Trial Outcomes & Findings for Safety, Tolerability, Analgesic Effect, and Feasibility of Intranasal CT001 in Pediatric Patients (NCT NCT06364072)
NCT ID: NCT06364072
Last Updated: 2026-02-02
Results Overview
Number and proportion of participants that respond to the treatment relative to baseline (i.e. reduction in pain score to 4 or below). Pain intensity was assessed with age-appropriate validated scales. Ages ≥1-\<5years: FLACC (Face, Legs, Activity, Cry, Consolability), total score 0-10 (0=no pain, 10=severe pain). Ages ≥5-\<9years: Wong-Baker FACES, categories 0,2,4,6,8,10 (range 0-10; higher=worse pain). Ages ≥9years: Numerical Rating Scale (NRS) 0-10 (0=no pain, 10=worst pain imaginable).
COMPLETED
PHASE2/PHASE3
155 participants
At baseline and at 15 and 30 min post first IMP dose
2026-02-02
Participant Flow
Total number of participants enrolled in the study: 155 (83 in Spain and 72 in the United Kingdom). There was no screen failures reported, only 1 participant was withdrawn from the study by the investigator due to pending surgical procedures which meant that the patient could not continue on the study. There were 155 participants in the full analysis set and in the safety population and 152 participants included in the efficacy analysis.
Participant milestones
| Measure |
CT001
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Overall Study
STARTED
|
155
|
|
Overall Study
COMPLETED
|
154
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
CT001
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
Safety, Tolerability, Analgesic Effect, and Feasibility of Intranasal CT001 in Pediatric Patients
Baseline characteristics by cohort
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Sex: Female, Male
Female
|
46 Participants
n=41 Participants
|
|
Sex: Female, Male
Male
|
109 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
68 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
84 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=41 Participants
|
|
Age, Customized
Age · 1 - < 5 years
|
22 Participants
n=41 Participants
|
|
Age, Customized
Age · 5 - < 9 years
|
38 Participants
n=41 Participants
|
|
Age, Customized
Age · 9 - < 18 years
|
95 Participants
n=41 Participants
|
|
Baseline pain intensity score
|
8 Score on a scale (0-10)
n=41 Participants
|
PRIMARY outcome
Timeframe: At baseline and 10, 15, 20, 30, 45 and 60 min after first IMP administration. If a second IMP dose was needed, sedation score was performed at the timepoints relative to first IMP administration.Population: Some participants had missing or non-assessable UMSS scores (e.g., early withdrawal, assessment not performed within the prespecified window, or incomplete data).
Sedation was assessed by sedation score on the University of Michigan Sedation Scale (UMSS). Scores range from 0 to 4, where 0 = awake/alert, 1 = minimally sedated (tired/sleepy, appropriate response to verbal conversation and/or sound), 2 = moderately sedated (somnolent/sleeping, easily aroused with light tactile stimulation or simple verbal command), 3 = deeply sedated (deep sleep, arousable only with significant physical stimulation), and 4 = unarousable. Lower scores indicate less sedation; higher scores indicate deeper sedation.
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Sedation
15 min
|
1 UMSS score
Interval 0.0 to 1.0
|
|
Sedation
Baseline
|
0 UMSS score
Interval 0.0 to 0.0
|
|
Sedation
10 min
|
0 UMSS score
Interval 0.0 to 1.0
|
|
Sedation
20 min
|
1 UMSS score
Interval 0.0 to 1.0
|
|
Sedation
30 min
|
1 UMSS score
Interval 0.0 to 1.0
|
|
Sedation
45 min
|
0 UMSS score
Interval 0.0 to 1.0
|
|
Sedation
60 min
|
0 UMSS score
Interval 0.0 to 1.0
|
PRIMARY outcome
Timeframe: At baseline and 10, 15, 20, 25, 30, 35, 45, 60 and 75 min after IMP administration.Population: The number analyzed differs across rows because pain intensity was assessed using age-appropriate scales, and not all participants had evaluable measurements at each time point. Some participants had missing or non-assessable pain scores (e.g., early withdrawal, assessment not performed within the prespecified window, or incomplete data), so they were excluded from the affected row(s), while remaining in the overall analyzed population.
Respiratory depression assessed by respiratory rate.
