Understanding the Pathogenesis and Treatment of Childhood Onset Dermatomyositis

NCT00035958 | Terminated Phase 2

• 2 other identifiers: P60AR047784NIAMS-078
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

Juvenile dermatomyositis (JDMS) is one of the most serious of the childhood rheumatic diseases. The theory behind this trial is that early introduction of etanercept or methotrexate will prove to be effective in the treatment of JDMS. Pretreatment muscle biopsies, we believe there will be abnormalities in the blood vessels that will be correlated with worse physical strength and daily functional ability. The long-term goal is to improve the treatment of this serious childhood onset rheumatic disease and to better understand the pathogenic mechanism for the development of the vasculopathy (disorder of blood vessels) of JDMS. Identification of the specific mechanism of the vasculopathy may allow for the rational introduction of biologic treatments focused on vascular growth.

Conditions

Dermatomyositis

Keywords

Juvenile Dermatomyositis; Pediatric; Randomized Clinical Trial; Pediatric Rheumatology

Interventions

Prednisone DRUG

Methotrexate DRUG

Etanercept DRUG

Eligibility Criteria

• Age: 4 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Definite or probable diagnosis of JDMS by the criteria of Bohan and Peter
• No prior systemic steroid treatment for JDMS
• If able to become pregnant (females) or impregnate (males) and are sexually active, then must have negative serum pregnancy test at baseline and be utilizing effective form of birth control
• Patient and/or parent or legal guardian must be willing to sign consent and assent forms

You may not qualify if:

• History of chronic or recurrent infections sufficient to preclude the use of etanercept
• Prior treatment with specific TNF-blocking agents
• Demonstration of cutaneous or gastrointestinal (GI) ulceration at the time of diagnosis
• JDMS-related pulmonary disease at time of diagnosis (interstitial lung disease or aspiration pneumonia confirmed by radiograph)
• JDMS-related cardiomyopathy (echocardiogram confirmation)
• Any uncontrolled, clinically significant pre-existent systemic disease (hepatic, renal, neurological, endocrine, cardiac, gastrointestinal, or hematologic disease) within 24 weeks of start of study
• Known HIV, hepatitis B surface antigen not related to vaccination, or hepatitis C antibody positivity
• Pregnant or nursing female
• Any of the following laboratory abnormalities at baseline: platelet count \< 100,000/cmm, total white cell count of \< 3000 cells/cmm, neutrophils \< 1000 cells/cmm, serum bilirubin \> 2 times upper limit of normal, estimated creatinine clearance of \< 90 mL/min/1.73 M2 BSA estimated by formula for males age 2 to \<13 (0.55 X ht in cms/serum creatinine), age 13-18 (0.7 X ht in cms/serum creatinine) and females age 2-18 (0.55 X ht in cms/serum creatinine)
• Received live virus vaccination within 3 months prior to study entry (contraindication for MTX or etanercept therapy)
• Past or current substance abuse or psychiatric history that would interfere with ability to give informed consent or comply with study requirements or physician instructions

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS): collaborator
• Immunex Corporation: collaborator

Study Sites (1)

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Dermatomyositis

Interventions

Prednisone; Methotrexate; Etanercept

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Immunoglobulin Fc Fragments; Immunoglobulin Fragments; Peptide Fragments; Peptides; Amino Acids, Peptides, and Proteins; Immunoglobulin Constant Regions; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Receptors, Tumor Necrosis Factor; Receptors, Cytokine; Receptors, Immunologic; Receptors, Cell Surface; Membrane Proteins

Study Officials

• Daniel J. Lovell, MD, MPH

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: May 7, 2002
• First Posted: May 8, 2002
• Study Start: August 1, 2002
• Primary Completion: August 1, 2002
• Study Completion: August 1, 2002
• Last Updated: August 1, 2013

Record last verified: 2013-07

Locations

US (1)