Ignite Creation Date:
2024-05-05 @ 11:25 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00035958
Status:
TERMINATED
Last Update Posted:
2013-08-01
First Post:
2002-05-07
Brief Title:
Understanding the Pathogenesis and Treatment of Childhood Onset Dermatomyositis
Sponsor:
Childrens Hospital Medical Center Cincinnati
Organization:
Childrens Hospital Medical Center Cincinnati
Study Overview
Official Title:
Toward Improved Understanding of Pathogenesis and Treatment of Childhood Onset Dermatomyositis
Status:
TERMINATED
Status Verified Date:
2013-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Incorporating the recommendations of the NIH-formed DSMB in the study procedures would make the project budget over the limit for this funding mechanism
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
Detailed Description:
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 PMTX and combination of oral prednisone and etanercept PE 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 PE will be tested and compared to both P alone and the combination of PMTX after 3 6 12 18 and 24 months of treatment In addition the combination of PMTX 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
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 PMTX and combination of oral prednisone and etanercept PE 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 PE will be tested and compared to both P alone and the combination of PMTX after 3 6 12 18 and 24 months of treatment In addition the combination of PMTX 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| NIAMS-078 | US NIH GrantContract | None | httpsreporternihgovquickSearchP60AR047784 |
| P60AR047784 | NIH | None | None |