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Uncontrolled GoutPhase 4NCT04087720

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

A Phase 4 study testing Pegloticase in people with Uncontrolled Gout and Kidney Transplant, run by Amgen at 8 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Uncontrolled Gout, Kidney Transplant
What is being tested?
Pegloticase
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
8 sitesUnited States
Study dates
9 Sep 2019to 7 Sep 2021
Phase
Phase 4
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

Who may be able to join

  • 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;
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  • 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:
  • 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.

Who cannot join

  • 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);
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  • 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;
  • 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.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites

  • University of Alabama Birmingham

    Birmingham, Alabama, 35294

  • Coastal Medical Research

    Brunswick, Georgia, 31520

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • Nephrology Consultants

    Huntsville, Alabama, 35805

  • Keck School of Medicine of USC

    Los Angeles, California, 90033

  • Amicis Research Center

    Northridge, California, 91324

  • Genesis Clinical Research

    Tampa, Florida, 33614

  • Clear Lake Specialties

    Webster, Texas, 77598

Other trials in United States

About this study

Official title: A Multicenter, Open-Label, Efficacy and Safety Study of Pegloticase in Patients With Uncontrolled Gout Who Have Undergone Kidney Transplantation

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.

Read the detailed description

The study design includes: 1) a Screening Period, lasting up to 35 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 acquired from Horizon in 2024.

Being tested
Pegloticase

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PegloticaseBiological

    intravenous (IV) infusion

Study groups in NCT04087720
Study groupRoleWhat they receive
PegloticaseExperimentalParticipants receive 8 mg pegloticase every 2 weeks from Day 1 through Week 22
Receives: Biological: Pegloticase

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

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

Secondary outcomes (3)

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

    sUA < 5 mg/dL responders are defined as participants achieving and maintaining sUA <5 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.

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

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

    The HAQ-Pain score consists of a doubly anchored, horizontal VAS 15 cm in length, and rates a participant's pain over the past week from 0 to 100 with 0 = no pain and 100 = severe pain. Baseline is defined as the last measurement taken prior to the first infusion of pegloticase. The 95% confidence interval is a two-sided normal theory-based 95% confidence interval.

    Measured: Baseline, Weeks 6, 14, 20, 24

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

    The HAQ-DI is a self-reported assessment of how a 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: Dressing and Grooming, Arising, Eating, Walking, Hygiene, Reach, Grip, and Activities. The HAQ-DI ranges from 0 to 3 with higher values indicating higher disability. Baseline is defined as the last measurement taken prior to the first infusion of pegloticase. The 95% confidence interval is a two-sided normal theory-based 95% confidence interval.

    Measured: Baseline, Weeks 6, 14, 20, 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Sep 2019
  2. Study start9 Sep 2019
  3. Primary completion6 Jul 2021
  4. Study completion7 Sep 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04087720
Sponsor's protocol ID
HZNP-KRY-406
Status last verified
Jun 2024
First submitted
11 Sep 2019
Last updated
26 Jun 2024
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

Publications

  • 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.

    PubMed 40371056

  • 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.

    PubMed 37138473

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Pegloticase in people with Uncontrolled Gout and Kidney Transplant, run by Amgen at 8 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 13 inclusion and 19 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 26 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 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.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Jul 2022). 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?

Percentage of Serum Uric Acid (sUA) < 6 mg/dL Responders During Month 6 (time frame: Month 6 (Weeks 20, 21, 22, 23, 24)). 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 NCT04087720, which also lists 2 linked publications.

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 26 Jun 2024.

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