An Open Label Study Evaluating the Safety and Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Dermatomyositis

Stanford University1 site in 1 country5 target enrollmentFebruary 2010

ConditionsDermatomyositis

InterventionsApremilast

DrugsApremilast

Overview

Phase: Not Applicable
Intervention: Apremilast
Conditions: Dermatomyositis
Sponsor: Stanford University
Enrollment: 5
Locations: 1
Primary Endpoint: 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.
Status: Terminated
Last Updated: 11 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

February 2010

End Date

September 2011

Last Updated

11 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Stanford University

Responsible Party

Principal Investigator

David Fiorentino

Principle Investigator

Stanford University

Eligibility Criteria

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 Exclusion Criteria
•Must meet the following laboratory criteria:

Exclusion Criteria

•History of inadequate response of cutaneous DM disease to greater than 2 of the following agents: methotrexate, cyclosporine, azathioprine, mycophenolate mofetil, IVIG, leflunomide, cyclophosphamide.
•History of inadequate response to thalidomide for dermatomyositis skin disease.
•Receiving topical therapy within 14 days of Study Day 0 (including but not limited to topical corticosteroids, tacrolimus, pimecrolimus). Exceptions: low potency corticosteroids will be allowed as background therapy for treatment of the face and scalp as needed, but dose must be stable 14 days prior to Study Day 0 and throughout the study
•Concurrent therapy with prednisone (or equivalent dose of systemic corticosteroid) at greater than 10 mg daily.
•Concurrent therapy with more than one of the following agents: methotrexate, azathioprine, mycophenolate mofetil, leflunomide.
•Receiving the following dosages of medications during the study or within 28 days before Study Day 0:
•Hydroxychloroquine at \>600 mg/day
•Chloroquine at \>400 mg/day
•Methotrexate at \>25 mg/week
•Mycophenolate mofetil at \>3 g/day

Arms & Interventions

apremilast

apremilast 20mg bid

Intervention: Apremilast

Outcomes

Primary Outcomes

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

Secondary Outcomes

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.)
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)