A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2011 by Stanford University.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Stanford University
ClinicalTrials.gov Identifier:
NCT01140503
First received: April 6, 2010
Last updated: July 22, 2011
Last verified: July 2011
  Purpose

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.


Condition Intervention
Dermatomyositis
Drug: Apremilast

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Study Evaluating the Safety and Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Dermatomyositis

Resource links provided by NLM:


Further study details as provided by Stanford University:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

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: 12 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 8
Study Start Date: February 2010
Estimated Study Completion Date: May 2012
Estimated Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: apremilast Drug: Apremilast
Apremilast 20mg PO BID

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 &#8805; 3000 /uL (&#8805; 3.0 X 10^9/L) and < 14,000/uL (< 14 X 10^9/L)
    • Platelets &#8805; 100,000 /uL (&#8805; 100 X 10^9/L)
    • Serum creatinine &#8804; 1.5 mg/dL (&#8804; 132.6 &#956;mol/L)
    • Total bilirubin &#8804; 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 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01140503

Contacts
Contact: Katharine Arefiev, MD (650) 723-6316 karefiev@stanford.edu

Locations
United States, California
Stanford University School of Medicine Recruiting
Stanford, California, United States, 94305
Contact: Katharine Arefiev, MD    650-723-6316    karefiev@stanford.edu   
Contact: Deborah Strahs, CCRC, CCRA    (650) 721-7147    dstrahs@stanford.edu   
Principal Investigator: David Franklin Fiorentino         
Sub-Investigator: Katharine Arefiev         
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: David Franklin Fiorentino Stanford University
Sub-Investigator: Katharine Arefiev Stanford University
  More Information

No publications provided

Responsible Party: David Franklin Fiorentino, Stanford University School of Medicine
ClinicalTrials.gov Identifier: NCT01140503     History of Changes
Other Study ID Numbers: SU-03302010-5522, 16975
Study First Received: April 6, 2010
Last Updated: July 22, 2011
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Myositis
Dermatomyositis
Muscular Diseases
Musculoskeletal Diseases
Polymyositis
Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases
Apremilast
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Anti-Inflammatory Agents
Therapeutic Uses
Antirheumatic Agents
Phosphodiesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Central Nervous System Agents

ClinicalTrials.gov processed this record on August 20, 2014