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Respiratory Depression
Age 9- <18 years, 15 min
|
20 Respiratory rate (rpm)
Standard Deviation 3.43
|
|
Respiratory Depression
Age 9- <18 years, 20 min
|
19 Respiratory rate (rpm)
Standard Deviation 3.68
|
|
Respiratory Depression
Age 9- <18 years, 25 min
|
19 Respiratory rate (rpm)
Standard Deviation 3.33
|
|
Respiratory Depression
Age 9- <18 years, 30 min
|
18 Respiratory rate (rpm)
Standard Deviation 3.13
|
|
Respiratory Depression
Age 9- <18 years, 35 min
|
16 Respiratory rate (rpm)
Standard Deviation 2.87
|
|
Respiratory Depression
Age 9- <18 years, 45 min
|
18 Respiratory rate (rpm)
Standard Deviation 3.06
|
|
Respiratory Depression
Age 9- <18 years, 60 min
|
18 Respiratory rate (rpm)
Standard Deviation 3.18
|
|
Respiratory Depression
Age 9- <18 years, 75 min
|
17 Respiratory rate (rpm)
Standard Deviation 2.26
|
|
Respiratory Depression
Age 1- <5 years, baseline
|
26 Respiratory rate (rpm)
Standard Deviation 6.09
|
|
Respiratory Depression
Age 1- <5 years, 10 min
|
24 Respiratory rate (rpm)
Standard Deviation 5.67
|
|
Respiratory Depression
Age 1- <5 years, 15 min
|
24 Respiratory rate (rpm)
Standard Deviation 5.27
|
|
Respiratory Depression
Age 1- <5 years, 20 min
|
23 Respiratory rate (rpm)
Standard Deviation 3.64
|
|
Respiratory Depression
Age 1- <5 years, 25 min
|
21 Respiratory rate (rpm)
Standard Deviation 3.44
|
|
Respiratory Depression
Age 1- <5 years, 30 min
|
22 Respiratory rate (rpm)
Standard Deviation 3.87
|
|
Respiratory Depression
Age 1- <5 years, 35 min
|
20 Respiratory rate (rpm)
Standard Deviation 4
|
|
Respiratory Depression
Age 1- <5 years, 45 min
|
22 Respiratory rate (rpm)
Standard Deviation 3.59
|
|
Respiratory Depression
Age 1- <5 years, 60 min
|
23.5 Respiratory rate (rpm)
Standard Deviation 4.24
|
|
Respiratory Depression
Age 1- <5 years, 75 min
|
24 Respiratory rate (rpm)
Standard Deviation 3.03
|
|
Respiratory Depression
Age 5- <9 years, baseline
|
24 Respiratory rate (rpm)
Standard Deviation 5.05
|
|
Respiratory Depression
Age 5- <9 years, 10 min
|
22 Respiratory rate (rpm)
Standard Deviation 4.34
|
|
Respiratory Depression
Age 5- <9 years, 15 min
|
20 Respiratory rate (rpm)
Standard Deviation 3.82
|
|
Respiratory Depression
Age 5- <9 years, 20 min
|
20 Respiratory rate (rpm)
Standard Deviation 3.28
|
|
Respiratory Depression
Age 5- <9 years, 25 min
|
21.5 Respiratory rate (rpm)
Standard Deviation 3.52
|
|
Respiratory Depression
Age 5- <9 years, 30 min
|
21 Respiratory rate (rpm)
Standard Deviation 3.48
|
|
Respiratory Depression
Age 5- <9 years, 35 min
|
23 Respiratory rate (rpm)
Standard Deviation 3.26
|
|
Respiratory Depression
Age 5- <9 years, 45 min
|
20 Respiratory rate (rpm)
Standard Deviation 3.42
|
|
Respiratory Depression
Age 5- <9 years, 60 min
|
21 Respiratory rate (rpm)
Standard Deviation 3.6
|
|
Respiratory Depression
Age 5- <9 years, 75 min
|
22 Respiratory rate (rpm)
Standard Deviation 3.87
|
|
Respiratory Depression
Age 9- <18 years, baseline
|
20 Respiratory rate (rpm)
Standard Deviation 4.31
|
|
Respiratory Depression
Age 9- <18 years, 10 min
|
20 Respiratory rate (rpm)
Standard Deviation 4.09
|
PRIMARY outcome
Timeframe: At baseline and 10, 15, 20, 25, 30, 35, 45, 60 and 75 min after IMP administration.Population: The number analyzed differs across rows because pain intensity was assessed using age-appropriate scales, and not all participants had evaluable measurements at each time point. Some participants had missing or non-assessable pain scores (e.g., early withdrawal, assessment not performed within the prespecified window, or incomplete data), so they were excluded from the affected row(s), while remaining in the overall analyzed population.
