Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency

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'

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01000844

Acronym

JOSC

Enrollment

Registered

2009-10-23

Start date

2009-11-30

Completion date

2017-10-31

Last updated

2019-10-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia

Keywords

Hemophilia, Joint, Cartilage, Damage, Prophylaxis, Factor VIII, FVIII, Disease, Arthropathy

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

8 Years to 18 Years

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

Exclusion criteria

* Unable or unwilling to record the study information

Design outcomes

Primary

Measure	Time frame
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

Outcome results