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Sponsors and Collaborators: |
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) Immunex Corporation |
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Information provided by: | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
ClinicalTrials.gov Identifier: | NCT00035958 |
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.
Condition | Intervention | Phase |
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Dermatomyositis |
Drug: Prednisone Drug: Methotrexate Drug: Etanercept |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Toward Improved Understanding of Pathogenesis and Treatment of Childhood Onset Dermatomyositis |
Estimated Enrollment: | 75 |
The mortality of JDMS is 3-39% with over 40% of patients demonstrating long-term disability. Current first line therapy is high-dose corticosteroids with the attendant significant drug-related toxicity. Over 30% of JDMS patients fail to respond adequately to steroids and require additional immunosuppression, none of which have been tested in prospective, randomized, controlled trials. The unique occlusive vasculopathy in JDMS is critical in the pathogenesis and predictive of prognosis but poorly understood. Elevated levels of tumor necrosis factor (TNF) alpha have been shown to be present in JDMS and are associated with a more severe and chronic course.
Seventy-five children with definite JDMS will be enrolled in a 24-month prospective, randomized, multicentered trial comparing 3 treatments: oral prednisone (P), combination of oral prednisone and methotrexate (P/MTX), and combination of oral prednisone and etanercept (P/E). Primary response measures will be muscle strength and mean duration of steroid therapy. Secondary response variables are disability in daily function and height and weight growth velocity (steroid toxicity measures). The combination of P/E will be tested and compared to both P alone and the combination of P/MTX after 3, 6, 12, 18, and 24 months of treatment. In addition, the combination of P/MTX will be compared to P alone after 3, 6, 12, 18, and 24 months of treatment. In pretreatment muscle biopsies, proangiogenic factors (such as vascular endothelial cell growth factor and basic fibroblast growth factor), angiostatic factors (such as angiostatin and endostatin), and vascular morphology (vessel number, width, length and area) will be quantified and tested for ability to predict muscle strength and functional ability 3, 6, 12, 18, and 24 months later.
Ages Eligible for Study: | 4 Years to 16 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion criteria at the time of enrollment:
Exclusion criteria at the time of enrollment:
United States, Ohio | |
Cincinnati Childrens Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 |
Principal Investigator: | Daniel J. Lovell, MD, MPH | Children's Hospital Medical Center, Cincinnati |
Study ID Numbers: | P60 AR47784, NIAMS-078 |
Study First Received: | May 7, 2002 |
Last Updated: | January 2, 2007 |
ClinicalTrials.gov Identifier: | NCT00035958 |
Health Authority: | United States: Food and Drug Administration |
Juvenile Dermatomyositis Pediatric Randomized Clinical Trial Pediatric Rheumatology |
Prednisone Juvenile dermatomyositis Skin Diseases TNFR-Fc fusion protein Folic Acid Myositis Dermatomyositis |
Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Polymyositis Connective Tissue Diseases Idiopathic myopathy Methotrexate |
Antimetabolites Anti-Inflammatory Agents Antimetabolites, Antineoplastic Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists Reproductive Control Agents Hormones Sensory System Agents Therapeutic Uses Abortifacient Agents Anti-Inflammatory Agents, Non-Steroidal Analgesics |
Dermatologic Agents Nucleic Acid Synthesis Inhibitors Antineoplastic Agents, Hormonal Nervous System Diseases Gastrointestinal Agents Enzyme Inhibitors Abortifacient Agents, Nonsteroidal Folic Acid Antagonists Immunosuppressive Agents Glucocorticoids Pharmacologic Actions Analgesics, Non-Narcotic Peripheral Nervous System Agents Antirheumatic Agents Central Nervous System Agents |