Trial Outcomes & Findings for Pegylated Recombinant Mammalian Uricase (PEG-uricase) as Treatment for Refractory Gout (NCT NCT00111657)
NCT ID: NCT00111657
Last Updated: 2014-10-03
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Baseline to Day 105
Results posted on
2014-10-03
Participant Flow
Participant milestones
| Measure |
Single Arm - Pegloticase
|
|---|---|
|
Overall Study
STARTED
|
37
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
16
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pegylated Recombinant Mammalian Uricase (PEG-uricase) as Treatment for Refractory Gout
Baseline characteristics by cohort
| Measure |
Single Arm - Pegloticase
n=37 Participants
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
25 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
12 Participants
n=5 Participants
|
|
Age, Continuous
|
57.6 years
STANDARD_DEVIATION 14.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
37 participants
n=5 Participants
|
|
Baseline urate concentration (pUA)
|
10.8 mg/dL
STANDARD_DEVIATION 1.2 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Day 105Outcome measures
| Measure |
Single Arm
n=30 Participants
|
|---|---|
|
Reduction in Plasma Uric Acid to Less Than 6 mg/dL.
|
17 Participants
|
SECONDARY outcome
Timeframe: Basline and day 134Population: 30 subject assesed at baseline. 21 subjects who completed study were assesed at day 134
Count of tenderness and swelling of 68 joints
Outcome measures
| Measure |
Single Arm
n=30 Participants
|
|---|---|
|
Clinical Response: Number of Swollen and Tender Joints
Number of Tender joints at baseline
|
13 joints
Interval 6.0 to 21.0
|
|
Clinical Response: Number of Swollen and Tender Joints
Number of Tender joints at day 134
|
2 joints
Interval 1.0 to 9.0
|
|
Clinical Response: Number of Swollen and Tender Joints
Number of Swollen jonts at baseline
|
9 joints
Interval 5.0 to 20.0
|
|
Clinical Response: Number of Swollen and Tender Joints
Number of Swollen jonts at day 134
|
6 joints
Interval 2.0 to 11.0
|
SECONDARY outcome
Timeframe: baseline and 7 weeks after last infusionPopulation: Data was not collected for this outcome as a result of a separate pilot study demonstrating that the measure was not useful.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline then weeklyPopulation: These data were not calculated because the effect size of serum irate reduction in plasma was so robust that there was no utility in this assessment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline, then prior to infusions and 7 wks after last infusionPopulation: One subject withdrew prior to completing the first infusion and is not included in this analysis.
Number of patients who developed antibodies to PEG-uricase
Outcome measures
| Measure |
Single Arm
n=29 Participants
|
|---|---|
|
Development of Antibodies to PEG-uricase
|
15 participants
|
SECONDARY outcome
Timeframe: 2 hoursPopulation: One subject withdrew prior to completing the first infusion and is not included in this analysis.
The highest drug concentration in the blood after the first infusion of study drug.
Outcome measures
| Measure |
Single Arm
n=29 Participants
|
|---|---|
|
Infusion 1: Maximum Concentration (Cmax) Value
|
25.6 mU/mL
Standard Deviation 5.0
|
SECONDARY outcome
Timeframe: 21 days after the infusionPopulation: One subject withdrew prior to completing the first infusion and is not included in this analysis.
The lowest drug concentration in the blood after the first infusion of study drug.
Outcome measures
| Measure |
Single Arm
n=29 Participants
|
|---|---|
|
Infusion 1: Minimum Concentration (Cmin)
|
4.9 mU/mL
Standard Deviation 4.6
|
Adverse Events
Single Arm - Pegloticase
Serious events: 4 serious events
Other events: 30 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Single Arm - Pegloticase
n=30 participants at risk
|
|---|---|
|
Gastrointestinal disorders
Gastrointestinal bleeding.
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Gastrointestinal disorders
Bowel perforation.