Peripheral oxygen saturation assessed by oxygen saturation rate (%)
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 35 min
|
98 Oxygen saturation rate (%)
Standard Deviation 0.89
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 45 min
|
99 Oxygen saturation rate (%)
Standard Deviation 1.4
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 60 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.39
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 75 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.64
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, baseline
|
98 Oxygen saturation rate (%)
Standard Deviation 1.32
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 10 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.5
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 15 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.42
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 20 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.28
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 25 min
|
97.5 Oxygen saturation rate (%)
Standard Deviation 1.73
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 30 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.44
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 35 min
|
97 Oxygen saturation rate (%)
Standard Deviation 1.68
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 45 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.43
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 60 min
|
98 Oxygen saturation rate (%)
Standard Deviation 2.08
|
|
Peripheral Oxygen Saturation
Age 5- <9 years, 75 min
|
98.5 Oxygen saturation rate (%)
Standard Deviation 1.9
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, baseline
|
99 Oxygen saturation rate (%)
Standard Deviation 1.35
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 10 min
|
99 Oxygen saturation rate (%)
Standard Deviation 1.54
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 15 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.52
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 20 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.55
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 25 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.5
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 30 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.61
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 35 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.46
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 45 min
|
98 Oxygen saturation rate (%)
Standard Deviation 2.07
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 60 min
|
98 Oxygen saturation rate (%)
Standard Deviation 2.22
|
|
Peripheral Oxygen Saturation
Age 9- <18 years, 75 min
|
98 Oxygen saturation rate (%)
Standard Deviation 2.23
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 25 min
|
97 Oxygen saturation rate (%)
Standard Deviation 1
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 30 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.4
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, baseline
|
98 Oxygen saturation rate (%)
Standard Deviation 1.05
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 10 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.15
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 15 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.17
|
|
Peripheral Oxygen Saturation
Age 1- <5 years, 20 min
|
98 Oxygen saturation rate (%)
Standard Deviation 1.22
|
PRIMARY outcome
Timeframe: At baseline and 10, 15, 20, 25, 30, 35, 45, 60 and 75 min after IMP administration.Population: The number analyzed differs across rows because pain intensity was assessed using age-appropriate scales, and not all participants had evaluable measurements at each time point. Some participants had missing or non-assessable pain scores (e.g., early withdrawal, assessment not performed within the prespecified window, or incomplete data), so they were excluded from the affected row(s), while remaining in the overall analyzed population.
Cardiovascular stability assessed by pulse rate (bpm)
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Cardiovascular Stability
Age 1- <5 years, baseline
|
117 Beats per minute
Standard Deviation 22.69
|
|
Cardiovascular Stability
Age 1- <5 years, 10 min
|
108 Beats per minute
Standard Deviation 17.78
|
|
Cardiovascular Stability
Age 1- <5 years, 15 min
|
111 Beats per minute
Standard Deviation 18.37
|
|
Cardiovascular Stability
