Refractory Chronic GoutEyesOnGOUTNCT01466166
Observational Study of the Use of Pegloticase (KRYSTEXXA®) in Refractory Chronic Gout
An observational study of Refractory Chronic Gout, run by Horizon Pharma Rheumatology LLC at 67 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Refractory Chronic Gout
- What is being tested?
- Pegloticase
- Type of treatment
- Injection or infusion
- How many people
- 188 participants
- Where
- 67 sitesUnited States
- Study dates
- 15 Nov 2011to 30 Jun 2017
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
67 sites in 1 country.
67 sites in 1 country
United States67 sites
Keystone Pain Institute, Ilumina Clinical Associates
Altoona, Pennsylvania, 16602
University of Michigan Health System
Ann Arbor, Michigan, 48109-5422
Arthritis Clinic of Northern Virginia, P.C.
Arlington, Virginia, 22205
Austin Regional Clinic
Austin, Texas, 78731
Walter Reed National Military Medical Center
Bethesda, Maryland, 20889-5630
Rheumatology Associates, PC
Birmingham, Alabama, 35205
UAB Rheumatology
Birmingham, Alabama, 35294
Bay Area Arthritis and Osteoporosis
Brandon, Florida, 33511
Global Research Partners & Consultants, Inc.
Calhoun, Georgia, 30701
Caro Health Plaza
Caro, Michigan, 48723
Physicians' Clinic of Iowa, P.C.
Cedar Rapids, Iowa, 52401
Low Country Rheumatology
Charleston, South Carolina, 29406
University of Chicago Medical Center
Chicago, Illinois, 60637
Mountain State Clinical Research
Clarksburg, West Virginia, 26301
Countryside Arthritis Center
Clearwater, Florida, 33761
NorthEast Rheumatology
Concord, North Carolina, 28025
Medvin Clinical Research
Covina, California, 91723
Denver Nephrologists, PC
Denver, Colorado, 80218
Duke University Medical Center
Durham, North Carolina, 27710
Saint Paul Rheumatology, PA
Eagan, Minnesota, 55121
Dr. Raj Marwah
El Paso, Texas, 79902
Horizon Research Group of Opelousas, LLC
Eunice, Louisiana, 70535
Rheumatic Disease Center, LLP
Glendale, Wisconsin, 53217
Infusion Associates
Grand Rapids, Michigan, 49525
Physicians East, PA
Greenville, North Carolina, 27832
Klein & Associates MD, PA.
Hagerstown, Maryland, 21740
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Diagnostic Clinic of Houston
Houston, Texas, 77004
Rheumatic Disease Clinical Research Center
Houston, Texas, 77004
Saadat Ansari, MD, LLC
Huntsville, Alabama, 35801
Diagnostic Rheumatology and Research PC
Indianapolis, Indiana, 46227
Science and Research Institute, Inc
Jupiter, Florida, 33458
Kansas City Internal Medicine
Kansas City, Missouri, 64114
Apex Clinical Research
Kennewick, Washington, 99336
Alliance Clinical Research, LLC
Laguna Hills, California, 92653
Advanced Medical Research, LLC
Lakewood, California, 90712
Justus J. Fiechtner, MD, PC
Lansing, Michigan, 48910
Arthritis Medical Clinic
Las Vegas, Nevada, 89118
Pacific Arthritis Care Center
Los Angeles, California, 90045
Arthritis & Osteoporosis Center of North Virginia
Manassas, Virginia, 20109
Ramesh C. Gupta, M.D.
Memphis, Tennessee, 38119
A & O Research Center
Miami, Florida, 33174
Arthritis and Diabetes Clinic, Inc.
Monroe, Louisiana, 71203
R Srinivasan, MD, Inc
Monterey Park, California, 91754
Central Kentucky Research Associates of Kentucky
Mount Sterling, Kentucky, 40353
Brigid Freyne, MD, Inc
Murrieta, California, 92563
Sentara Rheumatology Specialists
Norfolk, Virginia, 23502
Acme Research L.L.C.
Orangeburg, South Carolina, 29118
Research Integrity, LLC
Owensboro, Kentucky, 42303
Arthritis Research of Florida, Inc.
