Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

Plain-language summary

This study evaluates the safety and effect of the drug Apremilast in treating skin symptoms in patients with recalcitrant (treatment-resistant) dermatomyositis. It enrolled patients over 18 with a diagnosis of dermatomyositis whose skin disease persisted despite current treatments. The study was conducted in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Tulane University

Intervention

Apremilast 30mg

Start date

6/12/2018

Estimated completion

4/7/2021

Countries

United States

Related genes

Locations

• New Orleans, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Must understand the risks and the benefits/purpose of the study and provide signed and dated informed consent.
* Must be 18 years at time of signing the informed consent form.
* Willing to participate in all required evaluations and procedures in the study including the ability to swallow pills without difficulty.
* Patients must have a diagnosis of DM based upon the characteristic cutaneous findings proposed by Sontheimer\[6\] and/or a skin biopsy consistent with DM.
* Patients must be candidate for systemic therapy for their DM skin disease defined by inadequate response to aggressive sun protection along with the use of potent topical corticosteroids and/or immunomodulators.
* Patients with a diagnosis of dermatomyositis on steroid-sparing agent and/or systemic steroids (maximum dose of prednisone 1mg/Kg) and still having cutaneous disease activity of at least 5 on the CDASI scale.
* If on immunosuppressive treatments and/or steroids, patients must be on stable doses for at least 4 weeks (28 days).
* Patients must undergo age appropriate cancer screening.
* Females of childbearing potential (FCBP) must have a negative pregnancy test at screening (day 0 of the study and every month throughout the study). While on investigational product and for at least 28 days after taking the last dose of investigational product.

Exclusion Criteria:

* Increasing or changing dose of topical therapy within 14 days of study day 0 (including but not limited to topical corticosteroids, tacrolimus, pimecrolimus).
* Increasing or changing systemic steroids dosing within 28 days of study day 0.
* Increasing or changing dosing for concurrent therapy agents within 28 days or 5 half-lives of the biologic agent, whichever is longer, before study day 0: methotrexate, azathioprine, mycophenolate mofetil, hydroxychloroquine, dapsone, leflunomide, cyclosporine, biologic agents (anti-TNFs), IVIG, rituximab.
* History of any clinically significant (as determined by the investigators) cardiac, endocrinologic, pulmonary, neurologic, psychiatric, hepatic, renal, hematologic, immunologic, or other major uncontrolled disease.
* Any condition, including the presence of laboratory abnormalities, which places the patient at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
* Pregnant or breastfeeding.
* Untreated Latent Mycobacterium tuberculosis infection or active tuberculosis infection as indicated by a positive Purified Protein Derivative (PPD) skin test or T-spot.
* Any condition, including the presence of laboratory abnormalities that places the patient at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
* Patients with acute dermatomyositis onset and rapid progression of muscle disease or significant systemic involvement including pulmonary diseases associated with DM.
* Prior major surgery or major life-threatening medical illness within 2 weeks.
* Inflammatory bowel disease, malabsorption or any other gastrointestinal motility disorders that limit the absorption of the study drug.
* Active hepatitis B or C infection with detectible viral nucleic acid in the blood or known Human Immunodeficiency Virus (HIV) positivity.
* Prior history of suicide attempt at any time in the patient's lifetime prior to screening or randomization, or major psychiatric illness requiring hospitalization within the last 3 years.
* Active substance abuse or a history of substance abuse within 6 months prior to screening.
* Use of any investigational drug within 4 weeks prior to randomization, or 5 pharmacokinetic/pharmacodynamic half-lives, if known (whichever is longer).
* Prior treatment with apremilast.
* Any severe systemic illness requiring IV antibiotics within the two weeks prior to initiation of the study drug.
* Malignancy or history of malignancy within the past four years, except for:

  * treated \[ie, cured\] basal cell or squamous cell in situ skin carcinomas;
  * treated \[ie, cured\] cervical intraepithelial neoplasia (CIN) or carcinoma in situ of cervix with no evidence of recurrence within the previous 4 years.