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Cardiac disorders
Death
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Endocrine disorders
Hyperglycemia
|
3.3%
1/30 • Number of events 1 • Day 134
|
Other adverse events
| Measure |
Single Arm - Pegloticase
n=30 participants at risk
|
|---|---|
|
Cardiac disorders
Hypertension
|
13.3%
4/30 • Number of events 4 • Day 134
|
|
Cardiac disorders
Hypotension
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Gastrointestinal disorders
Abdominal cramping
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
3/30 • Number of events 4 • Day 134
|
|
Gastrointestinal disorders
Duodenal ulcer
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Gastrointestinal disorders
Heartburn
|
10.0%
3/30 • Number of events 4 • Day 134
|
|
Gastrointestinal disorders
Nausea
|
16.7%
5/30 • Number of events 6 • Day 134
|
|
Gastrointestinal disorders
Indigestion
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
General disorders
Chills
|
6.7%
2/30 • Number of events 3 • Day 134
|
|
General disorders
Fever
|
6.7%
2/30 • Number of events 3 • Day 134
|
|
General disorders
Lightheadedness
|
3.3%
1/30 • Number of events 2 • Day 134
|
|
Renal and urinary disorders
Urinary hesitancy
|
3.3%
1/30 • Number of events 2 • Day 134
|
|
Gastrointestinal disorders
Toothache
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Infections and infestations
Gastroenteritis
|
10.0%
3/30 • Number of events 3 • Day 134
|
|
Infections and infestations
Infected tophus
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Infections and infestations
Rectal abscess
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Infections and infestations
Skin abscess
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Infections and infestations
Thrush
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Infections and infestations
Upper respiratory infection
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Infections and infestations
Tooth abscess
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Infections and infestations
Urinary tract infection
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Blood and lymphatic system disorders
Anemia
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Endocrine disorders
Hyperkalemia
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
10.0%
3/30 • Number of events 3 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Charcot joint
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Draining tophus
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Gout flare
|
90.0%
27/30 • Number of events 119 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Pain in limb
|
6.7%
2/30 • Number of events 3 • Day 134
|
|
Nervous system disorders
Headache
|
10.0%
3/30 • Number of events 3 • Day 134
|
|
Nervous system disorders
Parasthesia
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Nervous system disorders
Tremor
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Eye disorders
Blurred vision
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Eye disorders
Conjunctival injection
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Psychiatric disorders
Confusion
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Psychiatric disorders
Decreased libido
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Psychiatric disorders
Excessive sleepiness
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Psychiatric disorders
Hallucinations
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Psychiatric disorders
Insomnia
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Psychiatric disorders
Labile affect
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Respiratory, thoracic and mediastinal disorders
Chest pain
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Respiratory, thoracic and mediastinal disorders
Chest tightness
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Respiratory, thoracic and mediastinal disorders
Obstructive sleep apnea
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxemia
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Skin and subcutaneous tissue disorders
Acne
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Skin and subcutaneous tissue disorders
Contact dermatitis
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.3%
4/30 • Number of events 5 • Day 134
|
|
Skin and subcutaneous tissue disorders
Foot ulcer
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Skin and subcutaneous tissue disorders
Hair loss
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Skin and subcutaneous tissue disorders
Leg ulcer
|
6.7%
2/30 • Number of events 2 • Day 134
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
6.7%
2/30 • Number of events 7 • Day 134
|
|
Skin and subcutaneous tissue disorders
Swellng of toes
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
6.7%
2/30 • Number of events 3 • Day 134
|
|
Skin and subcutaneous tissue disorders
Nevi
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Skin and subcutaneous tissue disorders
Seborrheic keratosis
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Cardiac disorders
Edema
|
10.0%
3/30 • Number of events 3 • Day 134
|
|
Endocrine disorders
Hyperglycemia
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Cardiac disorders
Syncope
|
3.3%
1/30 • Number of events 1 • Day 134
|
|
Musculoskeletal and connective tissue disorders
Chronic gouty arthritis
|
16.7%
5/30 • Number of events 5 • Day 134
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place