Dermatomyositis
Conditions
Brief summary
This study is designed to evaluate the safety and efficacy of an oral medicine (called apremilast) for treating skin involvement in patients with the disease dermatomyositis.
Interventions
DRUGApremilast
Apremilast 20mg PO BID
Sponsors
Stanford University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Must understand and voluntarily sign an informed consent form * Must be 18 years at time of signing informed consent form * Must be able to adhere to the study visit schedule and other protocol requirements * Patients must have a diagnosis of DM based upon the characteristic cutaneous findings proposed by Sontheimer1 and a skin biopsy consistent with DM * Subjects must be a candidate for systemic therapy for their DM skin disease: a subject is considered a candidate, if, in the judgment of the investigator, they are not adequately responding to aggressive sun protection along with the use of potent (e.g. class I or II) topical corticosteroids and/or immunomodulators * Must have cutaneous disease activity of at least moderate on a 5 point Likert scale (using the PGA) * Must have cutaneous disease activity score of at least 5 on the CDASI (activity) scale * Concurrent therapy with topical corticosteroids and/or prednisone and/or antimalarials is permitted as defined in
Exclusion criteria
. * Concurrent therapy with methotrexate azathioprine, mycophenolate mofetil, or leflunomide is permitted as defined in
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. | 16 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. | 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. |
| The Secondary Outcome Measure Will be Efficacy as Measured by the Mean Change in CDASI-activity at 12 Weeks | 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. |
Countries
United States
Participant flow
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.
Participants by arm
| Arm | Count |
|---|---|
| Apremilast All subjects received apremilast 20mg PO BID | 5 |
| Total | 5 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Apremilast |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| CDASI Activity Score | 31 units on a scale STANDARD_DEVIATION 6.8 |
| Dermatology Life Quality Index (DLQI) | 9 units on a scale STANDARD_DEVIATION 3.89 |
| Manual Muscle Testing (MMT-8) score | 147 units on a scale STANDARD_DEVIATION 4.21 |
| Patient Pruritis Scale (VAS) | 2.9 units on a scale STANDARD_DEVIATION 2.4 |
| Physician Global Assessment of Skin Activity (Likert) | 2 units on a scale STANDARD_DEVIATION 0 |
| Physician Global Assessment of Skin Activity (VAS) | 3.8 units on a scale STANDARD_DEVIATION 0.98 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 5 Participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 5 / 5 |
| serious Total, serious adverse events | 0 / 5 |
Outcome results
Primary
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.
Time frame: 16 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Apremilast | 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
The Secondary Outcome Measure Will be Efficacy as Measured by the Mean Change in CDASI-activity 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.
Time frame: Data collected at baseline at 12 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Apremilast | 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 |
Secondary
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.
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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Apremilast | 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 |