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Study of Pegloticase in Patients With Uncontrolled Gout Who Have Had a Kidney Transplant

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ClinicalTrials.gov Identifier: NCT04087720
Recruitment Status : Recruiting
First Posted : September 12, 2019
Last Update Posted : November 18, 2019
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Rheumatology LLC

Brief Summary:

This is a Phase 4, multicenter study of pegloticase in adult participants with uncontrolled gout who have undergone kidney transplantation. Approximately twenty participants will be enrolled. Study duration will be approximately 40 weeks.

The purpose of this study is to assess the efficacy and safety of pegloticase in kidney transplant participants with uncontrolled gout refractory to conventional urate lowering therapy.


Condition or disease Intervention/treatment Phase
Uncontrolled Gout Kidney Transplant Biological: Pegloticase Phase 4

Detailed Description:

This is a multicenter study of pegloticase in adult participants with uncontrolled gout who have undergone kidney transplantation.

The study design will include: 1) a Screening Period, lasting up to 28 days; 2) a 24-week treatment period which includes an End-of-Study (Week 24) /Early Termination Visit; 3) a safety follow-up phone/email Visit 30 days after the last infusion; and 4) a 3 month post-treatment follow up visit.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label, Efficacy and Safety Study of Pegloticase in Patients With Uncontrolled Gout Who Have Undergone Kidney Transplantation
Actual Study Start Date : September 9, 2019
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Gout
Drug Information available for: Pegloticase

Arm Intervention/treatment
Experimental: Pegloticase
Participants will receive 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
Biological: Pegloticase
Participants will receive 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22




Primary Outcome Measures :
  1. Proportion of serum uric acid responders (sUA <6 mg/dL) during Month 6 [ Time Frame: Month 6 ]
    Serum uric acid (sUA) responders are defined as participants achieving and maintaining sUA <6 mg/dL for at least 80% of the time during Month 6 (Weeks 20, 21, 22, 23, 24).


Secondary Outcome Measures :
  1. Proportion of serum uric responders (sUA <5 mg/dL) during Month 6 [ Time Frame: Month 6 ]
    Serum uric acid (sUA) responders are defined as participants achieving and maintaining sUA <5 mg/dL for at least 80% of the time during Month 6 (Weeks 20, 21, 22, 23, and 24).

  2. Proportion of participants with complete resolution of ≥ 1 tophi at Week 24 [ Time Frame: Week 24 ]
    Proportion of participants with complete resolution of ≥ 1 tophi (using digital photography) at Week 24 in participants with tophi at baseline.

  3. Mean change in Health Assessment Questionnaire (HAQ) pain score from baseline to Week 24 [ Time Frame: Week 24 ]
    The HAQ pain visual analog scale (VAS) is a measure of pain on a continuous 100 point scale. Participants were asked to indicate how much pain they had in the past week as a result of their illness by placing a vertical mark on a horizontal line from 0 (no pain) to 100 (severe pain). A score from 0 to 3 is obtained based on the location of the participant's mark.

  4. Mean change in Heath Assessment Questionnaire - Disability Index (HAQ-DI) score from baseline to Week 24 [ Time Frame: Week 24 ]
    The HAQ-DI is a self-reported assessment of how the participant's illness affects their ability to function in their daily life over the past week. The HAQ-DI for a participant is calculated as the mean of the following 8 category scores (range: 0 to 3): Dressing and Grooming, Arising, Eating, Walking, Hygiene, Reach, Grip, and Activities. A lower score demonstrates less disability.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Willing and able to give informed consent;
  • Willing and able to comply with the prescribed treatment protocol and evaluations for the duration of the study;
  • Adult men or women ≥ 18 years of age;
  • Is a recipient of a de novo kidney from a living or deceased donor and is >1 year post transplant prior to screening;
  • Is on a stable standard of care immunosuppression therapy for at least 3 months prior to screening;
  • Kidney allograft is functional at entry, based on an estimated GFR (eGFR) ≥ 15 mL/min/1.73m²;
  • Women of childbearing potential have a negative screening serum pregnancy test and will be required to use a medically approved form of birth control during their participation in the study;
  • Uncontrolled gout, defined as:

    1. Hyperuricemia during screening as documented by sUA ≥ 7 mg/dL during Screening and prior to entry into the Treatment Period (Note: the sUA may be repeated up to 3 times during the Screening Period to confirm eligibility), and
    2. Inability to maintain sUA <6 mg/dL on other urate-lowering therapy or intolerable side effects or contraindicated with conventional urate-lowering therapy, and
    3. At least 1 of the following:

    i. Evidence of tophaceous deposits ii. Recurrent gout flares defined as 2 or more flares in the 12 months prior to Screening iii. Presence of chronic gouty arthritis; and

  • Able to tolerate low-dose prednisone (<10 mg/day) as part of the required standard gout flare prophylaxis regimen for ≥ 1 week before the first infusion.

