Imaging of Arthropathy in Boys With Hemophilia in China

NCT03914716 | Completed

• 1 other identifier: 1000058500
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 1

Brief Summary

Hemophilia is a genetic condition characterized by marked phenotypic heterogeneity. Bleeding into a joint is the single most important risk factor for the development of hemophilic arthropathy (HA). It is thought that clinical and imaging manifestations of HA are at least partially attributable to genetic polymorphisms unrelated to the hemophilia genotype. Identifying and characterizing biologic factors that could explain differences in susceptibility to joint degeneration of patients with hemophilia would help stratify patients according to the risk of degeneration of their joints and develop personalized therapeutic and prophylactic strategies. This study is conducted in China.

Conditions

Hemophilia

Keywords

Hemophilia; Radiogenomics; Arthropathy

Outcome Measures

Primary Outcomes (1)

• Annualized total index joint bleeding rates (AJBRs)

  AJBRs will be calculated from prospectively collected joint bleeding logs and clinic records.

  Between baseline and 24 months

Secondary Outcomes (8)

• Number of participants with joint inflammation

  At baseline, 6 months and 24 months

• Number of participants with joint inflammation

  Between baseline and 24 months

• Number of participants with joint damage

  At baseline, 6 months and 24 months

• Number of participants with joint damage

  Between baseline and 24 months

• Number of participants with clinical arthropathy

  Every 6 months

Interventions

Study group DIAGNOSTIC_TEST

Subjects will have physical, imaging examinations and provide samples for biological markers

Eligibility Criteria

• Age: 4 Years - 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Hemophilia A with baseline FVIII levels of \<2%
• Clinical history of ≥ 50 exposure days to FVIII prior to the study start.
• On-demand treatment, prophylaxis FVIII infusions or treatment with plasma-derived products for \>3 months prior to enrollment into the study.

You may not qualify if:

• History of FVIII inhibitor (titer \>0.6 Bethesda Units \[BU\])
• Chronic renal failure (serum creatinine \>2.0 mg /dL).
• Chronic liver disease (alanine aminotransferase \[ALT\] \>200 U/L).
• Clinically documented immunodeficiency.
• Anticipation of need for major surgery during the study period.
• Association of diseases known to mimic or cause joint diseases such as symptomatic human immunodeficiency virus (HIV) infection, juvenile idiopathic arthritis, and metabolic bone diseases.
• Social barriers for participation in the study such as long distance between home and the comprehensive care centre, and documented track record of non-compliance to therapies or participation in clinical studies.
• Neuro-developmental/behavioral problems.
• Contraindications to MR imaging (presence of heart pacemakers, metallic foreign bodies in the eye, aneurysm clips, severe claustrophobia).

Sponsors & Collaborators

• Andrea Doria: lead
• Beijing Children's Hospital, Baxalta US Inc.: collaborator

Study Sites (1)

Beijing Children's Hospital

Beijing, China

Location

MeSH Terms

Conditions

Hemophilia A; 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; Musculoskeletal Diseases

Study Officials

• Andrea Doria, MD

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Radiologist, Senior Scientist, Research Director, Department of Diagnostic Imaging

Study Record Dates

• First Submitted: February 19, 2019
• First Posted: April 16, 2019
• Study Start: March 21, 2018
• Primary Completion: February 2, 2022
• Study Completion: November 30, 2023
• Last Updated: April 19, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)