Trial Outcomes & Findings for Repeat Dose Safety Study of NRL-1 in Epilepsy Subjects (NCT NCT02721069)
NCT ID: NCT02721069
Last Updated: 2022-01-31
Results Overview
Assess the number of participants who received at least one dose of NRL-1 (Valtoco) during the study and comprised the safety population.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
175 participants
Primary outcome timeframe
12 months
Results posted on
2022-01-31
Participant Flow
Participant milestones
| Measure |
NRL-1
Intranasal dose of NRL-1 will be administered at either 5 mg, 10 mg, 15 mg, or 20 mg based on the subject's body weight.
NRL-1
|
|---|---|
|
Overall Study
STARTED
|
175
|
|
Overall Study
COMPLETED
|
117
|
|
Overall Study
NOT COMPLETED
|
58
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Repeat Dose Safety Study of NRL-1 in Epilepsy Subjects
Baseline characteristics by cohort
| Measure |
NRL-1
n=175 Participants
Intranasal dose of NRL-1 will be administered at either 5 mg, 10 mg, 15 mg, or 20 mg based on the subject's body weight.
NRL-1
|
|---|---|
|
Age, Categorical
<=18 years
|
92 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
82 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
22.3 years
STANDARD_DEVIATION 14.87 • n=5 Participants
|
|
Sex: Female, Male
Female
|
93 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
82 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
143 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
175 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Subjects who received at least one dose of NRL-1.
Assess the number of participants who received at least one dose of NRL-1 (Valtoco) during the study and comprised the safety population.
Outcome measures
| Measure |
NRL-1 5mg
n=11 Participants
Intranasal dose of NRL-1 will be administered at 5 mg based on age and weight.
|
NRL-1 10mg
n=61 Participants
Intranasal dose of NRL-1 will be administered at 10 mg based on age and weight.
|
NRL-1 15 mg
n=46 Participants
Intranasal dose of NRL-1 will be administered at 15 mg based on age and weight.
|
NRL-1 20mg
n=57 Participants
Intranasal dose of NRL-1 will be administered at 20 mg based on age and weight.
|
|---|---|---|---|---|
|
Number of Participants Who Received NRL-1 (Valtoco)
|
9 Participants
|
54 Participants
|
46 Participants
|
54 Participants
|
Adverse Events
NRL-1 5mg
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
NRL-1 10mg
Serious events: 20 serious events
Other events: 34 other events
Deaths: 0 deaths
NRL-1 15mg
Serious events: 15 serious events
Other events: 24 other events
Deaths: 0 deaths
NRL-1 20mg
Serious events: 13 serious events
Other events: 21 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
NRL-1 5mg
n=9 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
NRL-1 10mg
n=54 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
NRL-1 15mg
n=46 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
NRL-1 20mg
n=54 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
|---|---|---|---|---|
|
Nervous system disorders
Seizure
|
11.1%
1/9 • Number of events 1 • 12 months
|
20.4%
11/54 • Number of events 16 • 12 months
|
17.4%
8/46 • Number of events 14 • 12 months
|
7.4%
4/54 • Number of events 5 • 12 months
|
|
Nervous system disorders
Status Epilepticus
|
0.00%
0/9 • 12 months
|
9.3%
5/54 • Number of events 7 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
1.9%
1/54 • Number of events 2 • 12 months
|
|
Nervous system disorders
Epilepsy
|
0.00%
0/9 • 12 months
|
3.7%
2/54 • Number of events 3 • 12 months
|
2.2%
1/46 • Number of events 2 • 12 months
|
3.7%
2/54 • Number of events 3 • 12 months
|
|
Nervous system disorders
Seizure Cluster
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
3.7%
2/54 • Number of events 4 • 12 months
|
|
Nervous system disorders
Amnesia
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Disturbance in Attention
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Lennox-Gastaut Syndrome
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Lethargy
|
11.1%
1/9 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Muscle Spasticity
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Partial Seizures
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Petit Mal Epilepsy
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Nervous system disorders
Postictal State
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Gastrointestinal disorders
Pancreatitis
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/9 • 12 months
|
3.7%
2/54 • Number of events 3 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Gastrointestinal disorders
Large Intestinal Ulcer
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Gastrointestinal disorders
Proctalgia
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Injury, poisoning and procedural complications
Hip Fracture
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Injury, poisoning and procedural complications
Joint Dislocation
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Injury, poisoning and procedural complications
Thermal Burn
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Injury, poisoning and procedural complications
Wound Dehiscence
