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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 : September 12, 2019
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Rheumatology LLC

Tracking Information
First Submitted Date  ICMJE September 11, 2019
First Posted Date  ICMJE September 12, 2019
Last Update Posted Date September 12, 2019
Actual Study Start Date  ICMJE September 9, 2019
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 11, 2019)
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).
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: September 11, 2019)
  • 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).
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Pegloticase in Patients With Uncontrolled Gout Who Have Had a Kidney Transplant
Official Title  ICMJE A Multicenter, Open-Label, Efficacy and Safety Study of Pegloticase in Patients With Uncontrolled Gout Who Have Undergone Kidney Transplantation
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.

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Uncontrolled Gout
  • Kidney Transplant
Intervention  ICMJE Biological: Pegloticase
Participants will receive 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
Study Arms  ICMJE Experimental: Pegloticase
Participants will receive 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
Intervention: Biological: Pegloticase
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 11, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2021
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jeff Nieves, PharmD 866-479-6742 clinicaltrials@horizontherapeutics.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04087720
Other Study ID Numbers  ICMJE HZNP-KRY-406
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Horizon Pharma Rheumatology LLC
Study Sponsor  ICMJE Horizon Pharma Rheumatology LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Jeff Nieves, PharmD Horizon Pharma Rheumatology LLC
PRS Account Horizon Pharma Rheumatology LLC
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP