Trial Outcomes & Findings for Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep (NCT NCT05301894)
NCT ID: NCT05301894
Last Updated: 2026-02-13
Results Overview
A TEAE is an adverse event (AE) that was not present prior to the initiation of study treatment or was an already present condition that worsened either in intensity or frequency following the initiation of study treatment.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
Results posted on
2026-02-13
Participant Flow
Participant milestones
| Measure |
NBI-827104
NBI-827104 administered orally
|
|---|---|
|
Overall Study
STARTED
|
19
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
19
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
19
|
Reasons for withdrawal
| Measure |
NBI-827104
NBI-827104 administered orally
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Study Terminated by Sponsor
|
15
|
|
Overall Study
Lack of Efficacy
|
2
|
Baseline Characteristics
Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
Baseline characteristics by cohort
| Measure |
NBI-827104
n=19 Participants
NBI-827104 administered orally
|
|---|---|
|
Age, Continuous
|
9.04 years
STANDARD_DEVIATION 1.78 • n=41 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=41 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
18 Participants
n=41 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
1 Participants
n=41 Participants
|
PRIMARY outcome
Timeframe: Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)Population: All enrolled participants who took at least 1 dose of NBI-827104.
A TEAE is an adverse event (AE) that was not present prior to the initiation of study treatment or was an already present condition that worsened either in intensity or frequency following the initiation of study treatment.
Outcome measures
| Measure |
NBI-827104
n=19 Participants
NBI-827104 administered orally
|
|---|---|
|
The Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
|
2 Participants
|
Adverse Events
NBI-827104
Serious events: 2 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
NBI-827104
n=19 participants at risk
NBI-827104 administered orally
|
|---|---|
|
Infections and infestations
Osteomyelitis acute
|
5.3%
1/19 • Number of events 1 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Seizure
|
5.3%
1/19 • Number of events 2 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Seizure cluster
|
5.3%
1/19 • Number of events 1 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
Other adverse events
| Measure |
NBI-827104
n=19 participants at risk
NBI-827104 administered orally
|
|---|---|
|
Eye disorders
Ocular hyperaemia
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Gastrointestinal disorders
Abdominal pain
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Gastrointestinal disorders
Diarrhoea
|
21.1%
4/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Gastrointestinal disorders
Vomiting
|
31.6%
6/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
General disorders
Fatigue
|
21.1%
4/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
General disorders
Gait disturbance
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
General disorders
Influenza like illness
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
General disorders
Pyrexia
|
15.8%
3/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
General disorders
Thirst
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Conjunctivitis
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Conjunctivitis bacterial
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Coronavirus infection
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Ear infection
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Eye infection
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Gastroenteritis
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Helicobacter gastritis
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Influenza
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Lymphangitis
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Nasopharyngitis
|
15.8%
3/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Parainfluenzae virus infection
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Pharyngitis
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Pharyngitis streptococcal
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Respiratory syncytial virus infection
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Streptococcal infection
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Tonsillitis
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Upper respiratory tract infection
|
21.1%
4/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Infections and infestations
Viral myositis
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Fall
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Foot fracture
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Head injury
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Limb injury
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Skin laceration
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Injury, poisoning and procedural complications
Wrist fracture
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Investigations
Neutrophil count decreased
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
15.8%
3/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Dizziness
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Dyspraxia
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Fine motor skill dysfunction
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Headache
|
21.1%
4/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Lethargy
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Memory impairment
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Seizure
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Somnolence
|
15.8%
3/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Nervous system disorders
Tremor
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Product Issues
Device dislocation
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Aggression
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Anxiety
|
15.8%
3/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Change in sustained attention
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Emotional disorder
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Insomnia
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Irritability
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Mood altered
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Nervousness
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Perseveration
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Regressive behaviour
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Psychiatric disorders
Tic
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Renal and urinary disorders
Pollakiuria
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Skin and subcutaneous tissue disorders
Erythema
|
5.3%
1/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.5%
2/19 • Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
|
Additional Information
Neurocrine Medical Information Call Center
Neurocrine Biosciences
Phone: 877-641-3461
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place