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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 October 2017
Main ID:  NCT01140503
Date of registration: 06/04/2010
Prospective Registration: Yes
Primary sponsor: Stanford University
Public title: A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis
Scientific title: An Open Label Study Evaluating the Safety and Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Dermatomyositis
Date of first enrolment: February 2010
Target sample size: 5
Recruitment status: Terminated
URL:  https://clinicaltrials.gov/show/NCT01140503
Study type:  Interventional
Study design:   
Phase:  N/A
Countries of recruitment
United States
Contacts
Name:     David Franklin Fiorentino
Address: 
Telephone:
Email:
Affiliation:  Stanford University
Name:     Katharine Arefiev
Address: 
Telephone:
Email:
Affiliation:  Stanford University
Key inclusion & exclusion 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:

- 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)

- Serum creatinine ≤ 1.5 mg/dL (≤ 132.6 μmol/L)

- Total bilirubin ≤ 2.0 mg/dL

- 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

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

- 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:

- Cyclophosphamide

- Intravenous immunoglobulin (IVIG)

- Any TNF inhibitor, including adalimumab, etanercept, infliximab, or certolizumab

- Rituximab

- Efalizumab

- Cyclosporine

- Oral FK506 (tacrolimus)

- Thalidomide

- 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 y



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Dermatomyositis
Intervention(s)
Drug: Apremilast
Primary Outcome(s)
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(s)
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 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.]
Secondary ID(s)
16975
SU-03302010-5522
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 04/03/2015
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT01140503
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