Mode
Text Size
Log in / Sign up
Phase 4 Completed N=13 Treatment

Pegloticase and Methotrexate Co-administered in Participants With Uncontrolled Gout Who Previously Failed Pegloticase Monotherapy

Uncontrolled Gout
Source: ClinicalTrials.gov NCT04772313 ↗
Enrolled (actual)
13
Serious AEs
8.3%
Results posted
Oct 2023
Primary outcomePrimary: Percentage of Serum Uric Acid (sUA) Responders (sUA < 6 mg/dL) During Month 6 — 9.1 percentage of participants
◆ Published Evidence
No publication linked

No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.

Summary

This is a Phase 4, multicenter, open-label trial of pegloticase with methotrexate (MTX) in adult participants with uncontrolled gout who were previously treated with pegloticase without a concomitant immunomodulator and stopped pegloticase due to failure to maintain serum uric acid (sUA) response and/or a clinically mild infusion reaction (IR). Approximately 30 participants will be enrolled. Pegloticase + MTX will be administered for approximately 24 weeks, with an optional extension up to 48 weeks. The trial design will include 5 distinct components: 1. Screening Period, lasting up to 42 days; 2. 6-week MTX Tolerability Assessment Period (hereafter referred to as the MTX Run-in Period); 3. 24-week Pegloticase + MTX Treatment Period, which will include a Week 24/End of Trial/Early Termination Visit (subjects that end MTX and pegloticase treatment prior to the Week 24 will remain on trial for follow up until the Week 24 visit) 4. Optional Pegloticase + MTX Extension Period up to 24 weeks 5. 30-Day Post Treatment Follow -up

Outcome Measures

OutcomeResultp-value
PRIMARY
Percentage of Serum Uric Acid (sUA) Responders (sUA < 6 mg/dL) During Month 6
9.1 —
SECONDARY
Percentage of sUA Responders (sUA < 6 mg/dL) During Month 3
9.1 —
SECONDARY
Percentage of Participants Who Experienced Any of the Following Events From Day 1 to Week 24: Infusion Reaction (IR) Leading to Discontinuation of Treatment, Anaphylaxis or Meeting Individual Participant sUA Discontinuation Criteria
81.8 —
SECONDARY
Mean Change From Baseline in Urate Deposition Volume Measured by Dual Energy Computed Tomography (DECT) to Week 24
-0.073; -1.540; -1.207 —
SECONDARY
Mean Change From Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI) Score at Weeks 14 and 24
0.25; 0.10 —
SECONDARY
Mean Change From Baseline in Health Assessment Questionnaire (HAQ) Pain Score at Weeks 14 and 24
10.67; -4.00 —
SECONDARY
Mean Change From Baseline in HAQ Health Score at Weeks 14 and 24
8.00; -11.40 —

Eligibility 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 trial.
  • Adult men or women ≥18 years of age.
  • Uncontrolled gout, defined by the following criteria:
  • Hyperuricemia during the Screening Period, defined as sUA ≥6 mg/dL, and;
  • Failure to maintain normalization of sUA with xanthine oxidase inhibitors at the maximum medically appropriate dose or with a contraindication to xanthine oxidase inhibitor therapy based on medical record review or subject interview, and;
  • Symptoms of gout, including at least 1 of the following:
  • Presence of at least 1 tophus
  • Recurrent flares, defined as 2 or more flares in the 12 months prior to Screening
  • Presence of chronic gouty arthritis
  • Subject was previously treated with pegloticase without concomitant immunomodulation and stopped pegloticase due to failure to maintain sUA reduction response (had ≥1 sUA >6 mg/dL within 2 weeks post pegloticase infusion) and did not experience an IR (Cohort 1) and/or stopped pegloticase treatment due to pegloticase-related clinically mild IR (Cohort 2).
  • Subject for whom the last pegloticase infusion occurred >6 months prior to Screening.
  • Willing to discontinue any oral urate-lowering therapy for at least 7 days prior to Day 1 and remain off other urate-lowering therapy during the Pegloticase + MTX Treatment Period.
  • Women of childbearing potential (including those with an onset of menopause 160 kg (352 pounds) at Screening.
  • Any serious acute bacterial infection, unless treated and completely resolved with antibiotics at least 2 weeks prior to the Week 6 Visit.
  • Severe chronic or recurrent bacterial infections, such as recurrent pneumonia or chronic bronchiectasis.
  • Current or chronic treatment with systemic immunosuppressive agents, such as MTX, azathioprine, cyclosporine, leflunomide, cyclophosphamide or mycophenolate mofetil.
  • Current treatment with prednisone >10 mg/day or equivalent dose of another corticosteroid on a chronic basis (defined as 3 months or longer).
  • Known history of any solid organ transplant surgery requiring maintenance immunosuppressive therapy.
  • Known history of hepatitis B virus surface antigen positivity or hepatitis B DNA positivity, unless treated, viral load is negative and no chronic or active infection confirmed by hepatitis B virus serology.
  • Known history of hepatitis C virus RNA positivity, unless treated and viral load is negative.
  • Known history of human immunodeficiency virus positivity.
  • glucose-6-phosphate dehydrogenase (G6PD) deficiency (tested at the Screening Visit).
  • Severe chronic renal impairment (eGFR 160/100 mm Hg) prior to Week -6.
  • Pregnant, planning to become pregnant, breastfeeding, planning to impregnate female partner, or not on an effective form of birth control, as determined by the Investigator.
  • Prior treatment with another recombinant uricase (rasburicase) or concomitant therapy with a PEG-conjugated drug.
  • Known allergy to pegylated products or history of anaphylactic reaction to a recombinant protein or porcine product.
  • Contraindication to MTX treatment or MTX treatment considered inappropriate.
  • Known intolerance to MTX.
  • Receipt of an investigational drug within 4 weeks or 5 half-lives, whichever is longer, prior to MTX administration at Week -6 or plan to take an investigational drug during the trial.
  • Current liver disease, as determined by alanine transaminase (ALT) or aspartate transaminase (AST) >1.25 × upper limit of normal (ULN) or albumin <lower limit of normal at the Screening Visit.
  • Currently receiving systemic or radiologic treatment for ongoing cancer, excluding nonmelanoma skin cancer.
  • History of malignancy within 5 years other than non-melanoma skin cancer or in situ carcinoma of cervix.
  • White blood cell count <4.0 × 10^9/L, hematocrit <32% or platelet count <75 × 10^9/L.
  • Diagnosis o
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04772313). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

Back to search