Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency

NCT01000844 | Completed DMC

• 1 other identifier: 01-436
• Study Type: observational
• Target: 11
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hemophilia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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

• Age: 8 Years - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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

You may qualify if:

• 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

You may not qualify if:

• Unable or unwilling to record the study information

Sponsors & Collaborators

• University of Colorado, Denver: lead
• Bayer: collaborator
• Rush University Medical Center: collaborator
• Phoenix Children's Hospital: collaborator
• The University of Texas Health Science Center, Houston: collaborator
• Oregon Health and Science University: collaborator
• Emory University: collaborator
• University of Texas Southwestern Medical Center: collaborator
• Prisma Health-Midlands: collaborator
• Indiana University School of Medicine: collaborator
• Intermountain Health Care, Inc.: collaborator
• Ann & Robert H Lurie Children's Hospital of Chicago: collaborator

Related Publications (1)

• 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.

  PMID: 32492157 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Whole Blood - EDTA, Citrate Plasma, and DNA

MeSH Terms

Conditions

Hemophilia A; Disease; Joint Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Musculoskeletal Diseases

Study Officials

• Marilyn Manco-Johnson, MD

  University of Colorado at Denver Health and Sciences Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2009
• First Posted: October 23, 2009
• Study Start: November 1, 2009
• Primary Completion: October 1, 2017
• Study Completion: October 1, 2017
• Last Updated: October 31, 2019

Record last verified: 2019-10