Palm Harbor, Florida, 34684
Family Clinical Trials, LLC
Pembroke Pines, Florida, 33026
Southern Ohio Rheumatology
Portsmouth, Ohio, 45662
Alliance Clinical Research
Poway, California, 92064
Shanahan Rheumatology and Immunotherapy, PLLC
Raleigh, North Carolina, 27617
Shores Rheumatology, PC
Saint Clair Shores, Michigan, 48081
Specialty Medical Clinic and Research Center
Sanford, North Carolina, 27330
Rheumatology Associates of Long Island
Smithtown, New York, 11787
Arthritis Research & Treatment Center
Stockbridge, Georgia, 30281
Jedidiah Clinical Research
Tampa, Florida, 33604
Midtown Medical Center
Tampa, Florida, 33614
Rheumatology Associates of North Jersey
Teaneck, New Jersey, 07666
New England Research Associates, LLC
Trumbull, Connecticut, 06611
Howard University Hospital
Washington D.C., District of Columbia, 20060
Washington DC Veteran's Affairs Medical Center
Washington D.C., District of Columbia, 20422
The Center for Rheumatology and Bone Research
Wheaton, Maryland, 20902
Clinical Pharmacology Study Groups
Worcester, Massachusetts, 01605
Reliant Medical Group, Inc.
Worcester, Massachusetts, 01605
About this study
Official title: Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
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.
Read the 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.
- Conditions
- Refractory Chronic Gout
- Being tested
- Pegloticase
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegloticaseBiological
Also called KRYSTEXXA®
Pegloticase 8 mg intravenous every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| Pegloticase | Participants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician. Receives: Biological: Pegloticase |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 52 weeks
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).
Measured: 52 weeks
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.
Measured: From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
Secondary outcomes (5)
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.
Measured: Week 24 and week 52
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.
Measured: Baseline, week 24 and week 48
Number of Swollen Joints Over Time
Measured: Baseline and weeks 24 and 52
Number of Tender Joints Over Time
Measured: Baseline and weeks 24 and 52
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.
Measured: Baseline and weeks 24 and 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2011 (estimated)
- Study start15 Nov 2011
- Primary completion30 Jun 2017
- Study completion30 Jun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Refractory Chronic GoutStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 188 participants (actual)
Sponsor and investigators
- Lead sponsor
- Horizon Pharma Rheumatology LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jeffery Nieves, PharmD, Study director, Horizon Pharma Rheumatology LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01466166
- Sponsor's protocol ID
- M0401
- Acronym
- EyesOnGOUT
- Status last verified
- Jun 2018
- First submitted
- 28 Oct 2011
- Last updated
- 11 Jan 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 21846852
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
- ClinicalTrials.gov ID
- NCT01466166
- Acronym / sponsor ID
- EyesOnGOUT / M0401
- Conditions
- Refractory Chronic Gout
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 188 participants (actual)
- Lead sponsor
- Horizon Pharma Rheumatology LLCIndustry (pharma, biotech, devices)
- Study start
- 15 Nov 2011 (actual)
- Primary completion
- 30 Jun 2017 (actual)
- Study completion
- 30 Jun 2017 (actual)
- First posted
- 7 Nov 2011 (estimated)
- Results
- Posted 11 Jan 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pegloticase | – | Biological: PegloticaseParticipants received pegloticase 8 mg by intravenous (IV) infusion every 2 weeks for up to 1 year, as prescribed by their treating physician. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegloticaseAlso: KRYSTEXXA®Pegloticase 8 mg intravenous every 2 weeks | Biological | Pegloticase |
Primary outcome
Number of Participants With Infusion Reactions
Time frame: 52 weeks
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.
Number of Participants With Anaphylaxis
Time frame: 52 weeks
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: 1. Respiratory compromise (e.g., dyspnea, wheeze-bronchospasm, stridor, reduced peak expiratory flow, hypoxemia). 2. 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).
Number of Participants With Immune Complex-related Events
Time frame: From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
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.
5 secondary outcomes
Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52
Time frame: Week 24 and week 52
Change From Baseline in Number of Gout Flares
Time frame: Baseline, week 24 and week 48
Number of Swollen Joints Over Time
Time frame: Baseline and weeks 24 and 52
Number of Tender Joints Over Time
Time frame: Baseline and weeks 24 and 52
Number of Palpable Tophi Over Time
Time frame: Baseline and weeks 24 and 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 21846852Sundy 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.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
Related trials for Refractory Chronic Gout
Active, not recruitingPhase 2
Evaluation of Efficacy and Safety of AR882 and XOI Co-administration in Uricase Treatment Failed Patients
Gout, Gouty Arthritis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffery Nieves, PharmD study director
Horizon Pharma Rheumatology LLC
67 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Refractory Chronic Gout, run by Horizon Pharma Rheumatology LLC at 67 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Jan 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Infusion Reactions (time frame: 52 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01466166, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jan 2019.