Trial Outcomes & Findings for A Study to Evaluate XEN1101 as Adjunctive Therapy in Focal Epilepsy (NCT NCT03796962)
NCT ID: NCT03796962
Last Updated: 2024-09-24
Results Overview
Median percent change in monthly (28 days) focal seizure frequency from baseline to DBP for XEN1101 versus placebo
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
325 participants
Primary outcome timeframe
From baseline (8 weeks prior to Day 0) through to the final dose (up to Day 56)
Results posted on
2024-09-24
Participant Flow
530 subjects were screened; 77 subjects screen failed; 124 subjects that entered the baseline period but were not randomized due seizure/eDiary-based eligibility criteria
Participant milestones
| Measure |
25 mg XEN1101
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
Placebo capsule
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
114
|
51
|
46
|
114
|
|
Overall Study
COMPLETED
|
88
|
43
|
45
|
109
|
|
Overall Study
NOT COMPLETED
|
26
|
8
|
1
|
5
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate XEN1101 as Adjunctive Therapy in Focal Epilepsy
Baseline characteristics by cohort
| Measure |
25 mg XEN1101
n=114 Participants
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
n=51 Participants
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
n=46 Participants
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
n=114 Participants
Placebo capsule
|
Total
n=325 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Customized
Age
|
38.7 years
STANDARD_DEVIATION 13.1 • n=5 Participants
|
41.7 years
STANDARD_DEVIATION 13.6 • n=7 Participants
|
40.0 years
STANDARD_DEVIATION 12.1 • n=5 Participants
|
42.9 years
STANDARD_DEVIATION 13.7 • n=4 Participants
|
40.8 years
STANDARD_DEVIATION 13.3 • n=21 Participants
|
|
Sex: Female, Male
Female
|
54 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
61 Participants
n=4 Participants
|
168 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
60 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
53 Participants
n=4 Participants
|
157 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
White or Caucasian
|
105 Participants
n=5 Participants
|
49 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
102 Participants
n=4 Participants
|
298 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Other
|
5 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Monthly seizure frequency in baseline
|
12.8 seizures/month (28 days)
n=5 Participants
|
14.5 seizures/month (28 days)
n=7 Participants
|
17.4 seizures/month (28 days)
n=5 Participants
|
13.4 seizures/month (28 days)
n=4 Participants
|
13.5 seizures/month (28 days)
n=21 Participants
|
PRIMARY outcome
Timeframe: From baseline (8 weeks prior to Day 0) through to the final dose (up to Day 56)Population: mITT
Median percent change in monthly (28 days) focal seizure frequency from baseline to DBP for XEN1101 versus placebo
Outcome measures
| Measure |
25 mg XEN1101
n=112 Participants
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
n=51 Participants
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
n=46 Participants
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
n=114 Participants
Placebo capsule
|
|---|---|---|---|---|
|
Median Percent Change in Focal Seizure Frequency
|
-52.8 Percent change
Interval -80.4 to -16.9
|
-46.4 Percent change
Interval -76.7 to -14.0
|
-33.2 Percent change
Interval -61.8 to 0.0
|
-18.2 Percent change
Interval -37.3 to 7.0
|
SECONDARY outcome
Timeframe: From baseline (8 weeks prior to Day 0) through to the final dose (up to Day 56)Population: mITT Population
Responders are defined as patients experiencing ≥50% reduction in monthly (28 days) focal seizure frequency from baseline to DBP
Outcome measures
| Measure |
25 mg XEN1101
n=112 Participants
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
n=51 Participants
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
n=46 Participants
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
n=114 Participants
Placebo capsule
|
|---|---|---|---|---|
|
50% XEN1101 Response Rate
|
61 Participants
|
22 Participants
|
13 Participants
|
17 Participants
|
SECONDARY outcome
Timeframe: From baseline (8 weeks prior to Day 0) through to the final dose (up to Day 56)Population: mITT population
Percent change from baseline in focal seizure frequency at Month 1 and Month 2 in the DBP
Outcome measures
| Measure |
25 mg XEN1101
n=112 Participants
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
n=51 Participants
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
n=46 Participants
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
n=114 Participants
Placebo capsule
|
|---|---|---|---|---|
|
Percent Change in Focal Seizure Frequency Over Time
Month 2 (Day 29 to Visit 8)
|
-64.7 Percent change from baseline
Interval -91.1 to -32.8
|
-52.2 Percent change from baseline
Interval -81.1 to -33.7
|
-30.3 Percent change from baseline
Interval -56.9 to 0.3
|
-21.8 Percent change from baseline
Interval -45.6 to 4.9
|
|
Percent Change in Focal Seizure Frequency Over Time
Month 1 (Day 1 to 28)
|
-55.3 Percent change from baseline
Interval -78.3 to -16.1
|
-40.0 Percent change from baseline
Interval -70.7 to -1.3
|
-40.1 Percent change from baseline
Interval -62.5 to -1.5
|
-14.9 Percent change from baseline
Interval -38.6 to 15.9
|
Adverse Events
25 mg XEN1101
Serious events: 3 serious events
Other events: 97 other events
Deaths: 0 deaths
20 mg XEN1101
Serious events: 2 serious events
Other events: 35 other events
Deaths: 0 deaths
10 mg XEN1101
Serious events: 2 serious events
Other events: 31 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 56 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
25 mg XEN1101
n=114 participants at risk
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
n=51 participants at risk
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
n=46 participants at risk
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
n=114 participants at risk
Placebo capsule
XEN1101: Oral dose
|
|---|---|---|---|---|
|
Psychiatric disorders
Confusional state
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Psychiatric disorders
Psychogenic seizure
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Psychiatric disorders
Psychotic disorder
|
0.00%
0/114 • 8 weeks
|
2.0%
1/51 • Number of events 1 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Psychiatric disorders
Somatic delusion
|
0.00%
0/114 • 8 weeks
|
2.0%
1/51 • Number of events 1 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Nervous system disorders
Dizziness
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Nervous system disorders
Muscle spasticity
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Nervous system disorders
Seizure
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Nervous system disorders
Partial seizures
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Nervous system disorders
Presyncope
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Metabolism and nutrition disorders
Hyponatremia
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Infections and infestations
Coronavirus infection
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Injury, poisoning and procedural complications
Pneumothorax traumatic
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.00%
0/114 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
Other adverse events
| Measure |
25 mg XEN1101
n=114 participants at risk
Capsule filled with 25 mg XEN1101
XEN1101: Oral dose
|
20 mg XEN1101
n=51 participants at risk
Capsule filled with 20 mg XEN1101
XEN1101: Oral dose
|
10 mg XEN1101
n=46 participants at risk
Capsule filled with 10 mg XEN1101
XEN1101: Oral dose
|
Placebo
n=114 participants at risk
Placebo capsule
XEN1101: Oral dose
|
|---|---|---|---|---|
|
Nervous system disorders
Dizziness
|
31.6%
36/114 • Number of events 36 • 8 weeks
|
25.5%
13/51 • Number of events 13 • 8 weeks
|
6.5%
3/46 • Number of events 3 • 8 weeks
|
7.0%
8/114 • Number of events 8 • 8 weeks
|
|
Nervous system disorders
Somnolence
|
14.9%
17/114 • Number of events 17 • 8 weeks
|
21.6%
11/51 • Number of events 11 • 8 weeks
|
10.9%
5/46 • Number of events 5 • 8 weeks
|
7.0%
8/114 • Number of events 8 • 8 weeks
|
|
Nervous system disorders
Headache
|
7.9%
9/114 • Number of events 9 • 8 weeks
|
11.8%
6/51 • Number of events 6 • 8 weeks
|
13.0%
6/46 • Number of events 6 • 8 weeks
|
7.9%
9/114 • Number of events 9 • 8 weeks
|
|
Nervous system disorders
Balance disorder
|
11.4%
13/114 • Number of events 13 • 8 weeks
|
7.8%
4/51 • Number of events 4 • 8 weeks
|
4.3%
2/46 • Number of events 2 • 8 weeks
|
1.8%
2/114 • Number of events 2 • 8 weeks
|
|
Nervous system disorders
Tremor
|
10.5%
12/114 • Number of events 12 • 8 weeks
|
5.9%
3/51 • Number of events 3 • 8 weeks
|
6.5%
3/46 • Number of events 3 • 8 weeks
|
1.8%
2/114 • Number of events 2 • 8 weeks
|
|
Nervous system disorders
Aphasia
|
7.0%
8/114 • Number of events 8 • 8 weeks
|
2.0%
1/51 • Number of events 1 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Nervous system disorders
Ataxia
|
4.4%
5/114 • Number of events 5 • 8 weeks
|
2.0%
1/51 • Number of events 1 • 8 weeks
|
6.5%
3/46 • Number of events 3 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Nervous system disorders
Dysarthria
|
7.0%
8/114 • Number of events 8 • 8 weeks
|
0.00%
0/51 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
Nervous system disorders
Memory impairment
|
5.3%
6/114 • Number of events 6 • 8 weeks
|
3.9%
2/51 • Number of events 2 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Nervous system disorders
Disturbance in attention
|
4.4%
5/114 • Number of events 5 • 8 weeks
|
5.9%
3/51 • Number of events 3 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Psychiatric disorders
Confusional state
|
5.3%
6/114 • Number of events 6 • 8 weeks
|
5.9%
3/51 • Number of events 3 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Psychiatric disorders
Anxiety
|
1.8%
2/114 • Number of events 2 • 8 weeks
|
9.8%
5/51 • Number of events 5 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
5.3%
6/114 • Number of events 6 • 8 weeks
|
|
Psychiatric disorders
Hallucination
|
0.00%
0/114 • 8 weeks
|
5.9%
3/51 • Number of events 3 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.00%
0/114 • 8 weeks
|
|
General disorders
Fatigue
|
12.3%
14/114 • Number of events 14 • 8 weeks
|
7.8%
4/51 • Number of events 4 • 8 weeks
|
10.9%
5/46 • Number of events 5 • 8 weeks
|
5.3%
6/114 • Number of events 6 • 8 weeks
|
|
General disorders
Gait disturbance
|
7.0%
8/114 • Number of events 8 • 8 weeks
|
3.9%
2/51 • Number of events 2 • 8 weeks
|
4.3%
2/46 • Number of events 2 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Gastrointestinal disorders
Nausea
|
6.1%
7/114 • Number of events 7 • 8 weeks
|
2.0%
1/51 • Number of events 1 • 8 weeks
|
2.2%
1/46 • Number of events 1 • 8 weeks
|
2.6%
3/114 • Number of events 3 • 8 weeks
|
|
Gastrointestinal disorders
Constipation
|
2.6%
3/114 • Number of events 3 • 8 weeks
|
5.9%
3/51 • Number of events 3 • 8 weeks
|
4.3%
2/46 • Number of events 2 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Eye disorders
Vision blurred
|
6.1%
7/114 • Number of events 7 • 8 weeks
|
2.0%
1/51 • Number of events 1 • 8 weeks
|
0.00%
0/46 • 8 weeks
|
0.88%
1/114 • Number of events 1 • 8 weeks
|
|
Infections and infestations
Urinary tract infection
|
1.8%
2/114 • Number of events 2 • 8 weeks
|
5.9%
3/51 • Number of events 3 • 8 weeks
|
8.7%
4/46 • Number of events 4 • 8 weeks
|
3.5%
4/114 • Number of events 4 • 8 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER