Clinical trial NCT01466166: Observational Study of the Use of Pegloticase (KRYSTEXXA®) in Refractory Chronic Gout
Official title: Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Refractory Chronic Gout
Lead sponsor: Horizon Pharma Rheumatology LLC
Sponsor class: INDUSTRY
First posted: 2011-11-06
Enrollment: 188
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The patient population in this study will be hyperuricemic (serum uric acid (SUA) \> 6 mg/dL) adult men and women (age 18 or greater) diagnosed with chronic gout and who are refractory to conventional therapy. Gout refractory to conventional therapy occurs in 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. To enter this study, the patient and the physician must have decided to begin treatment with KRYSTEXXA.

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Infusion Reactions
timeFrame: 52 weeks
measure: Number of Participants With Anaphylaxis
timeFrame: 52 weeks
measure: Number of Participants With Immune Complex-related Events
timeFrame: From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
Secondary outcomes:
measure: Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52
timeFrame: Week 24 and week 52
measure: Change From Baseline in Number of Gout Flares
timeFrame: Baseline, week 24 and week 48
measure: Number of Swollen Joints Over Time
timeFrame: Baseline and weeks 24 and 52
measure: Number of Tender Joints Over Time
timeFrame: Baseline and weeks 24 and 52
measure: Number of Palpable Tophi Over Time
timeFrame: Baseline and weeks 24 and 52
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Rheumatology Associates, PC
state: Alabama
city: Birmingham
country: United States
facility: UAB Rheumatology
state: Alabama
city: Huntsville
country: United States
facility: Saadat Ansari, MD, LLC
state: Alabama
city: Covina
country: United States
facility: Medvin Clinical Research
state: California
city: Laguna Hills
country: United States
facility: Alliance Clinical Research, LLC
state: California
city: Lakewood
country: United States
facility: Advanced Medical Research, LLC
state: California
city: Los Angeles
country: United States
facility: Pacific Arthritis Care Center
state: California
city: Monterey Park
country: United States
facility: R Srinivasan, MD, Inc
state: California
city: Murrieta
country: United States
facility: Brigid Freyne, MD, Inc
state: California
city: Poway
country: United States
facility: Alliance Clinical Research
state: California
city: Denver
country: United States
facility: Denver Nephrologists, PC
state: Colorado
city: Trumbull
country: United States
facility: New England Research Associates, LLC
state: Connecticut
city: Washington
country: United States
facility: Howard University Hospital
state: District of Columbia
city: Washington
country: United States
facility: Washington DC Veteran's Affairs Medical Center
state: District of Columbia
city: Brandon
country: United States
facility: Bay Area Arthritis and Osteoporosis
state: Florida
city: Clearwater
country: United States
facility: Countryside Arthritis Center
state: Florida
city: Jupiter
country: United States
facility: Science and Research Institute, Inc
state: Florida
city: Miami
country: United States
facility: A & O Research Center
state: Florida
city: Palm Harbor
country: United States
facility: Arthritis Research of Florida, Inc.
state: Florida
city: Pembroke Pines
country: United States
facility: Family Clinical Trials, LLC
state: Florida
city: Tampa
country: United States
facility: Jedidiah Clinical Research
state: Florida
city: Tampa
country: United States
facility: Midtown Medical Center
state: Florida
city: Calhoun
country: United States
facility: Global Research Partners & Consultants, Inc.
state: Georgia
city: Stockbridge
country: United States
facility: Arthritis Research & Treatment Center
state: Georgia
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Diagnostic Rheumatology and Research PC
state: Indiana
city: Cedar Rapids
country: United States
facility: Physicians' Clinic of Iowa, P.C.
state: Iowa
city: Mount Sterling
country: United States
facility: Central Kentucky Research Associates of Kentucky
state: Kentucky
city: Owensboro
country: United States
facility: Research Integrity, LLC
state: Kentucky
city: Eunice
country: United States
facility: Horizon Research Group of Opelousas, LLC
state: Louisiana
city: Monroe
country: United States
facility: Arthritis and Diabetes Clinic, Inc.
