Trial Outcomes & Findings for A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis (NCT NCT02864082)
NCT ID: NCT02864082
Last Updated: 2021-09-29
Results Overview
The number of participants with AEs will be assessed by the investigator and the incidence (severity and causality) of any local and systemic AEs will be reported.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Day 0 through Day 57 (Weeks 0-8)
Results posted on
2021-09-29
Participant Flow
Recruitment: March 8, 2017-February 13, 2018. Recruited at 5 clinics in United States associated with US Universities by practicing dermatologists
Each patient received both vehicle application and either PAT-001 0.1% or PAT-001 0.2% on 2 different identical matching parts of their bodies (e.g, upper thighs). Since each of the 19 patients were treated with both PAT-001 and vehicle, the number of treatment areas was 38 (19 x 2) but the number of patients was 19.
Participant milestones
| Measure |
Group 1 PAT-001 0.1% and Vehicle
Part 1 (8 weeks): Patients will have two comparable Treatment Areas on their bodies, for example both upper arms that have same degree of disease. PAT-001, 0.1% will be applied to one of those body parts for 8 weeks, applied twice a day. The matching body part will have vehicle applied twice a day for 8 weeks
Part 2 (4 weeks): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1% so that subjects cannot tell the difference between vehicle and PAT-001 based upon appearance.
|
Group 2 PAT-001 0.2% and Vehicle
Part 1 (8 weeks): Patients will have two comparable Treatment Areas on their bodies, for example both upper arms that have same degree of disease. PAT-001, 0.2% will be applied to one of those body parts for 8 weeks, applied twice a day. The matching body part will have vehicle applied twice a day for 8 weeks.
Part 2 (4 weeks): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1% so that subjects cannot tell the difference between vehicle and PAT-001 based upon appearance.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
9
|
|
Overall Study
COMPLETED
|
7
|
5
|
|
Overall Study
NOT COMPLETED
|
3
|
4
|
Reasons for withdrawal
| Measure |
Group 1 PAT-001 0.1% and Vehicle
Part 1 (8 weeks): Patients will have two comparable Treatment Areas on their bodies, for example both upper arms that have same degree of disease. PAT-001, 0.1% will be applied to one of those body parts for 8 weeks, applied twice a day. The matching body part will have vehicle applied twice a day for 8 weeks
Part 2 (4 weeks): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1% so that subjects cannot tell the difference between vehicle and PAT-001 based upon appearance.
|
Group 2 PAT-001 0.2% and Vehicle
Part 1 (8 weeks): Patients will have two comparable Treatment Areas on their bodies, for example both upper arms that have same degree of disease. PAT-001, 0.2% will be applied to one of those body parts for 8 weeks, applied twice a day. The matching body part will have vehicle applied twice a day for 8 weeks.
Part 2 (4 weeks): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1% so that subjects cannot tell the difference between vehicle and PAT-001 based upon appearance.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
2
|
Baseline Characteristics
A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
Baseline characteristics by cohort
| Measure |
Group 1 PAT-001 0.1% and Vehicle
n=10 Participants
Part 1 (weeks 0-8): Bilateral comparison. Patients will have two comparable Treatment Areas: PAT-001, 0.1% (e.g., left side) and Vehicle, 0.0% (e.g., right side).
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Group 2 PAT-001 0.2% and Vehicle
n=9 Participants
Part 1 (weeks 0-8): Bilateral comparison. Patients will have two comparable Treatment Areas: PAT-001, 0.2% (e.g., left side) and Vehicle, 0.0% (e.g., right side).
Part 2: (weeks 8-12) Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
37.5 years
STANDARD_DEVIATION 20.3 • n=5 Participants
|
47.2 years
STANDARD_DEVIATION 18.4 • n=7 Participants
|
42 years
STANDARD_DEVIATION 19.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 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
|
10 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
19 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
|
|
Region of Enrollment
United States
|
10 participants
n=5 Participants
|
9 participants
n=7 Participants
|
19 participants
n=5 Participants
|
|
BMI
|
28.8 kg/m^2
n=5 Participants
|
26.4 kg/m^2
n=7 Participants
|
27.7 kg/m^2
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 0 through Day 57 (Weeks 0-8)Population: Number of Participants with Adverse events
The number of participants with AEs will be assessed by the investigator and the incidence (severity and causality) of any local and systemic AEs will be reported.
