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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01140503
Recruitment Status : Terminated (Slow recruitment of participants)
First Posted : June 9, 2010
Results First Posted : March 4, 2015
Last Update Posted : March 4, 2015
Sponsor:
Information provided by (Responsible Party):
David Fiorentino, Stanford University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Dermatomyositis
Intervention Drug: Apremilast
Enrollment 5
Recruitment Details 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.
Pre-assignment Details  
Arm/Group Title Apremilast
Hide Arm/Group Description All subjects received apremilast 20mg PO BID
Period Title: Overall Study
Started 5
Completed 4
Not Completed 1
Reason Not Completed
Withdrawal by Subject             1
Arm/Group Title Apremilast
Hide Arm/Group Description All subjects received apremilast 20mg PO BID
Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
<=18 years
0
   0.0%
Between 18 and 65 years
5
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
5
 100.0%
Male
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
5
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Physician Global Assessment of Skin Activity (VAS)   [1] 
Median (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants
3.8  (0.98)
[1]
Measure Description: This is a visual analog scale (0-10cm) in which the physician estimates the global activity of the dermatomyositis skin disease. The score ranges from 0 to 10, with higher scores representing higher disease severity
Physician Global Assessment of Skin Activity (Likert)   [1] 
Median (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants
2  (0)
[1]
Measure Description: This is a 5 point scale (0=clear; 1=mild; 2=moderate; 3=severe; 4=very severe) in which the physician estimates the severity of the dermatomyositis skin disease activity
CDASI Activity Score   [1] 
Median (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants
31  (6.8)
[1]
Measure Description: This is a validated instrument (Cutaneous Dermatomyositis Activity and Severity Index) quantifying dermatomyositis skin disease activity by assigning severity points on redness, scale, and ulceration to specific body sites typically involved by dermatomyositis. Possible scores for the CDASI-a range from 0 to 100, with higher numbers represent higher severity of skin disease. Scores above 15 are considered to have moderate-to-severe activity. A clinically meaningful improvement in this scale is 4.
Manual Muscle Testing (MMT-8) score   [1] 
Median (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants
147  (4.21)
[1]
Measure Description: This is a validated measure of muscle strength. Scale goes from 0-150. 150 is perfect strength.
Patient Pruritis Scale (VAS)   [1] 
Median (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants
2.9  (2.40)
[1]
Measure Description: This is a patient-reported estimate of itching. The patient fills out a visual analog scale (0-10cm) to estimate the severity of itching. A higher score is more severe itch.
Dermatology Life Quality Index (DLQI)   [1] 
Median (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants
9  (3.89)
[1]
Measure Description: This is a validated instrument that estimates the impact of skin disease on the quality of life. score ranges from 0-30, with a higher score representing a greater adverse impact on quality of life.
1.Primary Outcome
Title 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.
Hide Description [Not Specified]
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Apremilast
Hide Arm/Group Description:
All subjects received apremilast 20mg PO BID
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: adverse events
12
2.Secondary Outcome
Title 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.
Hide Description This was an intent to treat analysis--dropouts are considered treatment failures. Missing data at 12 weeks imputed by last observation carried forward.
Time Frame Data collected at 12 weeks after baseline visit.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Apremilast
Hide Arm/Group Description:
All subjects received apremilast 20mg PO BID
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
1
3.Secondary Outcome
Title The Secondary Outcome Measure Will be Efficacy as Measured by the Mean Change in CDASI-activity at 12 Weeks
Hide Description 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.
Time Frame Data collected at baseline at 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Apremilast
Hide Arm/Group Description:
All subjects received apremilast 20mg PO BID
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
6  (4)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Apremilast
Hide Arm/Group Description All subjects received apremilast 20mg PO BID
All-Cause Mortality
Apremilast
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Apremilast
Affected / at Risk (%) # Events
Total   0/5 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Apremilast
Affected / at Risk (%) # Events
Total   5/5 (100.00%)    
Gastrointestinal disorders   
Indigestion *  1/5 (20.00%)  1
Nausea *  1/5 (20.00%)  1
Infections and infestations   
Urinary Tract Infection *  1/5 (20.00%)  1
Upper respiratory tract infection *  1/5 (20.00%)  1
Musculoskeletal and connective tissue disorders   
Myalgia *  1/5 (20.00%)  1
Back Pain *  1/5 (20.00%)  1
Left Knee Injury *  1/5 (20.00%)  1
Nervous system disorders   
headache *  2/5 (40.00%)  2
Migraine headache *  1/5 (20.00%)  1
Dizziness *  1/5 (20.00%) 
Renal and urinary disorders   
Elevated serum creatinine *  1/5 (20.00%)  1
Skin and subcutaneous tissue disorders   
Bruising *  1/5 (20.00%)  1
*
Indicates events were collected by non-systematic assessment
Study was terminated early due to low enrollment. Therefore we only have data on 5 participants.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: David Fiorentino
Organization: Stanford University Department of Dermatology
Phone: 650-721-7160
EMail: fiorentino@stanford.edu
Layout table for additonal information
Responsible Party: David Fiorentino, Stanford University
ClinicalTrials.gov Identifier: NCT01140503    
Other Study ID Numbers: SU-03302010-5522
16975
First Submitted: April 6, 2010
First Posted: June 9, 2010
Results First Submitted: January 28, 2015
Results First Posted: March 4, 2015
Last Update Posted: March 4, 2015