with serum uric acid levels \>15 mg/dl
* Patients with myelodysplasia and hemoglobin of \< 8.5 mg/dL
* Patients with chronic liver disease with more than one of the following:

* INR \> 1.7, not on Warfarin therapy
* Bilirubin 2 mg/dL
* Serum albumin \< 3.5 mg/dL
* Ascites
* Encephalopathy
* Current use of azathioprine, mercaptopurine, didanosine, cyclophosphamide, probenecid, lesinurad or pegloticase
* Enrollment in another randomized interventional clinical trial
* Any severe medical condition that, in the enrolleer's opinion, is likely to compromise the participant's ability to complete the trial
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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VA Office of Research and Development

FED

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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James R O'Dell

Role: STUDY_CHAIR

Omaha VA Nebraska-Western Iowa Health Care System, Omaha, NE

Locations

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VA Loma Linda Healthcare System, Loma Linda, CA

Loma Linda, California, United States

Site Status

VA San Diego Healthcare System, San Diego, CA

San Diego, California, United States

Site Status

San Francisco VA Medical Center, San Francisco, CA

San Francisco, California, United States

Site Status

Miami VA Healthcare System, Miami, FL

Miami, Florida, United States

Site Status

Edward Hines Jr. VA Hospital, Hines, IL

Hines, Illinois, United States

Site Status

VA Boston Healthcare System Jamaica Plain Campus, Jamaica Plain, MA

Boston, Massachusetts, United States

Site Status

Minneapolis VA Health Care System, Minneapolis, MN

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic Rochester MN ? RAIN 1

Rochester, Minnesota, United States

Site Status

Kansas City VA Medical Center, Kansas City, MO

Kansas City, Missouri, United States

Site Status

Omaha VA Nebraska-Western Iowa Health Care System, Omaha, NE

Omaha, Nebraska, United States

Site Status

University of Nebraska Medical Center ? RAIN 5

Omaha, Nebraska, United States

Site Status

Manhattan Campus of the VA NY Harbor Healthcare System, New York, NY

New York, New York, United States

Site Status

Asheville VA Medical Center, Asheville, NC

Asheville, North Carolina, United States

Site Status

Sanford Bismarck Medical Center ? RAIN 2

Bismarck, North Dakota, United States

Site Status

Cincinnati VA Medical Center, Cincinnati, OH

Cincinnati, Ohio, United States

Site Status

VA Portland Health Care System, Portland, OR

Portland, Oregon, United States

Site Status

Corporal Michael J. Crescenz VA Medical Center, Philadelphia, PA

Philadelphia, Pennsylvania, United States

Site Status

VA Pittsburgh Healthcare System University Drive Division, Pittsburgh, PA

Pittsburgh, Pennsylvania, United States

Site Status

Yankton Medical Clinic ? RAIN 3

Yankton, South Dakota, United States

Site Status

VA North Texas Health Care System Dallas VA Medical Center, Dallas, TX

Dallas, Texas, United States

Site Status

VA Salt Lake City Health Care System, Salt Lake City, UT

Salt Lake City, Utah, United States

Site Status

White River Junction VA Medical Center, White River Junction, VT

White River Junction, Vermont, United States

Site Status

Salem VA Medical Center, Salem, VA

Salem, Virginia, United States

Site Status

Countries

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United States

References

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Timilsina S, Brittan K, O'Dell JR, Brophy M, Davis-Karim A, Henrie AM, Neogi T, Newcomb J, Palevsky PM, Pillinger MH, Pittman D, Taylor TH, Wu H, Mikuls TR. Design and Rationale for the Veterans Affairs "Cooperative Study Program 594 Comparative Effectiveness in Gout: Allopurinol vs. Febuxostat" Trial. Contemp Clin Trials. 2018 May;68:102-108. doi: 10.1016/j.cct.2018.03.015. Epub 2018 Mar 27.

Reference Type BACKGROUND
PMID: 29597007 (View on PubMed)

O'Dell JR, Brophy MT, Pillinger MH, Neogi T, Palevsky PM, Wu H, Davis-Karim A, Newcomb JA, Ferguson R, Pittman D, Cannon GW, Taylor T, Terkeltaub R, Cannella AC, England BR, Helget LN, Mikuls TR. Comparative Effectiveness of Allopurinol and Febuxostat in Gout Management. NEJM Evid. 2022 Mar;1(3):10.1056/evidoa2100028. doi: 10.1056/evidoa2100028. Epub 2022 Feb 3.

Reference Type RESULT
PMID: 35434725 (View on PubMed)

Sanchez C, Campeau A, Liu-Bryan R, Mikuls TR, O'Dell JR, Gonzalez DJ, Terkeltaub R. Effective xanthine oxidase inhibitor urate lowering therapy in gout is linked to an emergent serum protein interactome of complement and inflammation modulators. Sci Rep. 2024 Oct 19;14(1):24598. doi: 10.1038/s41598-024-74154-5.

Reference Type DERIVED
PMID: 39426967 (View on PubMed)

Sanchez C, Campeau A, Liu-Bryan R, Mikuls TR, O'Dell JR, Gonzalez DJ, Terkeltaub R. Effective xanthine oxidase inhibitor urate lowering therapy in gout is linked to an emergent serum protein interactome of complement activation and inflammation modulators. Res Sq [Preprint]. 2024 May 9:rs.3.rs-4278877. doi: 10.21203/rs.3.rs-4278877/v1.

Reference Type DERIVED
PMID: 38766125 (View on PubMed)

Sanchez C, Campeau A, Liu-Bryan R, Mikuls T, O'Dell J, Gonzalez D, Terkeltaub R. Sustained xanthine oxidase inhibitor treat to target urate lowering therapy rewires a tight inflammation serum protein interactome. Res Sq [Preprint]. 2024 Jan 2:rs.3.rs-3770277. doi: 10.21203/rs.3.rs-3770277/v1.

Reference Type DERIVED
PMID: 38260556 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

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594

Identifier Type: -

Identifier Source: org_study_id

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