JOSC: Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency

Sponsor

University of Colorado, Denver (Other)

Overall Status

Completed

CT.gov ID

NCT01000844

Collaborator

Bayer (Industry), Rush University Medical Center (Other), Phoenix Children's Hospital (Other), The University of Texas Health Science Center, Houston (Other), Oregon Health and Science University (Other), Emory University (Other), University of Texas Southwestern Medical Center (Other), Prisma Health-Midlands (Other), Indiana University School of Medicine (Other), Intermountain Health Care, Inc. (Other), Ann & Robert H Lurie Children's Hospital of Chicago (Other)

Enrollment

Duration (Months)

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia

Study Design

Study Type:

Observational

Actual Enrollment :

11 participants

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'

Study Start Date :

Nov 1, 2009

Actual Primary Completion Date :

Oct 1, 2017

Actual Study Completion Date :

Oct 1, 2017

Outcome Measures

Primary Outcome Measures

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. [Enrollment, age 14, and study exit at age 18]

Eligibility Criteria

Criteria

Ages Eligible for Study:

8 Years to 18 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Inclusion Criteria:

Enrolled in the original JOS study, "A Randomized Prospective Study for the Prevention of Joint Disease in Children with Factor VIII Deficiency"
Written, informed consent of parent or guardian for the proposed study
The local hemophilia treatment center staff must evaluate the family's participation in the original treatment protocol and determine that the family is capable of complying with the continuation protocol