Trial Outcomes & Findings for Pilot Study Evaluating the Efficacy of a Topical PDE4 Inhibitor for Morphea (NCT NCT03351114)

NCT ID: NCT03351114

Last Updated: 2021-04-01

Results Overview

Change in dermal thickness on skin biopsy. A sentinel plaque will be selected at baseline. A 4 mm skin punch biopsy will be performed at baseline and at 12 weeks, on the same affected plaque.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 8 participants
• Primary outcome timeframe: Baseline, 12 weeks
• Results posted on: 2021-04-01

Participant Flow

Participant milestones

Measure	Crisaborole 2% Ointment Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Overall Study STARTED	8
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Crisaborole 2% Ointment Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Pilot Study Evaluating the Efficacy of a Topical PDE4 Inhibitor for Morphea

Baseline characteristics by cohort

Measure	Crisaborole 2% Ointment n=8 Participants Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Age, Continuous	51 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Change in dermal thickness on skin biopsy. A sentinel plaque will be selected at baseline. A 4 mm skin punch biopsy will be performed at baseline and at 12 weeks, on the same affected plaque.

Outcome measures

Measure	Crisaborole 2% Ointment n=7 Participants Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Number of Participants Who Had a Reduction in Dermal Thickness of Sentinel Plaque	5 Participants

SECONDARY outcome

Timeframe: Baseline and 12 weeks

The DIET (Dyspigmentation, Induration, Erythema, and Telangiectasias) score is a physician-assessed, previously validated score. Range is 0-12, and a higher score denotes increased activity or severity.

Outcome measures

Measure	Crisaborole 2% Ointment n=7 Participants Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Percentage of Reduction in DIET Score of Sentinel Plaque	33 percentage of reduction Standard Deviation 26

SECONDARY outcome

Timeframe: Baseline,12 weeks

LoSCAT (Localized Scleroderma Cutaneous Assessment Tool ) score is a physician-assessed, previously validated score. It has 2 components, the LoSSI (localized scleroderma skin severity index, range 0-162), which measures disease activity, and LoSDI (localized scleroderma damage index, range 0-216). The higher the number, the higher the disease activity and damage, respectively.

Outcome measures

Measure	Crisaborole 2% Ointment n=7 Participants Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Percentage of Reduction in LoSCAT Score LoSSI (Activity)	37.5 percentage of reduction Standard Deviation 29
Percentage of Reduction in LoSCAT Score LoSDI (Damage)	5 percentage of reduction Standard Deviation 36

SECONDARY outcome

Timeframe: Baseline and 12 weeks

Skindex-29 is a health-related quality of life score; minimum score 30, maximum 150. Lower score indicates a better quality of life.

Outcome measures

Measure	Crisaborole 2% Ointment n=7 Participants Crisaborole 2% ointment applied to affected skin twice per day. Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day.
Percentage of Reduction in Skindex-29 Score	25.7 percentage of reduction Standard Deviation 16.4

SECONDARY outcome

Timeframe: Baseline, 4, 8 and 12 weeks, optional at 20 weeks

Population: Data not collected.

B-Mode ultrasonographic imaging will be utilized to measure dermal thickness of the sentinel plaque

Outcome measures

Outcome data not reported

Adverse Events

Crisaborole 2% Ointment

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Adela R. Cardones

Duke University

Phone: 9196843110

Email: adela.cardones@duke.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place