state: Louisiana
city: Bethesda
country: United States
facility: Walter Reed National Military Medical Center
state: Maryland
city: Hagerstown
country: United States
facility: Klein & Associates MD, PA.
state: Maryland
city: Wheaton
country: United States
facility: The Center for Rheumatology and Bone Research
state: Maryland
city: Worcester
country: United States
facility: Clinical Pharmacology Study Groups
state: Massachusetts
city: Worcester
country: United States
facility: Reliant Medical Group, Inc.
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Caro
country: United States
facility: Caro Health Plaza
state: Michigan
city: Grand Rapids
country: United States
facility: Infusion Associates
state: Michigan
city: Lansing
country: United States
facility: Justus J. Fiechtner, MD, PC
state: Michigan
city: Saint Clair Shores
country: United States
facility: Shores Rheumatology, PC
state: Michigan
city: Eagan
country: United States
facility: Saint Paul Rheumatology, PA
state: Minnesota
city: Kansas City
country: United States
facility: Kansas City Internal Medicine
state: Missouri
city: Las Vegas
country: United States
facility: Arthritis Medical Clinic
state: Nevada
city: Teaneck
country: United States
facility: Rheumatology Associates of North Jersey
state: New Jersey
city: Smithtown
country: United States
facility: Rheumatology Associates of Long Island
state: New York
city: Concord
country: United States
facility: NorthEast Rheumatology
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: Physicians East, PA
state: North Carolina
city: Raleigh
country: United States
facility: Shanahan Rheumatology and Immunotherapy, PLLC
state: North Carolina
city: Sanford
country: United States
facility: Specialty Medical Clinic and Research Center
state: North Carolina
city: Portsmouth
country: United States
facility: Southern Ohio Rheumatology
state: Ohio
city: Altoona
country: United States
facility: Keystone Pain Institute, Ilumina Clinical Associates
state: Pennsylvania
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Low Country Rheumatology
state: South Carolina
city: Orangeburg
country: United States
facility: Acme Research L.L.C.
state: South Carolina
city: Memphis
country: United States
facility: Ramesh C. Gupta, M.D.
state: Tennessee
city: Austin
country: United States
facility: Austin Regional Clinic
state: Texas
city: El Paso
country: United States
facility: Dr. Raj Marwah
state: Texas
city: Houston
country: United States
facility: Diagnostic Clinic of Houston
state: Texas
city: Houston
country: United States
facility: Rheumatic Disease Clinical Research Center
state: Texas
city: Arlington
country: United States
facility: Arthritis Clinic of Northern Virginia, P.C.
state: Virginia
city: Manassas
country: United States
facility: Arthritis & Osteoporosis Center of North Virginia
state: Virginia
city: Norfolk
country: United States
facility: Sentara Rheumatology Specialists
state: Virginia
city: Kennewick
country: United States
facility: Apex Clinical Research
state: Washington
city: Clarksburg
country: United States
facility: Mountain State Clinical Research
state: West Virginia
city: Glendale
country: United States
facility: Rheumatic Disease Center, LLP
state: Wisconsin
Study officials:
affiliation: Horizon Pharma Rheumatology LLC
name: Jeffery Nieves, PharmD
role: STUDY_DIRECTOR
References:
citation: Sundy JS, Baraf HS, Yood RA, Edwards NL, Gutierrez-Urena SR, Treadwell EL, Vazquez-Mellado J, White WB, Lipsky PE, Horowitz Z, Huang W, Maroli AN, Waltrip RW 2nd, Hamburger SA, Becker MA. Efficacy and tolerability of pegloticase for the treatment of chronic gout in patients refractory to conventional treatment: two randomized controlled trials. JAMA. 2011 Aug 17;306(7):711-20. doi: 10.1001/jama.2011.1169.