Trial Outcomes & Findings for Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE) (NCT NCT01966419)
NCT ID: NCT01966419
Last Updated: 2021-09-16
Results Overview
To support the primary endpoint of confirmed clinical response, the investigator rated participants on a 4-point staging scale, where higher scores are worse. The stages are described as: Stage 0/1 = Participant has no tremor of the hand when the wrist is extended (asterixis) and no confusion, even about where they are (disorientation) Stage 2 = Participant has tremor of the hand when the wrist is extended (asterixis) and is confused, not knowing where they are (disorientation) Stage 3 = Participant is tired, falling asleep, answers questions but is confused, and doesn't know where they are (Stupor, arousable but falls asleep, responsive to verbal stimuli, Obvious confusion, Gross disorientation) Stage 4 = Participant is unconscious (in a coma)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
231 participants
Primary outcome timeframe
Baseline to End of Study (through 3 hours post end-of-infusion)
Results posted on
2021-09-16
Participant Flow
A total of 231 participants were enrolled (randomized) into the trial at multiple sites in the United States, Australia and Europe.
Participant milestones
| Measure |
Placebo
Participants receive matching placebo up to 5 days via continuous IV infusion in addition to standard of care (SOC)
|
Ornithine Phenylacetate
Participants receive ornithine phenylacetate for up to 5 days via continuous IV infusion in addition to SOC
|
|---|---|---|
|
Overall Study
STARTED
|
115
|
116
|
|
Overall Study
Intent to Treat Population (ITT)T)115
|
115
|
116
|
|
Overall Study
Safety Analysis Set (SAS)
|
112
|
114
|
|
Overall Study
COMPLETED
|
74
|
80
|
|
Overall Study
NOT COMPLETED
|
41
|
36
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE)
Baseline characteristics by cohort
| Measure |
Placebo
n=115 Participants
Participants receive matching placebo up to 5 days via continuous IV infusion in addition to SOC
|
Ornithine Phenylacetate
n=116 Participants
Participants receive ornithine phenylacetate up to 5 days via continuous IV infusion in addition to SOC
|
Total
n=231 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
60 years
STANDARD_DEVIATION 9.5 • n=5 Participants
|
50 years
STANDARD_DEVIATION 9.8 • n=7 Participants
|
59 years
STANDARD_DEVIATION 9.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
37 Participants
n=5 Participants
|
44 Participants
n=7 Participants
|
81 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
78 Participants
n=5 Participants
|
72 Participants
n=7 Participants
|
150 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
57 Participants
n=5 Participants
|
55 Participants
n=7 Participants
|
112 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
51 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
101 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
54 Participants
n=5 Participants
|
59 Participants
n=7 Participants
|
113 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
51 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
101 Participants
n=5 Participants
|
|
Region of Enrollment
Hungary
|
1 participants
n=5 Participants
|
4 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
62 participants
n=5 Participants
|
66 participants
n=7 Participants
|
128 participants
n=5 Participants
|
|
Region of Enrollment
Czechia
|
4 participants
n=5 Participants
|
1 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Region of Enrollment
Russia
|
7 participants
n=5 Participants
|
5 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
3 participants
n=5 Participants
|
10 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Region of Enrollment
Austria
|
2 participants
n=5 Participants
|
5 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
6 participants
n=5 Participants
|
1 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Denmark
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
11 participants
n=5 Participants
|
11 participants
n=7 Participants
|
22 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Bulgaria
|
2 participants
n=5 Participants
|
1 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Region of Enrollment
France
|
11 participants
n=5 Participants
|
10 participants
n=7 Participants
|
21 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to End of Study (through 3 hours post end-of-infusion)Population: Intention to Treat Analysis Set (Note: all participants did not start at the same time)
To support the primary endpoint of confirmed clinical response, the investigator rated participants on a 4-point staging scale, where higher scores are worse. The stages are described as: Stage 0/1 = Participant has no tremor of the hand when the wrist is extended (asterixis) and no confusion, even about where they are (disorientation) Stage 2 = Participant has tremor of the hand when the wrist is extended (asterixis) and is confused, not knowing where they are (disorientation) Stage 3 = Participant is tired, falling asleep, answers questions but is confused, and doesn't know where they are (Stupor, arousable but falls asleep, responsive to verbal stimuli, Obvious confusion, Gross disorientation) Stage 4 = Participant is unconscious (in a coma)
Outcome measures
| Measure |
Placebo
n=115 Participants
Participants receive matching placebo up to 5 days via continuous IV infusion in addition to SOC
|
Ornithine Phenylacetate
n=116 Participants
Participants receive ornithine phenylacetate up to 5 days via continuous IV infusion in addition to SOC
|
|---|---|---|
|
Percentage of Participants in Each HE Stage
Day 2, 5 pm : Stage 2
|
57 percentage of participants
|
61 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 5 pm : Stage 0/1
|
16 percentage of participants
|
16 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Baseline : Stage 4
|
11 percentage of participants
|
6 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Baseline : Stage 3
|
30 percentage of participants
|
40 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Baseline : Stage 2
