REduCing Immunogenicity to PegloticasE (RECIPE) Study

Sponsor
University of Alabama at Birmingham (Other)
Overall Status
Completed
CT.gov ID
NCT03303989
Collaborator
University of Michigan (Other)
35
1
2
33.5
1

Study Details

Study Description

Brief Summary

Pegloticase treatment for chronic refractory gout is limited by immunogenicity. The investigators propose the REduCing Immunogenicity to PegloticasE (RECIPE) trial to begin to investigate the question of whether a short course of immune modulating therapy with mycophenolate mofetil can significantly and safely attenuate immunogenicity to pegloticase and ensure patients afflicted with chronic refractory gout have better treatment outcomes and improved quality of life.

Condition or Disease Intervention/Treatment Phase
  • Drug: Pegloticase 8 MG/ML [Krystexxa]
  • Drug: MMF
  • Drug: Placebo
  • Drug: Pegloticase 8 MG/ML [Krystexxa]
Phase 2

Detailed Description

Pegloticase is highly efficacious therapy for chronic refractory gout patients (n = > 400K in the US alone). It decreases serum urate (sUA) levels to often undetectable levels and reduces tophi burden. However, its long-term real world effectiveness is severely limited due to its immunogenicity caused by anti-pegloticase antibody formation. REducing Immunogenicity to PEgloticase (RECIPE) is a Phase II, double-blind, placebo controlled, proof-of-concept trial in 32 subjects initiating pegloticase for treatment of chronic refractory gout. RECIPE will investigate the preliminary efficacy and safety of using immune modulating therapy with mycophenolate mofetil (MMF) to prevent immunogenicity conferred by pegloticase. Subjects will be randomized 3:1 to receive MMF vs. placebo in addition to everyone receiving pegloticase. The co-primary aims of the RECIPE trial are to 1) determine if a 12 week course of immune modulating therapy with daily MMF can safely and significantly attenuate immunogenicity to pegloticase as determined by the proportion of participants achieving and maintaining an sUA less than or equal to 6 mg/dL through 12 weeks, compared to concurrent controls, and 2) to assess the incidence and types of adverse events and infusion reaction. After 12 weeks of co-administration, all participants will continue on pegloticase for an additional 12 weeks without combination MMF immune modulating therapy to evaluate the longer term benefits (durability) and safety of this approach. The secondary aims are to: 1) Determine the 6 month durability of immune modulation after discontinuation of the short course of MMF by: a) assessing the absolute change in sUA from baseline to Week 24, and Week 12 to Week 24 and b) determining the proportion of participants with sUA ≤ 6 mg/dL through 24 weeks, and Week 12 to Week 24; 2) Identify and characterize the pegloticase immune response by immunoglobulin isotypes (IgG and IgM), specificities, and antibody titer; and 3) Examine patient reported outcomes (PROs) using the NIH supported Patient Reported Outcomes Measurement Information System (PROMIS) and Gout Impact Scale (GIS) instruments. The University of Alabama at Birmingham (UAB) and the University of Michigan (UM), two large academic gout and immunology research centers, which in aggregate see nearly 10,000 gout patients annually, will serve as the two lead study sites and are very well-positioned to address the clinical and immunologic questions posed.

Study Design

Study Type:
Interventional
Actual Enrollment :
35 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
Phase II, double blind, placebo controlled multisite proof-of-concept trial in subjects initiating pegloticase for treatment of chronic goutPhase II, double blind, placebo controlled multisite proof-of-concept trial in subjects initiating pegloticase for treatment of chronic gout
Masking:
Double (Participant, Investigator)
Masking Description:
Double blind
Primary Purpose:
Treatment
Official Title:
REduCing Immunogenicity to PegloticasE (RECIPE) Study
Actual Study Start Date :
Jun 14, 2018
Actual Primary Completion Date :
Apr 27, 2020
Actual Study Completion Date :
Mar 31, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: pegloticase + MMF

Participants randomized to this arm will receive pegloticase + mycophenolate mofetil.

Drug: Pegloticase 8 MG/ML [Krystexxa]
Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
Other Names:
  • Krystexxa
  • Drug: MMF
    Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
    Other Names:
  • Cell Cept
  • Placebo Comparator: pegloticase + placebo

    Participants randomized to this arm will receive pegloticase + placebo

    Drug: Placebo
    Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.