Age 1- <5 years, 20 min
|
109 Beats per minute
Standard Deviation 16.7
|
|
Cardiovascular Stability
Age 1- <5 years, 25 min
|
118 Beats per minute
Standard Deviation 9.84
|
|
Cardiovascular Stability
Age 1- <5 years, 30 min
|
109 Beats per minute
Standard Deviation 16.5
|
|
Cardiovascular Stability
Age 1- <5 years, 35 min
|
117 Beats per minute
Standard Deviation 7.95
|
|
Cardiovascular Stability
Age 1- <5 years, 45 min
|
110 Beats per minute
Standard Deviation 12.91
|
|
Cardiovascular Stability
Age 1- <5 years, 60 min
|
109 Beats per minute
Standard Deviation 12.73
|
|
Cardiovascular Stability
Age 1- <5 years, 75 min
|
110 Beats per minute
Standard Deviation 9.56
|
|
Cardiovascular Stability
Age 5- <9 years, baseline
|
103.5 Beats per minute
Standard Deviation 14.92
|
|
Cardiovascular Stability
Age 5- <9 years, 10 min
|
97.5 Beats per minute
Standard Deviation 11.74
|
|
Cardiovascular Stability
Age 5- <9 years, 15 min
|
96.5 Beats per minute
Standard Deviation 13.88
|
|
Cardiovascular Stability
Age 5- <9 years, 20 min
|
93 Beats per minute
Standard Deviation 12.75
|
|
Cardiovascular Stability
Age 5- <9 years, 25 min
|
96.5 Beats per minute
Standard Deviation 12.59
|
|
Cardiovascular Stability
Age 5- <9 years, 30 min
|
95 Beats per minute
Standard Deviation 14.48
|
|
Cardiovascular Stability
Age 5- <9 years, 35 min
|
93.5 Beats per minute
Standard Deviation 15.34
|
|
Cardiovascular Stability
Age 5- <9 years, 45 min
|
91 Beats per minute
Standard Deviation 13.97
|
|
Cardiovascular Stability
Age 5- <9 years, 60 min
|
92 Beats per minute
Standard Deviation 13.07
|
|
Cardiovascular Stability
Age 5- <9 years, 75 min
|
105 Beats per minute
Standard Deviation 14.43
|
|
Cardiovascular Stability
Age 9- <18 years, baseline
|
90 Beats per minute
Standard Deviation 15.02
|
|
Cardiovascular Stability
Age 9- <18 years, 10 min
|
87 Beats per minute
Standard Deviation 15.71
|
|
Cardiovascular Stability
Age 9- <18 years, 15 min
|
85 Beats per minute
Standard Deviation 13.6
|
|
Cardiovascular Stability
Age 9- <18 years, 20 min
|
83 Beats per minute
Standard Deviation 15.17
|
|
Cardiovascular Stability
Age 9- <18 years, 25 min
|
86 Beats per minute
Standard Deviation 13.49
|
|
Cardiovascular Stability
Age 9- <18 years, 30 min
|
80 Beats per minute
Standard Deviation 15.09
|
|
Cardiovascular Stability
Age 9- <18 years, 35 min
|
83 Beats per minute
Standard Deviation 14.46
|
|
Cardiovascular Stability
Age 9- <18 years, 45 min
|
83 Beats per minute
Standard Deviation 15.11
|
|
Cardiovascular Stability
Age 9- <18 years, 60 min
|
82 Beats per minute
Standard Deviation 14.77
|
|
Cardiovascular Stability
Age 9- <18 years, 75 min
|
80 Beats per minute
Standard Deviation 14.36
|
PRIMARY outcome
Timeframe: Through study completion; up to 7 daysNumber of reported adverse events.
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Number of Reported Adverse Events
|
132 Adverse events
|
PRIMARY outcome
Timeframe: Through study completion; up to 7 daysNumber of adverse events (AEs) reported per participant.
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Number of Adverse Events (AEs) Reported Per Participant
|
0.85 Adverse event per participant
|
PRIMARY outcome
Timeframe: 30 and 60 min post IMP administrationThe number of participants with nasal irritation was summarised using counts of participants for each timepoint (30 and 60 min post IMP administration) by type of nasal irritation.
Outcome measures
| Measure |
CT001
n=154 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Local Nasal Irritation
Itching, 60 min post IMP
|
7 Number of participants
|
|
Local Nasal Irritation
Nasal discharge, 30 min post IMP
|
0 Number of participants
|
|
Local Nasal Irritation
Nasal discharge, 60 min post IMP
|
1 Number of participants
|
|
Local Nasal Irritation
Other nasal irritation, 30 min post IMP
|
3 Number of participants
|
|
Local Nasal Irritation
Other nasal irritation, 60 min post IMP
|
1 Number of participants
|
|
Local Nasal Irritation
Redness, 30 min post IMP
|
2 Number of participants
|
|
Local Nasal Irritation
Redness, 60 min post IMP
|
1 Number of participants
|
|
Local Nasal Irritation
Sneezing, 30 min post IMP
|
1 Number of participants
|
|
Local Nasal Irritation
Sneezing, 60 min post IMP
|
0 Number of participants
|
|
Local Nasal Irritation
Itching, 30 min post IMP
|
16 Number of participants
|
PRIMARY outcome
Timeframe: At baseline and at 15 and 30 min post first IMP dosePopulation: As the results include two time points, some participants are represented in more than one category.