|
71 percentage of participants
|
68 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Baseline : Stage 0/1
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Baseline : Missing
|
3 percentage of participants
|
2 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 7 am : Stage 4
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 7 am : Stage 3
|
0 percentage of participants
|
4 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 7 am : Stage 2
|
2 percentage of participants
|
3 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 7 am : Stage 0/1
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 7 am : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 5 pm : Stage 4
|
0 percentage of participants
|
5 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 5 pm : Stage 3
|
7 percentage of participants
|
14 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 5 pm : Stage 2
|
32 percentage of participants
|
36 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 5 pm : Stage 0/1
|
0 percentage of participants
|
1 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 1, 5 pm : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 7 am : Stage 4
|
3 percentage of participants
|
4 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 7 am : Stage 3
|
21 percentage of participants
|
15 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 7 am : Stage 2
|
61 percentage of participants
|
69 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 7 am : Stage 0/1
|
10 percentage of participants
|
10 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 7 am : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 5 pm : Stage 4
|
3 percentage of participants
|
4 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 5 pm : Stage 3
|
14 percentage of participants
|
9 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 2, 5 pm : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 7 am : Stage 4
|
3 percentage of participants
|
2 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 7 am : Stage 3
|
15 percentage of participants
|
10 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 7 am : Stage 2
|
50 percentage of participants
|
55 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 7 am : Stage 0/1
|
21 percentage of participants
|
25 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 7 am : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 5 pm : Stage 4
|
3 percentage of participants
|
3 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 5 pm : Stage 3
|
7 percentage of participants
|
9 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 5 pm : Stage 2
|
46 percentage of participants
|
45 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 5 pm : Stage 0/1
|
20 percentage of participants
|
27 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 3, 5 pm : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 7 am : Stage 4
|
2 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 7 am : Stage 3
|
5 percentage of participants
|
9 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 7 am : Stage 2
|
42 percentage of participants
|
40 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 7 am : Stage 0/1
|
28 percentage of participants
|
30 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 7 am : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 5 pm : Stage 4
|
2 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 5 pm : Stage 3
|
5 percentage of participants
|
7 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 5 pm : Stage 2
|
42 percentage of participants
|
34 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 5 pm : Stage 0/1
|
28 percentage of participants
|
28 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 4, 5 pm : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 7 am : Stage 4
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 7 am : Stage 0/1
|
23 percentage of participants
|
32 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 7 am : Stage 2
|
29 percentage of participants
|
28 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 7 am : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 5 pm : Stage 4
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 5 pm : Stage 3
|
8 percentage of participants
|
3 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 5 pm : Stage 2
|
24 percentage of participants
|
22 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 7 am : Stage 4
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 7 am : Stage 3
|
10 percentage of participants
|
3 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 7 am : Stage 2
|
21 percentage of participants
|
24 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 7 am : Stage 0/1
|
23 percentage of participants
|
27 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 7 am : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 5 pm : Stage 4
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 5 pm : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 5 pm : Stage 0/1
|
23 percentage of participants
|
28 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 5 pm : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 5 pm : Stage 3
|
3 percentage of participants
|
3 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 5 pm : Stage 2
|
5 percentage of participants
|
7 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 6, 5 pm : Stage 0/1
|
6 percentage of participants
|
5 percentage of participants
|
|
Percentage of Participants in Each HE Stage
End of Study : Stage 4
|
5 percentage of participants
|
3 percentage of participants
|
|
Percentage of Participants in Each HE Stage
End of Study : Stage 3
|
10 percentage of participants
|
7 percentage of participants
|
|
Percentage of Participants in Each HE Stage
End of Study : Stage 2
|
31 percentage of participants
|
28 percentage of participants
|
|
Percentage of Participants in Each HE Stage
End of Study : Stage 0/1
|
45 percentage of participants
|
55 percentage of participants
|
|
Percentage of Participants in Each HE Stage
End of Study : Missing
