Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Imaging of Arthropathy in Boys With Hemophilia in China

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03914716
Recruitment Status : Recruiting
First Posted : April 16, 2019
Last Update Posted : October 23, 2020
Sponsor:
Collaborator:
Beijing Children's Hospital, Baxalta US Inc.
Information provided by (Responsible Party):
Andrea Doria, The Hospital for Sick Children

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.

Condition or disease Intervention/treatment Phase
Hemophilia Diagnostic Test: Study group Not Applicable

Detailed Description:
This will be a 3-year prospective cohort study conducted in a single centre (Beijing Children's Hospital, BCH, China) with a 2-year follow-up of patients Index joints (ankles, elbows and knees) of young Chinese boys with hemophilia A will be evaluated as follows: physical examination every 6 months using the Hemophilia Joint Health Score [HJHS], ultrasound imaging (gray-scale and color Doppler ultrasound [US]), and by laboratory (serum) at baseline, at 6, and 24 months. Magnetic resonance imaging (MRI) scans of index joints will be obtained at baseline, and 24 months. Features that will be captured either quantitatively or semantically in the imaging scans will be aggregated to generate "imaging phenotypes" which will be associated with clusters of co-expressed genes (metagenes) and clinical data.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Radiogenomics: Personalized Imaging of Arthropathy in Boys With Hemophilia in China
Actual Study Start Date : March 21, 2018
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : March 2024

Resource links provided by the National Library of Medicine



Intervention Details:
  • Diagnostic Test: Study group
    Subjects will have physical, imaging examinations and provide samples for biological markers


Primary Outcome Measures :
  1. Annualized total index joint bleeding rates (AJBRs) [ Time Frame: Between baseline and 24 months ]
    AJBRs will be calculated from prospectively collected joint bleeding logs and clinic records.


Secondary Outcome Measures :
  1. Number of participants with joint inflammation [ Time Frame: At baseline, 6 months and 24 months ]
    Assessed by ultrasound

  2. Number of participants with joint inflammation [ Time Frame: Between baseline and 24 months ]
    Assessed by MRI of the joints

  3. Number of participants with joint damage [ Time Frame: At baseline, 6 months and 24 months ]
    Assessed by ultrasound

  4. Number of participants with joint damage [ Time Frame: Between baseline and 24 months ]
    Assessed by MRI of the joints

  5. Number of participants with clinical arthropathy [ Time Frame: Every 6 months ]
    Assessed by the Hemophilia Joint Health Score tool (HJHS), version 2.1 - Joint score range: 0 (normal) to 16 (worse outcome).

  6. Presence of inflammatory biomarkers in plasma [ Time Frame: At baseline, 6 months and 24 months ]
    Measured by ELISA (enzyme-linked immunosorbent assay)

  7. Internal MRI-based soft tissue score change [ Time Frame: Between baseline and 24 months ]
    Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)

  8. Internal MRI-based osteochondral tissue score change [ Time Frame: Between baseline and 24 months ]
    Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)



Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   4 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03914716


Contacts
Layout table for location contacts
Contact: Andrea Doria, MD 416-813-7654 ext 306079 andrea.doria@sickkids.ca
Contact: Victor Blanchette, MD 416-813-5852 victor.blanchette@sickkids.ca

Locations
Layout table for location information
China
Beijing Children's Hospital Recruiting
Beijing, China
Contact: Runhui Wu, MD       runhuiwu@hotmail.com   
Sponsors and Collaborators
Andrea Doria
Beijing Children's Hospital, Baxalta US Inc.
Investigators
Layout table for investigator information
Principal Investigator: Andrea Doria, MD The Hospital for Sick Children
Layout table for additonal information
Responsible Party: Andrea Doria, Radiologist, Senior Scientist, Research Director, Department of Diagnostic Imaging, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT03914716    
Other Study ID Numbers: 1000058500
First Posted: April 16, 2019    Key Record Dates
Last Update Posted: October 23, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Andrea Doria, The Hospital for Sick Children:
Hemophilia
Radiogenomics
Arthropathy
Additional relevant MeSH terms:
Layout table for MeSH terms
Joint Diseases
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Musculoskeletal Diseases