Number and proportion of participants that respond to the treatment relative to baseline (i.e. reduction in pain score to 4 or below). Pain intensity was assessed with age-appropriate validated scales. Ages ≥1-\<5years: FLACC (Face, Legs, Activity, Cry, Consolability), total score 0-10 (0=no pain, 10=severe pain). Ages ≥5-\<9years: Wong-Baker FACES, categories 0,2,4,6,8,10 (range 0-10; higher=worse pain). Ages ≥9years: Numerical Rating Scale (NRS) 0-10 (0=no pain, 10=worst pain imaginable).
Outcome measures
| Measure |
CT001
n=152 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Analgesic Effect
Participants with pain intensity <=4 at 15min post first dose of CT001
|
82 Participants
|
|
Analgesic Effect
Participants with pain intensity > 4 at 15min post first dose of CT001
|
70 Participants
|
|
Analgesic Effect
Participants with pain intensity <=4 at 30min post first dose of CT001
|
134 Participants
|
|
Analgesic Effect
Participants with pain intensity > 4 at 30min post first dose of CT001
|
18 Participants
|
SECONDARY outcome
Timeframe: Prior to Emergency Department discharge (day of IMP administration)Treatment satisfaction as assessed by responses to the question: "How satisfied are you with the study drug that you/your child received? Please think about how it helped their pain, how it was given, any side effects, and how quickly you/your child recovered". Respondents answered using a 5-point Likert scale (very unsatisfied (1), unsatisfied (2), neutral (3), satisfied (4), very satisfied (5)).
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Treatment Satisfaction
|
5 Treatment satistaction score
Interval 5.0 to 5.0
|
SECONDARY outcome
Timeframe: Prior to Emergency Department discharge (day of IMP administration)Feasibility (i.e. acceptance of nasal administration) was addressed by the healthcare staff asking the participant: "If you were in this situation again and needed pain medication, would you like to receive the nasal spray (relative to an injection, tablet or suppository for the pain)?' If not possible by the participant, the parent/legal guardian assessed nasal acceptability. Answers were "yes, "no", "I don't know".
Outcome measures
| Measure |
CT001
n=153 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Feasibility (Acceptance of Nasal Administration)
"Yes"
|
143 Participants
|
|
Feasibility (Acceptance of Nasal Administration)
"No"
|
4 Participants
|
|
Feasibility (Acceptance of Nasal Administration)
"I don't know"
|
6 Participants
|
SECONDARY outcome
Timeframe: Assessed immediately post IMP administrationMedication errors, defined as any deviation in the IMP administration instructions that resulted in higher or lower dose than planned. Examples may include erroneous priming of the pump, too few/many pumps administered, etc.
Outcome measures
| Measure |
CT001
n=155 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Medication Errors
Reported number of participants with medication errors
|
3 Participants
|
|
Medication Errors
Number of participants with no medication errors
|
152 Participants
|
SECONDARY outcome
Timeframe: 30 min post (first) IMP administrationPain intensity was assessed with age-appropriate validated scales. Ages ≥1-\<5years: FLACC (Face, Legs, Activity, Cry, Consolability), total score 0-10 (0=no pain, 10=severe pain). Ages ≥5-\<9years: Wong-Baker FACES, categories 0,2,4,6,8,10 (range 0-10; higher=worse pain). Ages ≥9years: Numerical Rating Scale (NRS) 0-10 (0=no pain, 10=worst pain imaginable).
Outcome measures
| Measure |
CT001
n=152 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Maximum Change From Baseline in Pain Intensity Within 30 Min Post (First) IMP Administration.
|
-6 Scores on a scale
Interval -8.0 to -4.0
|
SECONDARY outcome
Timeframe: within 30 min post (first) IMP administration.Pain intensity was assessed with age-appropriate validated scales. Ages ≥1-\<5years: FLACC (Face, Legs, Activity, Cry, Consolability), total score 0-10 (0=no pain, 10=severe pain). Ages ≥5-\<9years: Wong-Baker FACES, categories 0,2,4,6,8,10 (range 0-10; higher=worse pain). Ages ≥9years: Numerical Rating Scale (NRS) 0-10 (0=no pain, 10=worst pain imaginable).
Outcome measures
| Measure |
CT001
n=152 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Number of Participants That Achieved a 30% (or More) Reduction in Pain Intensity Relative to Baseline
|
145 Participants
|
SECONDARY outcome
Timeframe: at 10, 15, 20, 30, 45 and 60 min post last dose of IMP administration.Population: Only patients who had evaluable pain-intensity measurements for the relevant endpoint/time point are included in this outcome measure. Although 155 patients were treated and included in the overall safety population, some did not have a valid post-baseline assessment (e.g., missing measurement, assessment outside the prespecified window, early discontinuation, or other clinical/technical reasons).