Outcome measures
| Measure |
Group 1 PAT-001 0.1%
n=10 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.1% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (Weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.2% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
n=10 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Group 2 Vehicle
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
|---|---|---|---|---|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Subjects with TEAE
|
7 participants
|
7 participants
|
5 participants
|
5 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Treatment Emergent Adverse Events (TEAE) within treatment area
|
4 participants
|
5 participants
|
3 participants
|
3 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Deaths due to TEAE
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Serious TEAE
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients who Discontinued in the trial due to TEAE
|
1 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients with Severe TEAE
|
1 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients with Moderate TEAE
|
3 participants
|
3 participants
|
2 participants
|
2 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients with Mild TEAE
|
4 participants
|
6 participants
|
3 participants
|
3 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients Where the TEAE was Considered Definitely Related to Treatment
|
1 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients where the TEAE was Considered Probably Related to Treatment
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients Where the TEAE was Considered Possibly Related to Treatment
|
1 participants
|
4 participants
|
1 participants
|
1 participants
|
|
Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)
Number of Patients Where the TEAE was Considered Not Related to Treatment
|
1 participants
|
5 participants
|
4 participants
|
4 participants
|
PRIMARY outcome
Timeframe: Up to Day 84 (Weeks 0-12)Population: Participants may have had more than one type of LSR
LSRs including burning/stinging, pain, and pruritus (itch) will be assessed in each Treatment Area using a four-point ordinal scale where 0=none, 1=mild, 2=moderate, and 3=severe (based on the investigator's evaluation of the skin reaction) at each clinic visit to allow a comparison between Treatment Groups and Test Articles. Only LSRs that require medical intervention (e.g., prescription medication) or require withholding or reduction in dosing frequency of the test articles will be documented in this LSR Table. Any LSRs that are not listed here will be recorded as AEs.
Outcome measures
| Measure |
Group 1 PAT-001 0.1%
n=10 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.1% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (Weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=10 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.2% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Group 2 Vehicle
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
|---|---|---|---|---|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site pruritis (severe)
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site irritation (mild)
|
3 participants
|
2 participants
|
0 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site dermatitis (moderate)
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site rash (mild)
|
1 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site reaction (mild)
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site pain (moderate)
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site Pruritis (moderate)
|
1 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site Pruritis (mild)
|
1 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle
Application site Dermatitis (mild)
|
0 participants
|
0 participants
|
1 participants
|
2 participants
|
SECONDARY outcome
Timeframe: Up to Day 57Overall severity of ichthyosis will be graded using a five-point scale Investigator Global Assessment (IGA) based upon a 5 point scale going from 0=clear., 1=almost clear, 2=mild, 3=moderate to 4=severe. Scoring is based upon investigator evaluation. This is a static morphological scale that refers to a point in time and not a comparison to Baseline.
Outcome measures
| Measure |
Group 1 PAT-001 0.1%
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.1% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (Weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=8 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.2% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Group 2 Vehicle
n=8 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
|---|---|---|---|---|
|
Number of Participants Achieving Improvement to State of Clear, Almost Clear or Mild in the Investigator's Global Assessment (IGA) Using a Five-point Scale at Day 57 (Part 1)
|
9 Participants
|
8 Participants
|
6 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Up to Day 57 (Weeks 0-8)Population: IGA score improvement of at least 1 point on IGA for scaling, erythema, papulation/lichenification and fissuring at Day 57 in patients receiving continuous 0.1% and 0.2% PAT-001
Overall severity of erythema (redness), scaling , fissuring (cracks in skin), and papulation/lichenification (skin thickening, increased pigmentation and/or exaggerated skin lines, formation of papules) will be graded using a five-point scale from 0=clear, 1=almost clear, 2=mild, 3=moderate to 4=severe. This is a static morphological scale that refers to a point in time and not a comparison to Baseline. This scoring is based upon investigator discretion.
