Trial Outcomes & Findings for Open Label Pilot Study of Apremilast in Treatment of Rosacea (NCT NCT01045551)

NCT ID: NCT01045551

Last Updated: 2016-12-13

Results Overview

Papule and pustule count consisted of direct measurement of the number of papules/pustules on the face. Papule and pustule count, compared between baseline and end of treatment Week 12 was calculated

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: Baseline to Week 12
• Results posted on: 2016-12-13

Participant Flow

10 patients recruited

Participant milestones

Measure	Apremilast 20 mg (Twice Per Day) All subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Open Label Pilot Study of Apremilast in Treatment of Rosacea

Baseline characteristics by cohort

Measure	Apremilast 20 mg (Twice Per Day) n=10 Participants All subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Gender Female	7 Participants n=5 Participants
Gender Male	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 12

Papule and pustule count consisted of direct measurement of the number of papules/pustules on the face. Papule and pustule count, compared between baseline and end of treatment Week 12 was calculated

Outcome measures

Measure	Open Label Apremilast 20 mg (Twice Per Day) n=10 Participants This is an open label study, therefore all subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Change From Baseline in the Total Number of Papulopustular Lesions at Week 12	-0.2 papule count Standard Deviation 25.77165

SECONDARY outcome

Timeframe: Baseline, week 12

The Physician Global 7-point Assessment. Scale range: 0-7. 0 = clear, 1 = minimal, 2 = mild, 3 = mild to moderate, 4= moderate, 5= moderate to severe, 6 = severe. 0 is a better outcome, 6 is a worse outcome. No subscales were used.

Outcome measures

Measure	Open Label Apremilast 20 mg (Twice Per Day) n=10 Participants This is an open label study, therefore all subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Change in Physician 7 Point Global Assessment From Baseline to Week 12	-0.90000 units on a scale Standard Deviation 0.99443

SECONDARY outcome

Timeframe: Baseline, Week 12

The change in the Physician Overall Erythema Severity. Scale range 0 - 3. 0 = none/absent, 1 = mild, 2 = moderate, 3 = severe. 0 is considered a better outcome, 3 is considered a worse outcome.

Outcome measures

Measure	Open Label Apremilast 20 mg (Twice Per Day) n=10 Participants This is an open label study, therefore all subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Change From Baseline in Erythema Rating Visit 8 (Week 12)	-0.7000 units on a scale Standard Deviation 0.48305

SECONDARY outcome

Timeframe: Baseline, Week 12

physician count of telangiectasias on the face at visit 1 (baseline) compared to at visit 8 (week 12)

Outcome measures

Measure	Open Label Apremilast 20 mg (Twice Per Day) n=10 Participants This is an open label study, therefore all subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Change From Baseline in Telangiectasia Count at Visit 8 (Week 12)	0 telangiectasia count Standard Error 0

SECONDARY outcome

Timeframe: Week 12, Week 16

Outcome measures

Measure	Open Label Apremilast 20 mg (Twice Per Day) n=10 Participants This is an open label study, therefore all subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks
Change From Visit 8 (Week 12) in Telangiectasia Count at Visit 9 (Week 16)	0.2000 telangiectasia count Standard Error 0.63246

Adverse Events

Apremilast 20 mg (Twice Per Day)

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Apremilast 20 mg (Twice Per Day) n=10 participants at risk All subjects will receive Apremilast 20mg taken orally twice per day. Apremilast: 20mg taken orally twice per day for 12 weeks The most frequently reported adverse event (AE) was infection. One patient (a 74 year old female) experienced 2 urinary tract infections during the course of the study, and another (a 63 year old female) experienced one urinary tract infection. Two patients experienced upper respiratory infections, which have previously been reported in patients taking apremilast. The second most common AE was loose stool, reported by two patients, which resolved quickly and without recurrence. All AE's were reported as mild and no patient withdrew from the study due to AEs. No patient required dosing modification or discontinuation.
Renal and urinary disorders urinary tract infection	20.0% 2/10 • Number of events 3 • Adverse event data was collected for approximately 4 years. Adverse event determination was achieved by regular solicitation subjects at study visits, from investigator assessment, and regular laboratory testing. In addition self-reporting by participants was encouraged.
Respiratory, thoracic and mediastinal disorders upper respiratory infection	20.0% 2/10 • Number of events 2 • Adverse event data was collected for approximately 4 years. Adverse event determination was achieved by regular solicitation subjects at study visits, from investigator assessment, and regular laboratory testing. In addition self-reporting by participants was encouraged.
Gastrointestinal disorders loose stool	20.0% 2/10 • Number of events 2 • Adverse event data was collected for approximately 4 years. Adverse event determination was achieved by regular solicitation subjects at study visits, from investigator assessment, and regular laboratory testing. In addition self-reporting by participants was encouraged.

Additional Information

Julian Mackay-Wiggan, MD, MS

Columbia University Medical Center

Phone: 212-305-6953

Email: jc299@cumc.columbia.edu

Results disclosure agreements

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