NCT04772313

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

A Phase 4, Multicenter, Open-label, Efficacy and Safety Trial of Pegloticase and Methotrexate Co-administered in Patients With Uncontrolled Gout Who Have Previously Received Pegloticase Monotherapy But Did Not Maintain a Serum Uric Acid Response

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

All participants who meet eligibility criteria at Screening will begin once-weekly subcutaneous (SC) MTX Run-in period. Participants must be able to tolerate MTX at a minimum dose of 15 mg during the 6-week MTX Run-in Period to be eligible to participate in the Pegloticase + MTX Treatment Period. All participants who meet the inclusion/exclusion criteria and complete the MTX Run-in Period will be considered enrolled participants. During the Pegloticase + MTX Treatment Period, pegloticase 8 mg will be administered IV every 2 weeks and MTX SC weekly. Two sequential cohorts of participants will be enrolled in this trial. Cohort 1 is targeted to enroll 10 participants who previously failed to maintain sUA response with pegloticase monotherapy and stopped pegloticase treatment without a history of pegloticase-related infusion reaction. If the safety assessment during Cohort 1 indicates that the pegloticase infusions are well tolerated, then the trial can begin enrolling Cohort 2 for 20 participants who failed to maintain sUA response with pegloticase monotherapy with or without a history of pegloticase-related clinically mild IRs. Study acquired from Horizon in 2024.

Conditions

• Uncontrolled Gout

Interventions

Type	Name	Description
BIOLOGICAL	Pegloticase	Participants will receive pegloticase with MTX for up to 24 weeks during the treatment period. Participants may opt to receive pegloticase with MTX for an additional 24 weeks.
DRUG	Methotrexate (MTX)	Participants will receive MTX during the run-in period then pegloticase with MTX for up to 24 weeks during the treatment period. Participants may opt to receive pegloticase with MTX for an additional 24 weeks.

Timeline

• Start date: 2021-03-08
• Primary completion: 2022-08-23
• Completion: 2023-04-24
• First posted: 2021-02-26
• Last updated: 2024-06-27
• Results posted: 2023-10-10

Locations

15 sites across 1 country: United States

Regulatory

• FDA-regulated drug study