Understanding the Pathogenesis and Treatment of Childhood Onset Dermatomyositis
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|ClinicalTrials.gov Identifier: NCT00035958|
Recruitment Status : Terminated (Incorporating the recommendations of the NIH-formed DSMB in the study procedures would make the project budget over the limit for this funding mechanism.)
First Posted : May 8, 2002
Last Update Posted : August 1, 2013
|Condition or disease||Intervention/treatment||Phase|
|Dermatomyositis||Drug: Prednisone Drug: Methotrexate Drug: Etanercept||Phase 2 Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||75 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Toward Improved Understanding of Pathogenesis and Treatment of Childhood Onset Dermatomyositis|
|Study Start Date :||August 2002|
|Actual Primary Completion Date :||August 2002|
|Actual Study Completion Date :||August 2002|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00035958
|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|