Trial Outcomes & Findings for Study Examining PrEP-001 in Healthy Subjects (NCT NCT03220048)
NCT ID: NCT03220048
Last Updated: 2019-11-12
Results Overview
Area Under the Curve (AUC) of total symptom scores (upper respiratory tract (URT), lower respiratory tract (LRT) and systemic viral symptoms (SVS)). Total symptom scores (from the symptom diary card) were used to calculate the AUC. The time unit used was minutes. Thus, the AUC unit is the total symptom score multiplied by the time period from first to last assessment in minutes (i.e score\*mins). The minimum AUC value would be 0, for a subject who did not report any symptoms. The maximum AUC value is not provided as it would be theoretical only, with no real meaning in terms of severity. Higher scores indicate worse outcome than lower scores.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
66 participants
Primary outcome timeframe
8 days
Results posted on
2019-11-12
Participant Flow
Participant milestones
| Measure |
Cohort A: Sentinel Group
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
|
Cohort B: PrEP-001
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
|
Cohort B: Placebo
Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
|
|---|---|---|---|
|
Overall Study
STARTED
|
11
|
27
|
28
|
|
Overall Study
COMPLETED
|
11
|
25
|
27
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study Examining PrEP-001 in Healthy Subjects
Baseline characteristics by cohort
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
|
Cohort B: PrEP-001
n=27 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
|
Cohort B: Placebo
n=28 Participants
Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
|
Total
n=66 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=39 Participants
|
27 Participants
n=41 Participants
|
28 Participants
n=35 Participants
|
66 Participants
n=31 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=39 Participants
|
8 Participants
n=41 Participants
|
11 Participants
n=35 Participants
|
23 Participants
n=31 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=39 Participants
|
19 Participants
n=41 Participants
|
17 Participants
n=35 Participants
|
43 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
White British
|
10 Participants
n=39 Participants
|
23 Participants
n=41 Participants
|
19 Participants
n=35 Participants
|
52 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
White Irish
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
White Other
|
0 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
3 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
Mixed - White & Black Caribbean
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
Mixed - White & Asian
|
0 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
Asian or Asian British - Pakistani
|
1 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
3 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
Asian - Other
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
Black or Black British - African
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Region of Enrollment
United Kingdom
|
11 participants
n=39 Participants
|
27 participants
n=41 Participants
|
28 participants
n=35 Participants
|
66 participants
n=31 Participants
|
PRIMARY outcome
Timeframe: 8 daysArea Under the Curve (AUC) of total symptom scores (upper respiratory tract (URT), lower respiratory tract (LRT) and systemic viral symptoms (SVS)). Total symptom scores (from the symptom diary card) were used to calculate the AUC. The time unit used was minutes. Thus, the AUC unit is the total symptom score multiplied by the time period from first to last assessment in minutes (i.e score\*mins). The minimum AUC value would be 0, for a subject who did not report any symptoms. The maximum AUC value is not provided as it would be theoretical only, with no real meaning in terms of severity. Higher scores indicate worse outcome than lower scores.
Outcome measures
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
Placebo Comparator
|
Cohort B: PrEP-001
n=25 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, or Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
PrEP-001
|
Cohort B: Placebo
n=27 Participants
Placebo Comparator
|
|---|---|---|---|
|
Primary Efficacy Endpoint: The Area Under the Curve (AUC) of Total Symptom Score From Day 1 (Post Viral Challenge) to Day 8 (Quarantine Discharge).
|
17956.5 score*mins
Standard Deviation 23780.83
|
4502.6 score*mins
Standard Deviation 6144.84
|
9859.6 score*mins
Standard Deviation 12215.84
|
SECONDARY outcome
Timeframe: 8 daysUsing the scheduled protocol assessments from Day 1 to Day 8, this endpoint represented the highest total symptom score (defined as the sum of all 10 individual composite symptoms). The minimum value, for subjects who had no symptoms, would be 0. The maximum value would be 30. Higher scores indicate worse outcome than lower scores.
Outcome measures
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
Placebo Comparator
|
Cohort B: PrEP-001
n=25 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, or Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
PrEP-001
|
Cohort B: Placebo
n=27 Participants
Placebo Comparator
|
|---|---|---|---|
|
Secondary Efficacy Endpoint: Symptom Scores: Peak Symptoms Score
|
5.2 Score
Standard Deviation 5.88
|
1.7 Score
Standard Deviation 1.9
|
3.3 Score
Standard Deviation 3.28
|
SECONDARY outcome
Timeframe: 8 daysThe number of subjects with viral shedding. Viral shedding was measured by PCR, testing the nasopharyngeal swab samples.
Outcome measures
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
Placebo Comparator
|
Cohort B: PrEP-001
n=25 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, or Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
PrEP-001
|
Cohort B: Placebo
n=27 Participants
Placebo Comparator
|
|---|---|---|---|
|
Secondary Efficacy Endpoint: Incidence(s) of Illness and Infection: Viral Shedding
|
7 Participants
|
13 Participants
|
21 Participants
|
SECONDARY outcome
Timeframe: 8 daysThe number of subjects with seroconversion. Seroconversion was measured by the ratio of Influenza A/Perth/16/2009 (H3N2) virus antibodies at follow-up versus pre-dose.