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Injury, poisoning and procedural complications
Wrist Fracture
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
General disorders
Pyrexia
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
General disorders
Chest Pain
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
General disorders
Sudden Unexplained Death in Epilepsy
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia Aspiration
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Failure
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Restrictive Pulmonary Disease
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Chronic Myeloid Leukaemia
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colorectal Adenocarcinoma
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leiomyoma
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Psychiatric disorders
Depression
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Psychiatric disorders
Hallucination, Visual
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Psychiatric disorders
Major Depression
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Psychiatric disorders
Suicidal Ideation
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Metabolism and nutrition disorders
Feeding Disorder
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/9 • 12 months
|
3.7%
2/54 • Number of events 2 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Reproductive system and breast disorders
Menorrhagia
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Reproductive system and breast disorders
Uterine Haemorrhage
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
0.00%
0/46 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Cardiac disorders
Mitral Valve Prolapse
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Surgical and medical procedures
Brain Lobectomy
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Infections and infestations
Pneumonia
|
0.00%
0/9 • 12 months
|
7.4%
4/54 • Number of events 4 • 12 months
|
2.2%
1/46 • Number of events 3 • 12 months
|
3.7%
2/54 • Number of events 2 • 12 months
|
|
Infections and infestations
Abscess
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Infections and infestations
Appendicitis Perforated
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Infections and infestations
Colonic Abscess
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
0.00%
0/54 • 12 months
|
|
Infections and infestations
Influenza
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
|
Infections and infestations
Pharyngitis Streptococcal
|
0.00%
0/9 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
0.00%
0/46 • 12 months
|
0.00%
0/54 • 12 months
|
Other adverse events
| Measure |
NRL-1 5mg
n=9 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
NRL-1 10mg
n=54 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
NRL-1 15mg
n=46 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
NRL-1 20mg
n=54 participants at risk
Intranasal dose of NRL-1 will be administered at 5, 10, 15, and 20 mg based on age and weight.
|
|---|---|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
11.1%
1/9 • Number of events 3 • 12 months
|
22.2%
12/54 • Number of events 13 • 12 months
|
6.5%
3/46 • Number of events 3 • 12 months
|
7.4%
4/54 • Number of events 6 • 12 months
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
0.00%
0/9 • 12 months
|
16.7%
9/54 • Number of events 10 • 12 months
|
15.2%
7/46 • Number of events 8 • 12 months
|
7.4%
4/54 • Number of events 4 • 12 months
|
|
Infections and infestations
Influenza
|
33.3%
3/9 • Number of events 4 • 12 months
|
9.3%
5/54 • Number of events 5 • 12 months
|
6.5%
3/46 • Number of events 3 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
|
Infections and infestations
Urinary Tract Infection
|
11.1%
1/9 • Number of events 2 • 12 months
|
7.4%
4/54 • Number of events 8 • 12 months
|
6.5%
3/46 • Number of events 3 • 12 months
|
5.6%
3/54 • Number of events 4 • 12 months
|
|
Nervous system disorders
Seizure
|
0.00%
0/9 • 12 months
|
7.4%
4/54 • Number of events 5 • 12 months
|
15.2%
7/46 • Number of events 8 • 12 months
|
3.7%
2/54 • Number of events 4 • 12 months
|
|
Nervous system disorders
Somnolence
|
0.00%
0/9 • 12 months
|
9.3%
5/54 • Number of events 5 • 12 months
|
8.7%
4/46 • Number of events 4 • 12 months
|
3.7%
2/54 • Number of events 2 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Discomfort
|
0.00%
0/9 • 12 months
|
0.00%
0/54 • 12 months
|
8.7%
4/46 • Number of events 11 • 12 months
|
11.1%
6/54 • Number of events 16 • 12 months
|
|
Gastrointestinal disorders
Diarrhoea
|
22.2%
2/9 • Number of events 2 • 12 months
|
7.4%
4/54 • Number of events 4 • 12 months
|
2.2%
1/46 • Number of events 1 • 12 months
|
3.7%
2/54 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Number of events 1 • 12 months
|
7.4%
4/54 • Number of events 5 • 12 months
|
4.3%
2/46 • Number of events 2 • 12 months
|
3.7%
2/54 • Number of events 4 • 12 months
|
|
General disorders
Pyrexia
|
11.1%
1/9 • Number of events 1 • 12 months
|
18.5%
10/54 • Number of events 10 • 12 months
|
6.5%
3/46 • Number of events 3 • 12 months
|
1.9%
1/54 • Number of events 1 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place