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

NCT01140503 | Terminated Results DMC

• 2 other identifiers: SU-03302010-552216975
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Dermatomyositis

Outcome Measures

Primary Outcomes (1)

• 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.

  16 weeks

Secondary Outcomes (2)

• 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

Study Arms (1)

apremilast

EXPERIMENTAL

apremilast 20mg bid

Drug: Apremilast

Interventions

Apremilast DRUG

Apremilast 20mg PO BID

apremilast

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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:

Sponsors & Collaborators

• Stanford University: lead

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Dermatomyositis

Interventions

apremilast

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Limitations and Caveats

Study was terminated early due to low enrollment. Therefore we only have data on 5 participants.

Results Point of Contact

• Title: David Fiorentino
• Organization: Stanford University Department of Dermatology

fiorentino@stanford.edu

650-721-7160

Study Officials

• David Franklin Fiorentino

  Stanford University

  PRINCIPAL INVESTIGATOR

• Katharine Arefiev

  Stanford University

  SUB INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: April 6, 2010
• First Posted: June 9, 2010
• Study Start: February 1, 2010
• Primary Completion: August 1, 2011
• Study Completion: September 1, 2011
• Last Updated: March 4, 2015
• Results First Posted: March 4, 2015

Record last verified: 2015-03

Locations

US (1)