Outcome measures
| Measure |
Group 1 PAT-001 0.1%
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.1% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (Weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=8 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.2% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
n=9 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Group 2 Vehicle
n=8 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
|---|---|---|---|---|
|
Number of Participants Achieving an Improvement of at Least 1 Point Score in the Individual Clinical Signs/Symptoms of Erythema, Scaling, Fissuring and Papulation/Lichenification Using a Five-point Scale
Scaling
|
9 participants
|
6 participants
|
7 participants
|
8 participants
|
|
Number of Participants Achieving an Improvement of at Least 1 Point Score in the Individual Clinical Signs/Symptoms of Erythema, Scaling, Fissuring and Papulation/Lichenification Using a Five-point Scale
Fissuring
|
6 participants
|
6 participants
|
5 participants
|
6 participants
|
|
Number of Participants Achieving an Improvement of at Least 1 Point Score in the Individual Clinical Signs/Symptoms of Erythema, Scaling, Fissuring and Papulation/Lichenification Using a Five-point Scale
Papulation/lichenification
|
9 participants
|
6 participants
|
6 participants
|
7 participants
|
|
Number of Participants Achieving an Improvement of at Least 1 Point Score in the Individual Clinical Signs/Symptoms of Erythema, Scaling, Fissuring and Papulation/Lichenification Using a Five-point Scale
Erythema
|
5 participants
|
5 participants
|
4 participants
|
6 participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 (0,1, 2, 3, and 4 hours post Dose)Serum concentrations for PAT-001 0.1% and PAT-001 0.2% looking at blood levels obtained at timepoints outlined
Outcome measures
| Measure |
Group 1 PAT-001 0.1%
n=2 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.1% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (Weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=3 Participants
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: PAT-001, 0.2% will be applied to one of the 2 areas twice a day for 8 weeks
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
Group 2 Vehicle
Part 1 (Weeks 0-8): Patients will have two comparable Treatment Areas of equal size and degree of severity: Vehicle will be applied to one of the 2 areas twice a day for 8 weeks
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
|
|---|---|---|---|---|
|
Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints
Pre-Dose
|
1.495 ng/mL
Standard Deviation 0.6718
|
2.48 ng/mL
Standard Deviation 1.795
|
—
|
—
|
|
Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints
1 Hour
|
1.5 ng/mL
Standard Deviation 0.6505
|
2.227 ng/mL
Standard Deviation 1.3725
|
—
|
—
|
|
Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints
2 Hours
|
1.425 ng/mL
Standard Deviation 0.5303
|
2.12 ng/mL
Standard Deviation 1.2257
|
—
|
—
|
|
Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints
3 Hours
|
1.37 ng/mL
Standard Deviation 0.297
|
2.063 ng/mL
Standard Deviation 1.5332
|
—
|
—
|
|
Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints
4 Hours
|
1.375 ng/mL
Standard Deviation 0.0778
|
1.453 ng/mL
Standard Deviation 0.3625
|
—
|
—
|
Adverse Events
Group 1 PAT-001 0.1%
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Group 2 PAT-001 0.2%
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Group 1 Vehicle
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Group 2 Vehicle
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group 1 PAT-001 0.1%
n=10 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: PAT-001, 0.1% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=9 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: PAT-001, 0.2% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
n=10 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: Vehicle, 0.0% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 Vehicle
n=9 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: Vehicle 0.0% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
|---|---|---|---|---|
|
Infections and infestations
C. difficile colitis
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Infections and infestations
Novavirus gastroenteritis
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Psychiatric disorders
mental status changes
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
Other adverse events
| Measure |
Group 1 PAT-001 0.1%
n=10 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: PAT-001, 0.1% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 PAT-001 0.2%
n=9 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: PAT-001, 0.2% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
Group 1 Vehicle
n=10 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: Vehicle, 0.0% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
|
Group 2 Vehicle
n=9 participants at risk
Part 1 (weeks 0-8) Patients will have two comparable identical Treatment Areas: Vehicle 0.0% will be applied to one of these areas.
Part 2 (weeks 8-12): Patients will apply PAT-001, 0.2% to both Treatment Areas.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
|
|---|---|---|---|---|
|
General disorders
Application site pruritis
|
30.0%
3/10 • Number of events 6 • 84 Days
|
0.00%
0/9 • 84 Days
|
20.0%
2/10 • Number of events 2 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
|
Infections and infestations
C. difficile colitis
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
20.0%
2/10 • Number of events 2 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Investigations
Blood triglycerides increased
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Skin and subcutaneous tissue disorders
Application site dermatitis
|
20.0%
2/10 • Number of events 2 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
1/10 • Number of events 1 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Infections and infestations
Application site folliculitis
|
10.0%
1/10 • Number of events 1 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Infections and infestations
Gastroenteritis novovirus
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Infections and infestations
Tooth infection
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
0.00%
0/10 • 84 Days
|
22.2%
2/9 • Number of events 2 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Infections and infestations
Urinary Tract Infection
|
10.0%
1/10 • Number of events 1 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
General disorders
Application site pain
|
10.0%
1/10 • Number of events 1 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Injury, poisoning and procedural complications
Procedural dizziness
|
10.0%
1/10 • Number of events 1 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Investigations
Transaminases increased
|
10.0%
1/10 • Number of events 1 • 84 Days
|
0.00%
0/9 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
0.00%
0/10 • 84 Days
|
11.1%
1/9 • Number of events 1 • 84 Days
|
0.00%
0/10 • 84 Days
|
0.00%
0/9 • 84 Days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place