    Drug: Pegloticase 8 MG/ML [Krystexxa]
    Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
    Other Names:
  • Krystexxa
  • Outcome Measures

    Primary Outcome Measures

    1. Proportion of Participants Achieving and Maintaining an sUA ≤ to 6 Milligram Per Deciliter (mg/dL) Through 12 Weeks [12 weeks]

      Proportion of participants achieving and maintaining an sUA ≤ to 6 mg/dL through 12 weeks, compared to concurrent controls.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Men and women > 18 years of age

    • Diagnosed with chronic refractory gout*

    • Defined as: Persons whose signs and symptoms are inadequately controlled with urate lowering therapy (e.g. xanthine oxidase inhibitors or uricosuric agents) at a medically appropriate dose or for whom these drugs are contraindicated.

    Exclusion Criteria:
    • Any serious acute bacterial infection (2 weeks prior to Visit 1), unless treated and completely resolved with antibiotics

    • Severe chronic or recurrent bacterial infections (such as recurrent pneumonia, chronic bronchiectasis)

    • Current immunocompromised condition, including current or chronic treatment with immunosuppressive agents

    • Subjects at risk for tuberculosis. Specifically, subjects with: i) current clinical, radiographic or laboratory evidence of active or latent TB; ii) a history of active TB within the last 3 years even if it was treated; iii) a history of active TB greater than 3 years ago unless there is documentation that the prior anti-TB treatment was appropriate in duration and type

    • Known Hepatitis B surface antigen-positive or Hepatitis B DNA positive subjects

    • Known Hepatitis C RNA-positive subjects

    • Human Immunodeficiency Virus (HIV) infection

    • G6PD deficiency (tested at Screening Visit 1)

    • Severe chronic renal impairment (glomerular filtration rate [GFR] <25 mL/min/1.73 m2) or currently on dialysis

    • Subjects having any transplant surgery requiring maintenance immunosuppressive therapy

    • Non-compensated congestive heart failure, uncontrolled arrhythmia, treatment for acute coronary syndrome (myocardial infarction or unstable angina), or hospitalization for congestive heart failure within 3 months of screening or uncontrolled blood pressure (>160/100 mm Hg) at baseline (Screening Visit 1 and Week 0/Baseline visits)

    • Participants who are pregnant, planning to become pregnant, breastfeeding, or not on an effective form of birth control (defined in Study Protocol section 7.1)

    • Prior treatment with pegloticase, another recombinant uricase, or concomitant therapy with a polyethylene glycol (PEG)-conjugated drug

    • Known allergy to pegylated products or history of anaphylactic reaction to a recombinant protein or porcine product

    • Subjects in whom MMF treatment is contraindicated or considered inappropriate

    • Recipient of an investigational drug within 4 weeks prior to study drug administration or plans to take an investigational agent during the study

    • Current liver disease as determined by alanine transaminase ALT or aspartate transaminase (AST) levels >3 times upper limit of normal

    • Currently receiving treatment for ongoing cancer, excluding non-melanoma skin cancer

    • History of malignancy within 5 years other than skin cancer or in situ carcinoma of cervix

    • Uncontrolled hyperglycemia with a plasma glucose value >240 mg/dL at screening

    • Diagnosed osteomyelitis

    • Individuals with hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) deficiency such as Lesch-Nyhan and Kelley-Seegmiller syndrome

    • Not good candidate for the study based on opinion of the Investigator (e.g., cognitive impairment) that might create undue risk to the participant or interfere with the participant's ability to comply with the protocol requirements, or to complete the study

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama at Birmingham Birmingham Alabama United States 35294

    Sponsors and Collaborators

    • University of Alabama at Birmingham
    • University of Michigan

    Investigators

    • Principal Investigator: Kenneth G Saag, MD, Professor

    Study Documents (Full-Text)

    More Information

    Publications

    None provided.
    Responsible Party:
    Kenneth Saag, MD, MSc, Professor of Medicine, University of Alabama at Birmingham
    ClinicalTrials.gov Identifier:
    NCT03303989
    Other Study ID Numbers:
    • 300000591
    First Posted:
    Oct 6, 2017
    Last Update Posted:
    Mar 16, 2022
    Last Verified:
    Mar 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    Participant Flow