Exclusion Criteria:

  • Any other organ transplant beside kidney;
  • Any severe infection, unless treated and completely resolved at least 2 weeks prior to Day 1;
  • Chronic or active hepatitis B (HBV) infection;
  • Known history of hepatitis C virus RNA positivity unless treated and viral load is negative;
  • Known history of Human Immunodeficiency Virus (HIV) positivity;
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency (tested at the Screening Visit);
  • Decompensated congestive heart failure or hospitalization for congestive heart failure within 3 months of the Screening Visit, uncontrolled arrhythmia, treatment for acute coronary syndrome (myocardial infarction or unstable angina), or uncontrolled blood pressure (>160/100 mmHg) at the end of the Screening Period (Day 1 prior to infusion);
  • Pregnant, planning to become pregnant, breastfeeding, planning to impregnate female partner, or not using an effective form of birth control, as determined by the Investigator;
  • Prior treatment with pegloticase, another recombinant uricase (rasburicase), or concomitant therapy with a polyethylene glycol-conjugated drug;
  • Known allergy to pegylated products or history of anaphylactic reaction to a recombinant protein or porcine product;
  • Receipt of an investigational drug within 4 weeks or 5 half-lives, whichever is longer, prior to Day 1, or plans to take an investigational drug during the study;
  • Currently receiving systemic or radiologic treatment for ongoing cancer;
  • History of malignancy within 5 years other than non-melanoma skin cancer, in situ carcinoma of cervix, early stage renal cell cancer or early stage prostate cancer that has been completely resected >2 years prior to screening;
  • Uncontrolled hyperglycemia with a plasma glucose value >240 mg/dL at Screening that is not subsequently controlled by the end of the Screening Period;
  • Diagnosis of osteomyelitis;
  • Known history of hypoxanthine-guanine phosphoribosyl-transferase deficiency, such as Lesch-Nyhan and Kelley-Seegmiller syndrome;
  • Unsuitable candidate for the study, based on the opinion of the Investigator (e.g., cognitive impairment), such that participation might create undue risk to the subject or interfere with the subject's ability to comply with the protocol requirements or complete the study;
  • Currently receiving allopurinol, febuxostat or other urate lowering medications and unable to discontinue medication 7 days prior to Day 1; or
  • Currently receiving probenecid and unable to discontinue medication within 3 days, prior to Day 1.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04087720


Contacts
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Contact: Jeff Nieves, PharmD 866-479-6742 clinicaltrials@horizontherapeutics.com

Locations
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United States, Alabama
University of Alabama Birmingham Recruiting
Birmingham, Alabama, United States, 35294
Contact: Stephanie Clark    205-934-1444    sbiggers@uabmc.edu   
Principal Investigator: Kenneth Saag, M.D.         
United States, California
Amicis Research Center Recruiting
Northridge, California, United States, 91324
Contact: Tyrone Rosales, BS    818-924-4708    Tyrone.rosales@amicisresearch.com   
Principal Investigator: Pascal Dabel, MD         
United States, Colorado
Colorado Kidney Care Withdrawn
Denver, Colorado, United States, 80230
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Devin Wall    314-362-4109    devinwall@wustl.edu   
Principal Investigator: Rowena Delos Santos, M.D.         
United States, Pennsylvania
BRCR Global USA Recruiting
Camp Hill, Pennsylvania, United States, 17011
Contact: Braulio Vizcayno    561-447-0614 ext 700    brauliov@brcrglobal.com   
Principal Investigator: Kaushal Patel, M.D.         
Sponsors and Collaborators
Horizon Pharma Rheumatology LLC
Investigators
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Study Director: Jeff Nieves, PharmD Horizon Pharma Rheumatology LLC

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Responsible Party: Horizon Pharma Rheumatology LLC
ClinicalTrials.gov Identifier: NCT04087720     History of Changes
Other Study ID Numbers: HZNP-KRY-406
First Posted: September 12, 2019    Key Record Dates
Last Update Posted: November 18, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Gout
Arthritis
Joint Diseases
Musculoskeletal Diseases
Crystal Arthropathies
Rheumatic Diseases
Purine-Pyrimidine Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases