Trial Outcomes & Findings for An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE (NCT NCT04912856)

NCT ID: NCT04912856

Last Updated: 2025-02-14

Results Overview

Safety and tolerability of XEN496 as assessed by incidence and severity of AEs and SAEs

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

8 participants

Primary outcome timeframe

From Screening/Baseline through to 4 weeks post last dose

Results posted on

2025-02-14

Participant Flow

Participant milestones

Participant milestones
Measure
Group 1: XEN496 Treatment in Preceding Study
24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child. Placebo: Placebo sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Group 2: Placebo Treatment in Preceding Study
24-day blinded transition/titration period. Subjects, who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child. Placebo: Placebo sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Overall Study
STARTED
5
3
Overall Study
COMPLETED
1
0
Overall Study
NOT COMPLETED
4
3

Reasons for withdrawal

Reasons for withdrawal
Measure
Group 1: XEN496 Treatment in Preceding Study
24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child. Placebo: Placebo sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Group 2: Placebo Treatment in Preceding Study
24-day blinded transition/titration period. Subjects, who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child. Placebo: Placebo sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Overall Study
Sponsor Decision; not due to safety reasons
4
2
Overall Study
Not reported in synoptic CSR.
0
1

Baseline Characteristics

An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Group 1: XEN496 Treatment in Preceding Study
n=5 Participants
24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Group 2: Placebo Treatment in Preceding Study
n=3 Participants
24-day blinded transition/titration period. Subjects, who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue or complete the study treatment will be required to taper off study drug over a period of up to 15 days. XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Total
n=8 Participants
Total of all reporting groups
Age, Categorical
<=18 years
5 Participants
n=5 Participants
3 Participants
n=7 Participants
8 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Female
3 Participants
n=5 Participants
2 Participants
n=7 Participants
5 Participants
n=5 Participants
Sex: Female, Male
Male
2 Participants
n=5 Participants
1 Participants
n=7 Participants
3 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
5 Participants
n=5 Participants
3 Participants
n=7 Participants
8 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
White
3 Participants
n=5 Participants
3 Participants
n=7 Participants
6 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
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Region of Enrollment
Belgium
1 participants
n=5 Participants
2 participants
n=7 Participants
3 participants
n=5 Participants
Region of Enrollment
United States
3 participants
n=5 Participants
1 participants
n=7 Participants
4 participants
n=5 Participants
Region of Enrollment
Australia
1 participants
n=5 Participants
0 participants
n=7 Participants
0 participants
n=5 Participants

PRIMARY outcome

Timeframe: From Screening/Baseline through to 4 weeks post last dose

Safety and tolerability of XEN496 as assessed by incidence and severity of AEs and SAEs

Outcome measures

Outcome measures
Measure
Group 1: XEN496 Treatment in Preceding Study
n=5 Participants
24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Group 2: Placebo Treatment in Preceding Study
n=3 Participants
24-day blinded transition/titration period. Subjects, who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue or complete the study treatment will be required to taper off study drug over a period of up to 15 days. XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Number of participants with any AEs
5 Participants
3 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Number of participants with any TEAE
5 Participants
3 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Participants with any treatment related TEAE
1 Participants
2 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Participants with any TEAE leading to discontinuation of study drug
0 Participants
0 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Participants with any TEAE leading to a dose reduction or treatment interruption
1 Participants
1 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Participants with any Severe TEAE
1 Participants
0 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Participants with any Serious TEAE
2 Participants
1 Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Participants with any TEAE leading to death
0 Participants
0 Participants

Adverse Events

Group 1: XEN496 Treatment in Preceding Study

Serious events: 2 serious events
Other events: 5 other events
Deaths: 0 deaths

Group 2: Placebo Treatment in Preceding Study

Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Group 1: XEN496 Treatment in Preceding Study
n=5 participants at risk
24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Group 2: Placebo Treatment in Preceding Study
n=3 participants at risk
24-day blinded transition/titration period. Subjects, who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue or complete the study treatment will be required to taper off study drug over a period of up to 15 days. XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Gastrointestinal disorders
Gastroenteritis
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Respiratory, thoracic and mediastinal disorders
Pneumonia
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Nervous system disorders
Seizure cluster
20.0%
1/5 • Number of events 2 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Gastrointestinal disorders
Intestinal malrotation
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.

Other adverse events

Other adverse events
Measure
Group 1: XEN496 Treatment in Preceding Study
n=5 participants at risk
24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Subjects who discontinue will be required to taper off study drug over a period of up to 15 days XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Group 2: Placebo Treatment in Preceding Study
n=3 participants at risk
24-day blinded transition/titration period. Subjects, who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects. Optimally-tolerated dose level established during the transition/titration period will be maintained throughout the duration of open-label period unless dose adjustment is required. Subjects who discontinue or complete the study treatment will be required to taper off study drug over a period of up to 15 days. XEN496: XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Renal and urinary disorders
Urinary retention
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Investigations
Alanine aminotransferase increased
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Investigations
Gamma-glutamyltransferase increased
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Eye disorders
Retinal Pigmentation
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Investigations
Blood pressure systolic increased
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Gastrointestinal disorders
Salivary gland enlargement
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Gastrointestinal disorders
Constipation
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Gastroenteritis
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
General disorders
Peripheral swelling
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
General disorders
Swelling face
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Skin and subcutaneous tissue disorders
Erythema
20.0%
1/5 • Number of events 2 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
General disorders
Pyrexia
40.0%
2/5 • Number of events 2 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Viral upper respiratory tract infection
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Gastrointestinal disorders
Vomiting
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Otitis Media
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Respiratory, thoracic and mediastinal disorders
Cough
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Gastrointestinal disorders
Diarrhoea
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Investigations
Respiratory synctial virus test positive
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Congenital, familial and genetic disorders
Intestinal malrotation
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Gastrointestinal bacterial overgrowth
20.0%
1/5 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
0.00%
0/3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Gastrointestinal disorders
Blood in the stool
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Upper respiratory tract infection
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Pneumonia
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
66.7%
2/3 • Number of events 3 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Renal and urinary disorders
Urinary hesitation
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Respiratory tract infection
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
66.7%
2/3 • Number of events 2 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
General disorders
Fatigue
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Psychiatric disorders
Initial Insomnia
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Ear and labyrinth disorders
Ear Swelling
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Skin and subcutaneous tissue disorders
Dermatitis allergic
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Infections and infestations
Bronchitis viral
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Skin and subcutaneous tissue disorders
Blister
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Psychiatric disorders
Abnormal behaviour
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
Nervous system disorders
Epilepsy
0.00%
0/5 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.
33.3%
1/3 • Number of events 1 • Collection of adverse event data began at Visit 1 when consent was obtained and continued throughout the course subject's participation in the study. The intended duration of the collection period was 3 years. Following the termination of the study, the collection period was up to 2 years and 3 months.

Additional Information

Dr. Noam Butterfield

Xenon Pharmaceuticals

Phone: 604 484 3300

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: GT60