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

NCT04087720 | Completed Phase 4 Results FDA Drug

• 1 other identifier: HZNP-KRY-406
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 8

Brief Summary

The primary objective of this study is to evaluate the effect of pegloticase on the response rate of sustained serum uric acid (sUA) reduction to sUA \< 6 mg/dL during Month 6 of treatment.

Conditions

Uncontrolled Gout; Kidney Transplant

Outcome Measures

Primary Outcomes (1)

• Percentage of Serum Uric Acid (sUA) < 6 mg/dL Responders During Month 6

  sUA \< 6 mg/dL responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 6, which includes pre-infusion and post-infusion results at Week 20, results at Week 21, pre-infusion and post-infusion results at Week 22, results at Week 23, results at Week 24, and unscheduled assessments of sUA collected between Week 20 and Week 24. Two-sided Exact Clopper-Pearson confidence interval is used for the calculation of the 95% confidence interval.

  Month 6 (Weeks 20, 21, 22, 23, 24)

Secondary Outcomes (3)

• Percentage of sUA < 5 mg/dL Responders During Month 6

  Month 6 (Weeks 20, 21, 22, 23, and 24)

• Change From Baseline in Health Assessment Questionnaire (HAQ) Pain Visual Analog Scale (VAS) Score Through Week 24

  Baseline, Weeks 6, 14, 20, 24

• Change From Baseline in Heath Assessment Questionnaire - Disability Index (HAQ-DI) Score Through Week 24

  Baseline, Weeks 6, 14, 20, 24

Study Arms (1)

Pegloticase

EXPERIMENTAL

Participants receive 8 mg pegloticase every 2 weeks from Day 1 through Week 22

Biological: Pegloticase

Interventions

Pegloticase BIOLOGICAL

intravenous (IV) infusion

Pegloticase

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 glomerular filtration rate (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:
• 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
• Inability to maintain sUA \<6 mg/dL on other urate-lowering therapy or intolerable side effects or contraindicated with conventional urate-lowering therapy, and
• 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;
• 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.

You may not qualify if:

• 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 virus infection;
• Known history of hepatitis C virus ribonucleic acid (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 mm Hg) 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;

Sponsors & Collaborators

• Amgen: lead

Study Sites (8)

University of Alabama Birmingham

Birmingham, Alabama, 35294, United States

Location

Nephrology Consultants

Huntsville, Alabama, 35805, United States

Location

Keck School of Medicine of USC

Los Angeles, California, 90033, United States

Location

Amicis Research Center

Northridge, California, 91324, United States

Location

Genesis Clinical Research

Tampa, Florida, 33614, United States

Location

Coastal Medical Research

Brunswick, Georgia, 31520, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Clear Lake Specialties

Webster, Texas, 77598, United States

Location

Related Publications (2)

• Dalbeth N, Abdellatif A, Botson JK, Saag KG, Kumar A, Padnick-Silver L, Vranic Z, Marder BA, Becce F. Monosodium Urate Crystal Depletion and Bone Erosion Response in Kidney Transplant Recipients With Uncontrolled Gout Treated With Pegloticase: PROTECT Serial Dual-Energy Computed Tomography Findings. Transplant Direct. 2025 May 12;11(6):e1803. doi: 10.1097/TXD.0000000000001803. eCollection 2025 Jun.

  PMID: 40371056 DERIVED

• Abdellatif A, Zhao L, Chamberlain J, Cherny K, Xin Y, Marder BA, Scandling JD, Saag K. Pegloticase efficacy and safety in kidney transplant recipients; results of the phase IV, open-label PROTECT clinical trial. Clin Transplant. 2023 Sep;37(9):e14993. doi: 10.1111/ctr.14993. Epub 2023 May 3.

  PMID: 37138473 DERIVED

MeSH Terms

Interventions

Pegloticase

Results Point of Contact

• Title: Supra Verma, MD
• Organization: Horizon Therapeutics USA, Inc.

clinicaltrials@horizontherapeutics.com

866-479-6742

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 11, 2019
• First Posted: September 12, 2019
• Study Start: September 9, 2019
• Primary Completion: July 6, 2021
• Study Completion: September 7, 2021
• Last Updated: June 26, 2024
• Results First Posted: July 26, 2022

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Locations

US (8)