Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency (JOSC)
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively until the age of six. At the age of six, the proportion of children on each treatment arm who developed bone or cartilage damage as determined by X-Ray or MRI was assessed. In addition, the function and structure of the index joints (defined as knees, ankles, and elbows)were evaluated using a physical assessment scale specially designed for preschool children.
The specific aim of the Joint Outcome Study Continuation (JOSC) is to extend observations of the children participating in the original JOS until the subjects reach the age of 18 years in order to determine the natural history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy. In addition, plasma and DNA will be collected and banked yearly for current and future studies of biomarkers and predictors of hemophilia outcomes.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Continuation of Children Enrolled in Protocol #95-011, 'A Randomized Prospective Study for the Prevention of Joint Disease in Children With Severe Factor VIII Deficiency'|
- Determine the natuaral history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy. [ Time Frame: Enrollment, age 14, and study exit at age 18 ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples With DNA
|Study Start Date:||November 2009|
|Estimated Study Completion Date:||January 2020|
|Estimated Primary Completion Date:||January 2019 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01000844
|Principal Investigator:||Marilyn Manco-Johnson, MD||University of Colorado at Denver Health and Sciences Center|