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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
intravenous (IV) infusion
University of Alabama Birmingham
Birmingham, Alabama, United States
Nephrology Consultants
Huntsville, Alabama, United States
Keck School of Medicine of USC
Los Angeles, California, United States
Amicis Research Center
Northridge, California, United States
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.
Time frame: Month 6 (Weeks 20, 21, 22, 23, 24)
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.
Time frame: 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
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.
Time frame: Baseline, Weeks 6, 14, 20, 24
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.
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Genesis Clinical Research
Tampa, Florida, United States
Coastal Medical Research
Brunswick, Georgia, United States
Duke University Medical Center
Durham, North Carolina, United States
Clear Lake Specialties
Webster, Texas, United States
Time frame: Baseline, Weeks 6, 14, 20, 24