Trial Outcomes & Findings for Safety and Efficacy of ARQ-252 Cream 0.3% in Subjects With Non-Segmental Facial Vitiligo (NCT NCT04811131)
NCT ID: NCT04811131
Last Updated: 2024-07-12
Results Overview
Achievement of F-VASI75 was declared with a ≥75% improvement from Baseline in F-VASI. F-VASI measures the % of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (none), 10% (only specks), 25% (pigmented area \> depigmented area), 50% (depigmented and pigmented areas equal), 75% (depigmented area \> pigmented area), 90% (specks of pigment), or 100% (no pigment). F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
TERMINATED
PHASE2
114 participants
Week 24
2024-07-12
Participant Flow
Participants were randomized at 31 study centers in the United States and Canada.
The study was early terminated and only available descriptive statistics are shown.
Participant milestones
| Measure |
Active IP and Active Phototherapy
Participants receive ARQ-252 cream 0.3% twice daily (BID) with phototherapy for 24 weeks.
|
Active IP and Sham Phototherapy
Participants receive ARQ-252 cream 0.3% BID with sham phototherapy for 24 weeks.
|
Vehicle and Active Phototherapy
Participants receive vehicle cream BID with phototherapy for 24 weeks.
|
Vehicle and Sham Phototherapy
Participants receive vehicle cream BID with sham phototherapy for 24 weeks.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
34
|
37
|
31
|
12
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
34
|
37
|
31
|
12
|
Reasons for withdrawal
| Measure |
Active IP and Active Phototherapy
Participants receive ARQ-252 cream 0.3% twice daily (BID) with phototherapy for 24 weeks.
|
Active IP and Sham Phototherapy
Participants receive ARQ-252 cream 0.3% BID with sham phototherapy for 24 weeks.
|
Vehicle and Active Phototherapy
Participants receive vehicle cream BID with phototherapy for 24 weeks.
|
Vehicle and Sham Phototherapy
Participants receive vehicle cream BID with sham phototherapy for 24 weeks.
|
|---|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
0
|
1
|
0
|
|
Overall Study
Lack of Efficacy
|
0
|
0
|
0
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
0
|
0
|
|
Overall Study
Sponsor request
|
31
|
35
|
27
|
10
|
|
Overall Study
Withdrawal by Subject
|
3
|
1
|
3
|
1
|
Baseline Characteristics
Safety and Efficacy of ARQ-252 Cream 0.3% in Subjects With Non-Segmental Facial Vitiligo
Baseline characteristics by cohort
| Measure |
Active IP and Active Phototherapy
n=34 Participants
Participants receive ARQ-252 cream 0.3% BID with phototherapy.
|
Active IP and Sham Phototherapy
n=36 Participants
Participants receive ARQ-252 cream 0.3% BID with sham phototherapy.
|
Vehicle and Active Phototherapy
n=30 Participants
Participants receive vehicle cream BID with phototherapy.
|
Vehicle and Sham Phototherapy
n=12 Participants
Participants receive vehicle cream BID with sham phototherapy.
|
Total
n=112 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
47.5 years
STANDARD_DEVIATION 12.75 • n=5 Participants
|
46.2 years
STANDARD_DEVIATION 10.84 • n=7 Participants
|
47.9 years
STANDARD_DEVIATION 12.90 • n=5 Participants
|
43.5 years
STANDARD_DEVIATION 7.23 • n=4 Participants
|
46.8 years
STANDARD_DEVIATION 11.65 • n=21 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
53 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
59 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
12 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
35 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
22 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
76 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
American-Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Asian
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
White
|
26 Participants
n=5 Participants
|
27 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
81 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
More than One Race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Facial Vitiligo Area Scoring Index (F-VASI) Baseline Score
|
0.743 units on a scale
STANDARD_DEVIATION 0.5970 • n=5 Participants
|
0.820 units on a scale
STANDARD_DEVIATION 0.8062 • n=7 Participants
|
0.656 units on a scale
STANDARD_DEVIATION 0.5943 • n=5 Participants
|
1.061 units on a scale
STANDARD_DEVIATION 0.6571 • n=4 Participants
|
0.779 units on a scale
STANDARD_DEVIATION 0.6780 • n=21 Participants
|
PRIMARY outcome
Timeframe: Week 24Population: No participants were analyzed due to early study termination.
Achievement of F-VASI75 was declared with a ≥75% improvement from Baseline in F-VASI. F-VASI measures the % of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (none), 10% (only specks), 25% (pigmented area \> depigmented area), 50% (depigmented and pigmented areas equal), 75% (depigmented area \> pigmented area), 90% (specks of pigment), or 100% (no pigment). F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Weeks 4, 8, 12, 16, 20, and 24Population: All randomized participants with data available are included.
The mean (SD) percentage change from baseline in F-VASI scores across study time points is presented. F-VASI measures the % of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (none), 10% (only specks), 25% (pigmented area \> depigmented area), 50% (depigmented and pigmented areas equal), 75% (depigmented area \> pigmented area), 90% (specks of pigment), or 100% (no pigment). F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Outcome measures
| Measure |
Active IP and Active Phototherapy
n=34 Participants
Participants receive ARQ-252 cream 0.3% BID with phototherapy for 24 weeks.
|
Active IP and Sham Phototherapy
n=37 Participants
Participants receive ARQ-252 cream 0.3% BID with sham phototherapy for 24 weeks.
|
Vehicle and Active Phototherapy
n=31 Participants
Participants receive vehicle cream BID with phototherapy for 24 weeks.
|
Vehicle and Sham Phototherapy
n=12 Participants
Participants receive vehicle cream BID with sham phototherapy for 24 weeks.
