Trial Outcomes & Findings for Study of Pegloticase in Participants With Uncontrolled Gout Who Have Had a Kidney Transplant (NCT NCT04087720)
NCT ID: NCT04087720
Last Updated: 2024-06-26
Results Overview
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.
COMPLETED
PHASE4
20 participants
Month 6 (Weeks 20, 21, 22, 23, 24)
2024-06-26
Participant Flow
Participant milestones
| Measure |
Pegloticase
Participants received 8 mg pegloticase by intravenous (IV) infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Pegloticase
Participants received 8 mg pegloticase by intravenous (IV) infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Withdrawal by Subject - COVID-19 Concerns
|
3
|
Baseline Characteristics
Study of Pegloticase in Participants With Uncontrolled Gout Who Have Had a Kidney Transplant
Baseline characteristics by cohort
| Measure |
Pegloticase
n=20 Participants
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Age, Continuous
|
53.9 years
STANDARD_DEVIATION 10.87 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Month 6 (Weeks 20, 21, 22, 23, 24)Population: Intent-to-treat (ITT) population: participants who received at least 1 dose of pegloticase. Participants in the ITT population with no lapse or cessation in treatment due to COVID-19 prior to the analysis time-point.
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.
Outcome measures
| Measure |
Pegloticase
n=18 Participants
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Percentage of Serum Uric Acid (sUA) < 6 mg/dL Responders During Month 6
|
88.9 percentage of participants
Interval 65.3 to 98.6
|
SECONDARY outcome
Timeframe: Month 6 (Weeks 20, 21, 22, 23, and 24)Population: ITT population: participants who received at least 1 dose of pegloticase. Participants in the ITT population with no lapse or cessation in treatment due to COVID-19 prior to the analysis time-point.
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.
Outcome measures
| Measure |
Pegloticase
n=18 Participants
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Percentage of sUA < 5 mg/dL Responders During Month 6
|
88.9 percentage of participants
Interval 65.3 to 98.6
|
SECONDARY outcome
Timeframe: Baseline, Weeks 6, 14, 20, 24Population: ITT population: participants who received at least 1 dose of pegloticase. Participants with an assessment at given time point.
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.
Outcome measures
| Measure |
Pegloticase
n=20 Participants
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Change From Baseline in Health Assessment Questionnaire (HAQ) Pain Visual Analog Scale (VAS) Score Through Week 24
Change at Week 6
|
-16.11 score on a scale
Standard Deviation 29.230
|
|
Change From Baseline in Health Assessment Questionnaire (HAQ) Pain Visual Analog Scale (VAS) Score Through Week 24
Change at Week 14
|
-34.81 score on a scale
Standard Deviation 24.025
|
|
Change From Baseline in Health Assessment Questionnaire (HAQ) Pain Visual Analog Scale (VAS) Score Through Week 24
Change at Week 20
|
-34.63 score on a scale
Standard Deviation 27.543
|
|
Change From Baseline in Health Assessment Questionnaire (HAQ) Pain Visual Analog Scale (VAS) Score Through Week 24
Change at Week 24
|
-33.05 score on a scale
Standard Deviation 31.590
|
SECONDARY outcome
Timeframe: Baseline, Weeks 6, 14, 20, 24Population: ITT population: participants who received at least 1 dose of pegloticase. Participants with an assessment at given time point.
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.
Outcome measures
| Measure |
Pegloticase
n=20 Participants
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Change From Baseline in Heath Assessment Questionnaire - Disability Index (HAQ-DI) Score Through Week 24
Change at Week 6
|
-0.30 score on a scale
Standard Deviation 0.522
|
|
Change From Baseline in Heath Assessment Questionnaire - Disability Index (HAQ-DI) Score Through Week 24
Change at Week 14
|
-0.52 score on a scale
Standard Deviation 0.616
|
|
Change From Baseline in Heath Assessment Questionnaire - Disability Index (HAQ-DI) Score Through Week 24
Change at Week 20
|
-0.38 score on a scale
Standard Deviation 0.523
|
|
Change From Baseline in Heath Assessment Questionnaire - Disability Index (HAQ-DI) Score Through Week 24
Change at Week 24
|
-0.25 score on a scale
Standard Deviation 0.611
|
Adverse Events
Pegloticase
Serious adverse events
| Measure |
Pegloticase
n=20 participants at risk
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Gastrointestinal disorders
Duodenal ulcer
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Gastrointestinal disorders
Enterocolonic fistula
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Cellulitis
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Diverticulitis
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Localised infection
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
Other adverse events
| Measure |
Pegloticase
n=20 participants at risk
Participants received 8 mg pegloticase by IV infusion every 2 weeks from Day 1 through Week 22
|
|---|---|
|
Cardiac disorders
Tachycardia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Ear and labyrinth disorders
Inner ear disorder
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Ear and labyrinth disorders
Tinnitus
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Ear and labyrinth disorders
Vertigo
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Eye disorders
Conjunctival haemorrhage
|
10.0%
2/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Eye disorders
Ocular hyperaemia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Eye disorders
Photophobia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Gastrointestinal disorders
Abdominal hernia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Gastrointestinal disorders
Haemorrhoids
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Gastrointestinal disorders
Melaena
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Gastrointestinal disorders
Stomatitis
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
General disorders
Asthenia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
General disorders
Oedema
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
General disorders
Pyrexia
|
10.0%
2/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Cellulitis
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Pneumonia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Skin bacterial infection
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Infections and infestations
Subcutaneous abscess
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Injury, poisoning and procedural complications
Eye injury
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Injury, poisoning and procedural complications
Wound
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Investigations
Alanine aminotransferase increased
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Investigations
Aspartate aminotransferase increased
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Investigations
Blood calcium decreased
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Investigations
Haemoglobin decreased
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Metabolism and nutrition disorders
Gout
|
45.0%
9/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.0%
2/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Musculoskeletal and connective tissue disorders
Gouty tophus
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Nervous system disorders
Headache
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Nervous system disorders
Hypoaesthesia
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Nervous system disorders
Presyncope
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
2/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
|
Skin and subcutaneous tissue disorders
Blister
|
5.0%
1/20 • From the date of first dose of treatment through 30 days after the last dose of treatment (up to Week 22 plus 30 days).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
- Publication restrictions are in place
Restriction type: OTHER