Clinical trial NCT01000844: Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency
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 type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia
Keywords: Hemophilia
Joint
Cartilage
Damage
Prophylaxis
Factor VIII
FVIII
Disease
Arthropathy
Lead sponsor: University of Colorado, Denver
Sponsor class: OTHER
Collaborators: name: Bayer
name: Rush University Medical Center
name: Phoenix Children's Hospital
name: The University of Texas Health Science Center, Houston
name: Oregon Health and Science University
name: Emory University
name: University of Texas Southwestern Medical Center
name: Prisma Health-Midlands
name: Indiana University School of Medicine
name: Intermountain Health Care, Inc.
name: Ann & Robert H Lurie Children's Hospital of Chicago
First posted: 2009-10-23
Enrollment: 11
Sex: MALE
Minimum age (years): 8
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Study population will be invited based on their enrollment in the original Joint Outcome Study (JOS). This population

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.


Eligibility criteria: 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

Primary outcomes:
measure: 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.
timeFrame: Enrollment, age 14, and study exit at age 18
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: University of Colorado at Denver Health and Sciences Center
name: Marilyn Manco-Johnson, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Warren BB, Thornhill D, Stein J, Fadell M, Ingram JD, Funk S, Norton KL, Lane HD, Bennett CM, Dunn A, Recht M, Shapiro A, Manco-Johnson MJ. Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study. Blood Adv. 2020 Jun 9;4(11):2451-2459. doi: 10.1182/bloodadvances.2019001311.