    Recruitment Details
    Pre-assignment Detail
    Arm/Group Title Pegloticase + MMF Pegloticase + Placebo
    Arm/Group Description Participants randomized to this arm will receive pegloticase + mycophenolate mofetil. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. MMF: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Participants randomized to this arm will receive pegloticase + placebo Placebo: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
    Period Title: Overall Study
    STARTED 24 11
    COMPLETED 22 10
    NOT COMPLETED 2 1

    Baseline Characteristics

    Arm/Group Title Pegloticase + MMF Pegloticase + Placebo Total
    Arm/Group Description Participants randomized to this arm will receive pegloticase + mycophenolate mofetil. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. MMF: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Participants randomized to this arm will receive pegloticase + placebo Placebo: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Total of all reporting groups
    Overall Participants 22 10 32
    Age (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    55
    (9.4)
    55.5
    (10.7)
    55.2
    (9.7)
    Sex: Female, Male (Count of Participants)
    Female
    3
    13.6%
    1
    10%
    4
    12.5%
    Male
    19
    86.4%
    9
    90%
    28
    87.5%
    Ethnicity (NIH/OMB) (Count of Participants)
    Hispanic or Latino
    0
    0%
    0
    0%
    0
    0%
    Not Hispanic or Latino
    22
    100%
    10
    100%
    32
    100%
    Unknown or Not Reported
    0
    0%
    0
    0%
    0
    0%
    Race (NIH/OMB) (Count of Participants)
    American Indian or Alaska Native
    1
    4.5%
    0
    0%
    1
    3.1%
    Asian
    0
    0%
    0
    0%
    0
    0%
    Native Hawaiian or Other Pacific Islander
    1
    4.5%
    0
    0%
    1
    3.1%
    Black or African American
    5
    22.7%
    0
    0%
    5
    15.6%
    White
    15
    68.2%
    10
    100%
    25
    78.1%
    More than one race
    0
    0%
    0
    0%
    0
    0%
    Unknown or Not Reported
    0
    0%
    0
    0%
    0
    0%
    Region of Enrollment (participants) [Number]
    United States
    22
    100%
    10
    100%
    32
    100%
    American College of Rheumatology / European League Against Rheumatism Flare Criteria Score (Score on a scale) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [Score on a scale]
    13.5
    (2.8)
    13.8
    (2.7)
    13.5
    (2.6)
    Gout Flare History (flare within the last year) (Count of Participants)
    Yes
    15
    68.2%
    5
    50%
    20
    62.5%
    No
    7
    31.8%
    5
    50%
    12
    37.5%
    Duration of Gout (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    13.3
    (9.8)
    13.4
    (7.4)
    13.4
    (9.0)
    PROMIS Pain Intensity (units on a scale) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [units on a scale]
    50.8
    (11.3)
    45.0
    (12.4)
    49.0
    (11.8)
    PROMIS Physical Function (units on a scale) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [units on a scale]
    37.5
    (7.8)
    33.8
    (6.4)
    36.3
    (8.6)
    Gout Impact Score (Score on a scale) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [Score on a scale]
    45.7
    (7.5)
    46.4
    (7.1)
    45.9
    (7.3)
    Serum Urate Level (mg/dL) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [mg/dL]
    8.9
    (1.8)
    9.8
    (1.3)
    9.2
    (1.7)
    Body Mass Index (Count of Participants)
    25 to < 30
    3
    13.6%
    1
    10%
    4
    12.5%
    30 to < 45
    18
    81.8%
    7
    70%
    25
    78.1%
    ≥45
    1
    4.5%
    2
    20%
    3
    9.4%
    Comorbidities (Count of Participants)
    Yes
    3
    13.6%
    2
    20%
    5
    15.6%
    No
    19
    86.4%
    8
    80%
    27
    84.4%
    Yes
    8
    36.4%
    6
    60%
    14
    43.8%
    No
    14
    63.6%
    4
    40%
    18
    56.3%
    Yes
    18
    81.8%
    7
    70%
    25
    78.1%
    No
    4
    18.2%
    3
    30%
    7
    21.9%
    Yes
    8
    36.4%
    4
    40%
    12
    37.5%
    No
    14
    63.6%
    6
    60%
    20
    62.5%
    Yes
    4
    18.2%
    5
    50%
    9
    28.1%
    No
    18
    81.8%
    5
    50%
    23
    71.9%

