Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis (NCT NCT01140503)

NCT ID: NCT01140503

Last Updated: 2015-03-04

Results Overview

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 5 participants
• Primary outcome timeframe: 16 weeks
• Results posted on: 2015-03-04

Participant Flow

This is a single center study in which patients were recruited from a clinical practice. Participant recruitment lasted from December, 2009 until August, 2011. The first patient was screened in February, 2010 and the last patient visit was in July, 2011.

Participant milestones

Measure	Apremilast All subjects received apremilast 20mg PO BID
Overall Study STARTED	5
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Apremilast All subjects received apremilast 20mg PO BID
Overall Study Withdrawal by Subject	1

Baseline Characteristics

A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis

Baseline characteristics by cohort

Measure	Apremilast n=5 Participants All subjects received apremilast 20mg PO BID
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	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	5 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
Physician Global Assessment of Skin Activity (VAS)	3.8 units on a scale STANDARD_DEVIATION 0.98 • n=5 Participants
Physician Global Assessment of Skin Activity (Likert)	2 units on a scale STANDARD_DEVIATION 0 • n=5 Participants
CDASI Activity Score	31 units on a scale STANDARD_DEVIATION 6.8 • n=5 Participants
Manual Muscle Testing (MMT-8) score	147 units on a scale STANDARD_DEVIATION 4.21 • n=5 Participants
Patient Pruritis Scale (VAS)	2.9 units on a scale STANDARD_DEVIATION 2.40 • n=5 Participants
Dermatology Life Quality Index (DLQI)	9 units on a scale STANDARD_DEVIATION 3.89 • n=5 Participants

PRIMARY outcome

Timeframe: 16 weeks

Outcome measures

Measure	Apremilast n=5 Participants All subjects received apremilast 20mg PO BID
The Primary Endpoint Analysis Will be Safety, as Measured by the Number of Adverse Events and Serious Adverse Events Occuring During 12 Weeks of Therapy and 4 Weeks of Followup.	12 adverse events

SECONDARY outcome

Timeframe: Data collected at 12 weeks after baseline visit.

This was an intent to treat analysis--dropouts are considered treatment failures. Missing data at 12 weeks imputed by last observation carried forward.

Outcome measures

Measure	Apremilast n=5 Participants All subjects received apremilast 20mg PO BID
The Secondary Outcome Measure Will be Efficacy, as Measured by the Number of Participants Experiencing a 30% Decreased in the CDASI-a Score at 12 Weeks.	1 participants

SECONDARY outcome

Timeframe: Data collected at baseline at 12 weeks

The CDASI (Cutaneous Dermatomyositis Activity and Severity Index) is a validated instrument to measure skin disease activity in dermatomyositis. A clinically meaningful change is a decrease of 4 points. All missing data are imputed using last observation carried forward. Calculation is performed as the score at 12 weeks minus the score at baseline.

Outcome measures

Measure	Apremilast n=5 Participants All subjects received apremilast 20mg PO BID
The Secondary Outcome Measure Will be Efficacy as Measured by the Mean Change in CDASI-activity at 12 Weeks	6 units on a scale Standard Deviation 4

Adverse Events

Apremilast

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Apremilast n=5 participants at risk All subjects received apremilast 20mg PO BID
Nervous system disorders headache	40.0% 2/5 • Number of events 2
Musculoskeletal and connective tissue disorders Myalgia	20.0% 1/5 • Number of events 1
Infections and infestations Urinary Tract Infection	20.0% 1/5 • Number of events 1
Skin and subcutaneous tissue disorders Bruising	20.0% 1/5 • Number of events 1
Gastrointestinal disorders Indigestion	20.0% 1/5 • Number of events 1
Musculoskeletal and connective tissue disorders Back Pain	20.0% 1/5 • Number of events 1
Nervous system disorders Migraine headache	20.0% 1/5 • Number of events 1
Infections and infestations Upper respiratory tract infection	20.0% 1/5 • Number of events 1
Musculoskeletal and connective tissue disorders Left Knee Injury	20.0% 1/5 • Number of events 1
Nervous system disorders Dizziness	20.0% 1/5
Gastrointestinal disorders Nausea	20.0% 1/5 • Number of events 1
Renal and urinary disorders Elevated serum creatinine	20.0% 1/5 • Number of events 1

Additional Information

David Fiorentino

Stanford University Department of Dermatology

Phone: 650-721-7160

Email: fiorentino@stanford.edu

Results disclosure agreements

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

Restriction type: LTE60