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

This study is enrolling participants by invitation only.
Sponsor:
Collaborators:
Bayer
Rush University Medical Center
Phoenix Children's Hospital
The University of Texas Health Science Center, Houston
Oregon Health and Science University
Emory University
University of Texas Southwestern Medical Center
Brewer, Erin, PharmD
Indiana University School of Medicine
Intermountain Health Care, Inc.
Ann & Robert H Lurie Children's Hospital of Chicago
Information provided by (Responsible Party):
University of Colorado, Denver
ClinicalTrials.gov Identifier:
NCT01000844
First received: October 22, 2009
Last updated: April 5, 2013
Last verified: October 2010
  Purpose

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
Hemophilia

Study Type: Observational
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'

Resource links provided by NLM:


Further study details as provided by University of Colorado, Denver:

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. [ Time Frame: Enrollment, age 14, and study exit at age 18 ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA

Whole Blood - EDTA, Citrate Plasma, and DNA


Estimated Enrollment: 50
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)
  Eligibility

Ages Eligible for Study:   8 Years to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01000844

Sponsors and Collaborators
University of Colorado, Denver
Bayer
Rush University Medical Center
Phoenix Children's Hospital
The University of Texas Health Science Center, Houston
Oregon Health and Science University
Emory University
University of Texas Southwestern Medical Center
Brewer, Erin, PharmD
Indiana University School of Medicine
Intermountain Health Care, Inc.
Ann & Robert H Lurie Children's Hospital of Chicago
Investigators
Principal Investigator: Marilyn Manco-Johnson, MD University of Colorado at Denver Health and Sciences Center
  More Information

No publications provided

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT01000844     History of Changes
Other Study ID Numbers: 01-0436
Study First Received: October 22, 2009
Last Updated: April 5, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by University of Colorado, Denver:
Hemophilia
Joint
Cartilage
Damage
Prophylaxis
Factor VIII
FVIII
Disease
Arthropathy

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 01, 2014