    Outcome Measures

    1. Primary Outcome
    Title Proportion of Participants Achieving and Maintaining an sUA ≤ to 6 Milligram Per Deciliter (mg/dL) Through 12 Weeks
    Description Proportion of participants achieving and maintaining an sUA ≤ to 6 mg/dL through 12 weeks, compared to concurrent controls.
    Time Frame 12 weeks

    Outcome Measure Data

    Analysis Population Description
    32 participants received at least one dose of pegloticase and were included in modified intention-to-treat analyses.
    Arm/Group Title Pegloticase + MMF Pegloticase + Placebo
    Arm/Group Description Participants randomized to this arm will receive pegloticase + mycophenolate mofetil. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. MMF: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Participants randomized to this arm will receive pegloticase + placebo Placebo: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
    Measure Participants 22 10
    Yes
    19
    86.4%
    4
    40%
    No
    3
    13.6%
    6
    60%
    Statistical Analysis 1
    Statistical Analysis Overview Comparison Group Selection Pegloticase + MMF, Pegloticase + Placebo
    Comments Fisher's exact tests were performed to compare baseline and clinical characteristics between treatment groups as appropriate.
    Type of Statistical Test Superiority
    Comments Efficacy of MMF was examined by the proportion of responders in MMF+Peg compared to PBO+Peg. Rates of the primary outcome were compared using proportions and 95% confidence intervals and tested for differences using Fisher's exact test.
    Statistical Test of Hypothesis p-Value <0.01
    Comments
    Method Fisher Exact
    Comments

    Adverse Events

    Time Frame 36 weeks
    Adverse Event Reporting Description Adverse event was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related. Adverse Events were monitored/assessed based on organ system class.
    Arm/Group Title Pegloticase + MMF Pegloticase + Placebo
    Arm/Group Description Participants randomized to this arm will receive pegloticase + mycophenolate mofetil. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. MMF: Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Participants randomized to this arm will receive pegloticase + placebo Placebo: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months. Pegloticase 8 MG/ML [Krystexxa]: Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
    All Cause Mortality
    Pegloticase + MMF Pegloticase + Placebo
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 0/22 (0%) 0/10 (0%)
    Serious Adverse Events
    Pegloticase + MMF Pegloticase + Placebo
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 2/22 (9.1%) 1/10 (10%)
    Cardiac disorders
    Chest Pain 1/22 (4.5%) 1 0/10 (0%) 0
    Gastrointestinal disorders
    Abdominal Pain 1/22 (4.5%) 1 0/10 (0%) 0
    Injury, poisoning and procedural complications
    Motor Vehicle Crash 1/22 (4.5%) 1 0/10 (0%) 0
    Skin and subcutaneous tissue disorders
    Infusion Reaction 0/22 (0%) 0 1/10 (10%) 1
    Other (Not Including Serious) Adverse Events
    Pegloticase + MMF Pegloticase + Placebo
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 15/22 (68.2%) 7/10 (70%)
    Cardiac disorders
    Cardia 2/22 (9.1%) 2 1/10 (10%) 1
    Gastrointestinal disorders
    Gastrointestinal 4/22 (18.2%) 4 1/10 (10%) 1
    General disorders
    Other (Lab abnormality, anxiety, neurological, and oral pain) 9/22 (40.9%) 11 5/10 (50%) 5
    Infections and infestations
    Infections 2/22 (9.1%) 2 0/10 (0%) 0
    Musculoskeletal and connective tissue disorders
    Musculoskeletal (arthralgia, myalgia, low back pain, orthopedic trauma, bursitis tendonitis) 9/22 (40.9%) 19 1/10 (10%) 2
    Skin and subcutaneous tissue disorders
    Skin 2/22 (9.1%) 2 1/10 (10%) 1

    Limitations/Caveats

    [Not Specified]

    More Information

    Certain Agreements

    All Principal Investigators ARE employed by the organization sponsoring the study.

    Results Point of Contact

    Name/Title Jeff Foster
    Organization UNIVERSITY OF ALABAMA AT BIRMINGHAM
    Phone 2059966086
    Email pjfoster@uabmc.edu
    Responsible Party:
    Kenneth Saag, MD, MSc, Professor of Medicine, University of Alabama at Birmingham
    ClinicalTrials.gov Identifier:
    NCT03303989
    Other Study ID Numbers:
    • 300000591
    First Posted:
    Oct 6, 2017
    Last Update Posted:
    Mar 16, 2022
    Last Verified:
    Mar 1, 2022