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each HE Stage
Day 5, 7 am : Stage 3
|
9 percentage of participants
|
7 percentage of participants
|
Adverse Events
Placebo
Serious events: 34 serious events
Other events: 31 other events
Deaths: 15 deaths
Ornithine Phenylacetate
Serious events: 29 serious events
Other events: 30 other events
Deaths: 11 deaths
Serious adverse events
| Measure |
Placebo
n=112 participants at risk
Participants receive matching placebo up to 5 days via continuous IV infusion in addition to SOC
|
Ornithine Phenylacetate
n=114 participants at risk
Participants receive ornithine phenylacetate up to 5 days via continuous IV infusion in addition to SOC
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Cardiac disorders
Cardio-respiratory arrest
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Cardiac disorders
Ventricular tachycardia
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Gastrointestinal disorders
Ascites
|
1.8%
2/112 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Gastrointestinal disorders
Localised intraabdominal fluid collection
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
General disorders
General physical health deterioration
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
General disorders
Generalised oedema
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
General disorders
Multi-organ failure
|
3.6%
4/112 • Number of events 4 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
General disorders
Oedema peripheral
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
General disorders
Pyrexia
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Hepatobiliary disorders
Chronic hepatic failure
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
1.8%
2/114 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Hepatobiliary disorders
Hepatic cirrhosis
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Hepatobiliary disorders
Hepatic failure
|
1.8%
2/112 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
1.8%
2/114 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Hepatobiliary disorders
Hepatic function abnormal
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Erysipelas
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Lobar pneumonia
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Peritonitis bacterial
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Pneumonia
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
2.6%
3/114 • Number of events 3 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Sepsis
|
1.8%
2/112 • Number of events 3 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
1.8%
2/114 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Septic shock
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Metabolism and nutrition disorders
Hypovolaemia
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatocellular carcinoma
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Nervous system disorders
Encephalopathy
|
1.8%
2/112 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Nervous system disorders
Epilepsy
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Nervous system disorders
Hepatic encephalopathy
|
8.0%
9/112 • Number of events 9 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
7.9%
9/114 • Number of events 9 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Nervous system disorders
Intracranial pressure increased
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Nervous system disorders
Thalamus haemorrhage
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Renal and urinary disorders
Anuria
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Renal and urinary disorders
Renal failure
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Renal and urinary disorders
Renal failure acute
|
2.7%
3/112 • Number of events 3 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Reproductive system and breast disorders
Pelvic haemorrhage
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
1.8%
2/112 • Number of events 2 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Hepatic hydrothorax
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
0.00%
0/112 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Vascular disorders
Hypotension
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.00%
0/114 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Vascular disorders
Shock haemorrhagic
|
0.89%
1/112 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
0.88%
1/114 • Number of events 1 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
Other adverse events
| Measure |
Placebo
n=112 participants at risk
Participants receive matching placebo up to 5 days via continuous IV infusion in addition to SOC
|
Ornithine Phenylacetate
n=114 participants at risk
Participants receive ornithine phenylacetate up to 5 days via continuous IV infusion in addition to SOC
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.1%
8/112 • Number of events 11 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
11.4%
13/114 • Number of events 14 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
General disorders
Pyrexia
|
6.2%
7/112 • Number of events 7 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
5.3%
6/114 • Number of events 6 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Infections and infestations
Urinary tract infection
|
3.6%
4/112 • Number of events 4 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
7.0%
8/114 • Number of events 8 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
7.1%
8/112 • Number of events 10 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
7.9%
9/114 • Number of events 9 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
8.0%
9/112 • Number of events 9 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
4.4%
5/114 • Number of events 5 • 20 days
Adverse events are reported in the safety population. All serious adverse events are reported. Non-serious treatment-emergent adverse events are reported at the 5% reporting threshold.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place