|
|---|---|---|---|---|
|
Percentage of Change From Baseline in F-VASI Score
Week 8
|
-57.160 percentage of change from baseline
Standard Deviation 27.3703
|
-27.917 percentage of change from baseline
Standard Deviation 23.4974
|
-39.570 percentage of change from baseline
Standard Deviation 27.5064
|
-16.667 percentage of change from baseline
Standard Deviation 23.5702
|
|
Percentage of Change From Baseline in F-VASI Score
Week 12
|
-62.963 percentage of change from baseline
Standard Deviation NA
NA due to n=1
|
-54.157 percentage of change from baseline
Standard Deviation 16.7882
|
-33.333 percentage of change from baseline
Standard Deviation NA
NA due to n=1
|
—
|
|
Percentage of Change From Baseline in F-VASI Score
Week 16
|
—
|
-10.100 percentage of change from baseline
Standard Deviation 55.6893
|
—
|
—
|
|
Percentage of Change From Baseline in F-VASI Score
Week 4
|
-21.009 percentage of change from baseline
Standard Deviation 25.0121
|
-7.775 percentage of change from baseline
Standard Deviation 11.8841
|
-11.589 percentage of change from baseline
Standard Deviation 21.5439
|
2.857 percentage of change from baseline
Standard Deviation 6.3888
|
SECONDARY outcome
Timeframe: Weeks 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The change from baseline in F-BSA affected over time. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Weeks 4, 8, 12, 16, 20, and 24.Population: No participants were analyzed due to early study termination.
The VNS is a patient-reported measure of vitiligo treatment success, which has a 5-point scale, with 1 being more noticeable and 5 being no longer noticeable. The VNS will be completed for vitiligo on the face. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Weeks 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
Change from Baseline in the VitiQoL, an instrument consisting of 16-item questionnaire (with a 7-point numerical scale from 0 - Not at all to 6 - All of the time) designed to assess disease specific health-related quality of life (HRQL) in patients suffering from vitiligo and also provide an objective measure of disease status, burden of disease, and treatment outcome. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The PaGIC-V category, a 7-point scale comparing facial vitiligo at baseline with the subject's treated facial vitiligo at the study visit (with 1- very much improved and 7 - very much worse), and time to achieve a PaGIC-V of very much improved or much improved. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The percentage of participants achieving F-VASI50. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The time to achieve F-VASI50. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The percentage of participants achieving F-VASI90. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The percentage of subjects with FsIGA, (a four point scale of vitiligo severity for the face only) of clear or almost clear (0 or 1) and time to achieve FsIGA of clear or almost clear (0 or 1). The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The time to achieve F-VASI75 50. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
FsIGA of clear or almost clear (0 or 1) plus 2-grade improvement from baseline. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4, 8, 12, 16, 20, and 24Population: No participants were analyzed due to early study termination.
The time to a PaGIC-V score of 1 'very much improved' or 2 'improved'. The PaGIC-V category, a 7-point scale comparing facial vitiligo at baseline with the subject's treated facial vitiligo at the study visit (with 1- very much improved and 7 - very much worse), and time to achieve a PaGIC-V of very much improved or much improved. The endpoint was not assessed due to early study termination.
Outcome measures
Outcome data not reported
Adverse Events
Active IP and Active Phototherapy
Active IP and Sham Phototherapy
Vehicle and Active Phototherapy
Vehicle and Sham Phototherapy
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Active IP and Active Phototherapy
n=34 participants at risk
Participants receive ARQ-252 cream 0.3% BID with phototherapy for 24 weeks.
|
Active IP and Sham Phototherapy
n=36 participants at risk
Participants receive ARQ-252 cream 0.3% BID with sham phototherapy for 24 weeks.
|
Vehicle and Active Phototherapy
n=30 participants at risk
Participants receive vehicle cream BID h phototherapy for 24 weeks.
|
Vehicle and Sham Phototherapy
n=12 participants at risk
Participants receive vehicle cream BID with sham phototherapy for 24 weeks.
|
|---|---|---|---|---|
|
General disorders
Application site pain
|
5.9%
2/34 • Up to ~22 weeks
All treated participants are included
|
2.8%
1/36 • Up to ~22 weeks
All treated participants are included
|
3.3%
1/30 • Up to ~22 weeks
All treated participants are included
|
8.3%
1/12 • Up to ~22 weeks
All treated participants are included
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/34 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/36 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/30 • Up to ~22 weeks
All treated participants are included
|
8.3%
1/12 • Up to ~22 weeks
All treated participants are included
|
|
Infections and infestations
Oral herpes
|
0.00%
0/34 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/36 • Up to ~22 weeks
All treated participants are included
|
6.7%
2/30 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/12 • Up to ~22 weeks
All treated participants are included
|
|
Injury, poisoning and procedural complications
Sunburn
|
5.9%
2/34 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/36 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/30 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/12 • Up to ~22 weeks
All treated participants are included
|
|
Injury, poisoning and procedural complications
Fibula fracture
|
0.00%
0/34 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/36 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/30 • Up to ~22 weeks
All treated participants are included
|
8.3%
1/12 • Up to ~22 weeks
All treated participants are included
|
|
Nervous system disorders
Dysguesia
|
0.00%
0/34 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/36 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/30 • Up to ~22 weeks
All treated participants are included
|
8.3%
1/12 • Up to ~22 weeks
All treated participants are included
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/34 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/36 • Up to ~22 weeks
All treated participants are included
|
0.00%
0/30 • Up to ~22 weeks
All treated participants are included
|
8.3%
1/12 • Up to ~22 weeks
All treated participants are included
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place