A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis
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ClinicalTrials.gov Identifier: NCT01140503
Recruitment Status :
(Slow recruitment of participants)
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 Outcome Measures :
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. [ Time Frame: 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 [ Time Frame: 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.
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Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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:
Hemoglobin ≥ 12 g/dL
White blood cell (WBC) count ≥ 3000 /uL (≥ 3.0 X 10^9/L) and < 14,000/uL (< 14 X 10^9/L)
Platelets ≥ 100,000 /uL (≥ 100 X 10^9/L)
Aspartate transaminase (AST [serum glutamic oxaloacetic transaminase, SGOT]) and alanine transaminase (ALT [serum glutamate pyruvic transaminase, SGPT]) ; 1.5x upper limit of normal (ULN) unless, in the opinion, of the investigator, the elevation is secondary to active muscle inflammation.
Females of childbearing potential (FCBP) must have a negative urine pregnancy test at screening (Visit 1). In addition, sexually active FCBP must agree to use TWO of the following adequate forms of contraception while on study medication: oral, injectable, or implantable hormonal contraceptives; tubal ligation; intrauterine device; barrier contraceptive with spermicide; or vasectomized partner. A FCBP must agree to have pregnancy tests every 28 days while on study medication.
Males (including those who have had a vasectomy) must agree to use barrier contraception (latex condoms) when engaging in sexual activity with FCBP while on study medication and for 84 days after taking the last dose of study medication
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
Azathioprine at >3 mg/kg/day
Leflunomide at >20mg/day
Treatment with the following biologic agents:
Adalimumab, etanercept, efalizumab, or infliximab within 12 weeks of Study Day 0 and for the study duration
IVIG within 12 weeks of Study Day 0 and for the study duration
Rituximab within 9 months of Study Day 0 and for the study duration
Alefacept within 24 weeks of Study Day 0 and for the study duration
Have received fluctuating doses of any of the following medications 28 days before Study Day 0: methotrexate, mycophenolate mofetil, azathioprine, leflunomide, dapsone
Have received fluctuating doses of hydroxychloroquine 2 months before Study Day 0
Have received fluctuating doses of chloroquine 3 months before Study Day 0
Have received fluctuating doses of prednisone within 14 days prior to Study Day 0
Received leflunomide >20 mg/day in the 6 months prior to Study Day 0
Treatment with any investigational drug therapy within 28 days before Study Day 0 or biologic therapies within 30 days or 5 half-lives of the biologic agent, whichever is longer, before Study Day 0
Currently receiving any of the following medications:
Intravenous immunoglobulin (IVIG)
Any TNF inhibitor, including adalimumab, etanercept, infliximab, or certolizumab
Oral FK506 (tacrolimus)
History of any clinically significant (as determined by the investigator) 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 subject 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
Latent Mycobacterium tuberculosis infection as indicated by a positive Purified Protein Derivative (PPD) skin test.
History of active Mycobacterium tuberculosis infection (any subspecies) within 3 years prior to the screening visit and without documentation of successful treatment
* Subjects who completed treatment at least 3 years prior to screening but lack documentation may not be enrolled in the study. Subjects who completed treatment at least 3 years prior to screening are allowed if successful treatment was completed at least 3 years prior to screening and is documented and available for verification
History of incompletely treated latent Mycobacterium tuberculosis infection as indicated by:
Subject's medical records documenting incomplete treatment for Mycobacterium tuberculosis
Subject's self reported history of incomplete treatment for Mycobacterium tuberculosis
Any clinically significant abnormality on 12-lead ECG at screening
Presence of hepatitis B surface antigens (HBsAg) or Hepatitis B core antibody positive at screening
Antibodies to hepatitis C virus at screening
History of human immunodeficiency virus (HIV) infection
Malignancy or history of malignancy except for treated (i.e. cured) basal-cell skin carcinomas
Abnormal chest x-ray findings other than that consistent with dermatomyositis-associated interstitial lung disease. Chest x-rays performed within 3 months prior to start of study drug are acceptable
Any condition, including the presence of laboratory abnormalities, that places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study