Outcome measures
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
Placebo Comparator
|
Cohort B: PrEP-001
n=25 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, or Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
PrEP-001
|
Cohort B: Placebo
n=27 Participants
Placebo Comparator
|
|---|---|---|---|
|
Secondary Efficacy Endpoint: Incidence(s) of Illness and Infection: Seroconversion
|
9 Participants
|
12 Participants
|
16 Participants
|
SECONDARY outcome
Timeframe: 8 daysViral load data was supplied in Log10 Copies/mL. These values were used to calculate the Area Under the Curve (AUC) of Viral Load for each subject.
Outcome measures
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
Placebo Comparator
|
Cohort B: PrEP-001
n=25 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, or Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
PrEP-001
|
Cohort B: Placebo
n=27 Participants
Placebo Comparator
|
|---|---|---|---|
|
Secondary Efficacy Endpoint: Viral Load Parameters: Area Under the Curve (AUC) of Viral Load, as Measured by Nasopharyngeal Swab RT-qPCR.
|
33021.8 mins*log10 copy number/mL
Standard Deviation 19676.07
|
23781.6 mins*log10 copy number/mL
Standard Deviation 14127.65
|
32944.4 mins*log10 copy number/mL
Standard Deviation 17059.07
|
SECONDARY outcome
Timeframe: 8 daysTotal weight of nasal discharge (in grams) was calculated as the sum of mucus weights taken from Day 1 (Post viral challenge) to Day 8 (Quarantine Discharge).
Outcome measures
| Measure |
Cohort A: Sentinel Group
n=11 Participants
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
Placebo Comparator
|
Cohort B: PrEP-001
n=25 Participants
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, or Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
PrEP-001
|
Cohort B: Placebo
n=27 Participants
Placebo Comparator
|
|---|---|---|---|
|
Secondary Efficacy Endpoint: Total Weight of Nasal Discharge Produced Post Viral Challenge to Quarantine Discharge
|
26.580 grams
Standard Deviation 49.5158
|
3.959 grams
Standard Deviation 5.1349
|
13.414 grams
Standard Deviation 20.7986
|
Adverse Events
Cohort A: Sentinel Group
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Cohort B: PrEP-001
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Cohort B: Placebo
Serious events: 0 serious events
Other events: 21 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort A: Sentinel Group
n=11 participants at risk
Sentinel group in which subjects received a challenge virus inoculum volume of 100uL on Day 0.
|
Cohort B: PrEP-001
n=27 participants at risk
PrEP-001 6400μg dose administered equally over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
|
Cohort B: Placebo
n=28 participants at risk
Nasal dose of placebo Comparator equally divided over both nostrils and on 2 consecutive days, using a single dose nasal powder device, as per randomisation schedule.
|
|---|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
54.5%
6/11
|
7.4%
2/27
|
21.4%
6/28
|
|
Investigations
Aspartate aminotransferase increased
|
36.4%
4/11
|
11.1%
3/27
|
14.3%
4/28
|
|
Investigations
Blood cholesterol increased
|
18.2%
2/11
|
7.4%
2/27
|
14.3%
4/28
|
|
Investigations
Activated partial thromboplastin time prolonged
|
0.00%
0/11
|
7.4%
2/27
|
14.3%
4/28
|
|
Investigations
Low density lipoprotein increased
|
18.2%
2/11
|
0.00%
0/27
|
10.7%
3/28
|
|
Investigations
C-reactive protein increased
|
18.2%
2/11
|
3.7%
1/27
|
3.6%
1/28
|
|
Investigations
Lipase increased
|
9.1%
1/11
|
7.4%
2/27
|
3.6%
1/28
|
|
Investigations
Blood fibrinogen increased
|
0.00%
0/11
|
3.7%
1/27
|
7.1%
2/28
|
|
Investigations
Blood triglycerides increased
|
0.00%
0/11
|
3.7%
1/27
|
7.1%
2/28
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/11
|
3.7%
1/27
|
7.1%
2/28
|
|
Investigations
White blood cell count increased
|
0.00%
0/11
|
3.7%
1/27
|
3.6%
1/28
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
27.3%
3/11
|
22.2%
6/27
|
32.1%
9/28
|
|
Injury, poisoning and procedural complications
Procedural haemorrhage
|
18.2%
2/11
|
22.2%
6/27
|
32.1%
9/28
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
0.00%
0/11
|
7.4%
2/27
|
0.00%
0/28
|
|
Nervous system disorders
Nervous system disorders
|
36.4%
4/11
|
3.7%
1/27
|
3.6%
1/28
|
|
Nervous system disorders
Headache
|
27.3%
3/11
|
3.7%
1/27
|
3.6%
1/28
|
|
Infections and infestations
Infections and infestations
|
9.1%
1/11
|
7.4%
2/27
|
7.1%
2/28
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/11
|
7.4%
2/27
|
3.6%
1/28
|
|
General disorders
General disorders and administration site conditions
|
9.1%
1/11
|
7.4%
2/27
|
3.6%
1/28
|
|
General disorders
Pyrexia
|
0.00%
0/11
|
7.4%
2/27
|
0.00%
0/28
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place