Pain intensity was assessed with age-appropriate validated scales. Ages ≥1-\<5years: FLACC (Face, Legs, Activity, Cry, Consolability), total score 0-10 (0=no pain, 10=severe pain). Ages ≥5-\<9years: Wong-Baker FACES, categories 0,2,4,6,8,10 (range 0-10; higher=worse pain). Ages ≥9years: Numerical Rating Scale (NRS) 0-10 (0=no pain, 10=worst pain imaginable).
Outcome measures
| Measure |
CT001
n=152 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Change From Baseline in Pain Intensity
30 min
|
-6 Scores on a scale
Interval -8.0 to -4.0
|
|
Change From Baseline in Pain Intensity
10 min
|
-2 Scores on a scale
Interval -4.0 to -1.0
|
|
Change From Baseline in Pain Intensity
15 min
|
-4 Scores on a scale
Interval -6.0 to -3.0
|
|
Change From Baseline in Pain Intensity
20 min (1 dose)
|
-5 Scores on a scale
Interval -7.0 to -4.0
|
|
Change From Baseline in Pain Intensity
25 min (2 doses)
|
-4 Scores on a scale
Interval -6.0 to -3.0
|
|
Change From Baseline in Pain Intensity
35 min (2 doses)
|
-6 Scores on a scale
Interval -8.0 to -5.0
|
|
Change From Baseline in Pain Intensity
45 min
|
-6 Scores on a scale
Interval -8.0 to -5.0
|
|
Change From Baseline in Pain Intensity
60 min
|
-6 Scores on a scale
Interval -8.0 to -5.0
|
|
Change From Baseline in Pain Intensity
75 min (2 doses)
|
-7 Scores on a scale
Interval -8.0 to -5.0
|
SECONDARY outcome
Timeframe: During the 60 min period post last dose of IMPNumber of children receiving additional analgesics.
Outcome measures
| Measure |
CT001
n=139 Participants
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Number of Children Receiving Additional Analgesics
|
17 participants
|
Adverse Events
CT001
Serious adverse events
| Measure |
CT001
n=155 participants at risk
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Surgical and medical procedures
Hospitalisation
|
2.6%
4/155 • Through study completion; up to 7 days
|
Other adverse events
| Measure |
CT001
n=155 participants at risk
All participants received one or potentially two consecutive doses of CT001.
|
|---|---|
|
Eye disorders
Lacrimation increased
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Eye disorders
Vision blurred
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Gastrointestinal disorders
Diarrhoea
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Gastrointestinal disorders
Nausea
|
9.0%
14/155 • Through study completion; up to 7 days
|
|
Gastrointestinal disorders
Vomiting
|
16.1%
25/155 • Through study completion; up to 7 days
|
|
Injury, poisoning and procedural complications
Product use complaint
|
12.3%
19/155 • Through study completion; up to 7 days
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Nervous system disorders
Dizziness
|
14.2%
22/155 • Through study completion; up to 7 days
|
|
Nervous system disorders
Headache
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Nervous system disorders
Hypoaesthesia
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Nervous system disorders
Paraesthesia
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Psychiatric disorders
Abnormal behaviour
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Psychiatric disorders
Anger
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Psychiatric disorders
Confusional state
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Psychiatric disorders
Depressed mood
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Psychiatric disorders
Euphoric mood
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Psychiatric disorders
Mood altered
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Respiratory, thoracic and mediastinal disorders
Nasal discomfort
|
1.3%
2/155 • Through study completion; up to 7 days
|
|
Respiratory, thoracic and mediastinal disorders
Nasal pruritus
|
8.4%
13/155 • Through study completion; up to 7 days
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
2.6%
4/155 • Through study completion; up to 7 days
|
|
Skin and subcutaneous tissue disorders
Erythema
|
1.3%
2/155 • Through study completion; up to 7 days
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.65%
1/155 • Through study completion; up to 7 days
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.5%
10/155 • Through study completion; up to 7 days
|
|
Surgical and medical procedures
Hospitalisation
|
2.6%
4/155 • Through study completion; up to 7 days
|
|
Vascular disorders
Hot flush
|
0.65%
1/155 • Through study completion; up to 7 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place