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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01466166
Other study ID # M0401
Secondary ID
Status Completed
Phase
First received
Last updated
Start date November 15, 2011
Est. completion date June 30, 2017

Study information

Verified date June 2018
Source Horizon Pharma Rheumatology LLC
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 188
Est. completion date June 30, 2017
Est. primary completion date June 30, 2017
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
Pegloticase
Pegloticase 8 mg intravenous every 2 weeks

Locations

Country Name City State
United States Keystone Pain Institute, Ilumina Clinical Associates Altoona Pennsylvania
United States University of Michigan Health System Ann Arbor Michigan
United States Arthritis Clinic of Northern Virginia, P.C. Arlington Virginia
United States Austin Regional Clinic Austin Texas
United States Walter Reed National Military Medical Center Bethesda Maryland
United States Rheumatology Associates, PC Birmingham Alabama
United States UAB Rheumatology Birmingham Alabama
United States Bay Area Arthritis and Osteoporosis Brandon Florida
United States Global Research Partners & Consultants, Inc. Calhoun Georgia
United States Caro Health Plaza Caro Michigan
United States Physicians' Clinic of Iowa, P.C. Cedar Rapids Iowa
United States Low Country Rheumatology Charleston South Carolina
United States University of Chicago Medical Center Chicago Illinois
United States Mountain State Clinical Research Clarksburg West Virginia
United States Countryside Arthritis Center Clearwater Florida
United States NorthEast Rheumatology Concord North Carolina
United States Medvin Clinical Research Covina California
United States Denver Nephrologists, PC Denver Colorado
United States Duke University Medical Center Durham North Carolina
United States Saint Paul Rheumatology, PA Eagan Minnesota
United States Dr. Raj Marwah El Paso Texas
United States Horizon Research Group of Opelousas, LLC Eunice Louisiana
United States Rheumatic Disease Center, LLP Glendale Wisconsin
United States Infusion Associates Grand Rapids Michigan
United States Physicians East, PA Greenville North Carolina
United States Klein & Associates MD, PA. Hagerstown Maryland
United States Penn State Milton S. Hershey Medical Center Hershey Pennsylvania
United States Diagnostic Clinic of Houston Houston Texas
United States Rheumatic Disease Clinical Research Center Houston Texas
United States Saadat Ansari, MD, LLC Huntsville Alabama
United States Diagnostic Rheumatology and Research PC Indianapolis Indiana
United States Science and Research Institute, Inc Jupiter Florida
United States Kansas City Internal Medicine Kansas City Missouri
United States Apex Clinical Research Kennewick Washington
United States Alliance Clinical Research, LLC Laguna Hills California
United States Advanced Medical Research, LLC Lakewood California
United States Justus J. Fiechtner, MD, PC Lansing Michigan
United States Arthritis Medical Clinic Las Vegas Nevada
United States Pacific Arthritis Care Center Los Angeles California
United States Arthritis & Osteoporosis Center of North Virginia Manassas Virginia
United States Ramesh C. Gupta, M.D. Memphis Tennessee
United States A & O Research Center Miami Florida
United States Arthritis and Diabetes Clinic, Inc. Monroe Louisiana
United States R Srinivasan, MD, Inc Monterey Park California
United States Central Kentucky Research Associates of Kentucky Mount Sterling Kentucky
United States Brigid Freyne, MD, Inc Murrieta California
United States Sentara Rheumatology Specialists Norfolk Virginia
United States Acme Research L.L.C. Orangeburg South Carolina
United States Research Integrity, LLC Owensboro Kentucky
United States Arthritis Research of Florida, Inc. Palm Harbor Florida
United States Family Clinical Trials, LLC Pembroke Pines Florida
United States Southern Ohio Rheumatology Portsmouth Ohio
United States Alliance Clinical Research Poway California
United States Shanahan Rheumatology and Immunotherapy, PLLC Raleigh North Carolina
United States Shores Rheumatology, PC Saint Clair Shores Michigan
United States Specialty Medical Clinic and Research Center Sanford North Carolina
United States Rheumatology Associates of Long Island Smithtown New York
United States Arthritis Research & Treatment Center Stockbridge Georgia
United States Jedidiah Clinical Research Tampa Florida
United States Midtown Medical Center Tampa Florida
United States Rheumatology Associates of North Jersey Teaneck New Jersey
United States New England Research Associates, LLC Trumbull Connecticut
United States Howard University Hospital Washington District of Columbia
United States Washington DC Veteran's Affairs Medical Center Washington District of Columbia
United States The Center for Rheumatology and Bone Research Wheaton Maryland
United States Clinical Pharmacology Study Groups Worcester Massachusetts
United States Reliant Medical Group, Inc. Worcester Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Horizon Pharma Rheumatology LLC

Country where clinical trial is conducted

United States, 

References & Publications (1)

Sundy JS, Baraf HS, Yood RA, Edwards NL, Gutierrez-Urena SR, Treadwell EL, Vázquez-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. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants With Infusion Reactions Infusion reactions were defined as adverse events (AEs) or clusters of events, not attributable to another cause that occurred during or within 2 hours after the infusion of pegloticase. Any other case that occurred outside of the 2-hour window was categorized per Investigator discretion. 52 weeks
Primary Number of Participants With Anaphylaxis Anaphylaxis was defined using the National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network (NIAID/FAAN) criteria: Acute onset of an illness (minutes to several hours) with involvement of the skin, mucosal tissue, or both (e.g., generalized hives; pruritus or flushing; swollen lips, tongue, or uvula), and at least 1 of the following:
Respiratory compromise (e.g., dyspnea, wheeze-bronchospasm, stridor, reduced peak expiratory flow, hypoxemia).
Reduced blood pressure (i.e., systolic blood pressure < 90 mm Hg or greater than 30% decrease from that patient's baseline) or associated symptoms of end-organ failure (e.g., hypotonia [collapse], syncope, incontinence).
52 weeks
Primary Number of Participants With Immune Complex-related Events Immune complex-related events were defined as any presumptive immune complex-related disorders that were confirmed by an appropriate investigation of the event and of complement markers (C3 and C4 levels). Clinical manifestations could have included skin rash, arthralgia, arthritis, proteinuria, serum sickness, and cryoglobulinemia. From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
Secondary Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52 Normalization of serum uric acid was defined as serum uric acid value less than 6 mg/dL. Week 24 and week 52
Secondary Change From Baseline in Number of Gout Flares The number of gout flares occurring in the 2 weeks prior to each visit. Baseline number of flares was calculated as the average number of flares that occurred in the 6-month baseline period divided by 12 weeks. Baseline, week 24 and week 48
Secondary Number of Swollen Joints Over Time Baseline and weeks 24 and 52
Secondary Number of Tender Joints Over Time Baseline and weeks 24 and 52
Secondary Number of Palpable Tophi Over Time Gout tophi are nodular deposits of urate crystals and inflammatory cells in joints, soft tissues, bones, and in some organs. Baseline and weeks 24 and 52

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