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Imaging of Arthropathy in Boys With Hemophilia in China

Primary Purpose

Hemophilia

Status

Active

Phase

Not Applicable

Locations

China

Study Type

Interventional

Intervention

Study group

About this trial

This is an interventional diagnostic trial for Hemophilia focused on measuring Hemophilia, Radiogenomics, Arthropathy

Eligibility Criteria

4 Years - 11 Years (Child)MaleDoes not accept healthy volunteers

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).

Sites / Locations

Beijing Children's Hospital

Outcomes

Primary Outcome Measures

Annualized total index joint bleeding rates (AJBRs)

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

Secondary Outcome Measures

Number of participants with joint inflammation

Assessed by ultrasound

Number of participants with joint inflammation

Assessed by MRI of the joints

Number of participants with joint damage

Assessed by ultrasound

Number of participants with joint damage

Assessed by MRI of the joints

Number of participants with clinical arthropathy

Assessed by the Hemophilia Joint Health Score tool (HJHS), version 2.1 - Joint score range: 0 (normal) to 16 (worse outcome).

Presence of inflammatory biomarkers in plasma

Measured by ELISA (enzyme-linked immunosorbent assay)

Internal MRI-based soft tissue score change

Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)

Internal MRI-based osteochondral tissue score change

Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)

Full Information

NCT ID

NCT03914716

First Posted

February 19, 2019

Last Updated

April 21, 2023

Sponsor

Andrea Doria

Collaborators

Beijing Children's Hospital, Baxalta US Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03914716

Brief Title

Imaging of Arthropathy in Boys With Hemophilia in China

Official Title

Radiogenomics: Personalized Imaging of Arthropathy in Boys With Hemophilia in China

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

March 21, 2018 (Actual)

Primary Completion Date

June 2023 (Anticipated)

Study Completion Date

March 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Andrea Doria

Collaborators

Beijing Children's Hospital, Baxalta US Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia

Keywords

Hemophilia, Radiogenomics, Arthropathy

7. Study Design

Primary Purpose

Diagnostic

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

60 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Diagnostic Test

Intervention Name(s)

Study group

Intervention Description

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

Primary Outcome Measure Information:

Title

Annualized total index joint bleeding rates (AJBRs)

Description

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

Time Frame

Between baseline and 24 months

Secondary Outcome Measure Information:

Title

Number of participants with joint inflammation

Description

Assessed by ultrasound

Time Frame

At baseline, 6 months and 24 months

Title

Number of participants with joint inflammation

Description

Assessed by MRI of the joints

Time Frame

Between baseline and 24 months

Title

Number of participants with joint damage

Description

Assessed by ultrasound

Time Frame

At baseline, 6 months and 24 months

Title

Number of participants with joint damage

Description

Assessed by MRI of the joints

Time Frame

Between baseline and 24 months

Title

Number of participants with clinical arthropathy

Description

Assessed by the Hemophilia Joint Health Score tool (HJHS), version 2.1 - Joint score range: 0 (normal) to 16 (worse outcome).

Time Frame

Every 6 months

Title

Presence of inflammatory biomarkers in plasma

Description

Measured by ELISA (enzyme-linked immunosorbent assay)

Time Frame

At baseline, 6 months and 24 months

Title

Internal MRI-based soft tissue score change

Description

Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)

Time Frame

Between baseline and 24 months

Title

Internal MRI-based osteochondral tissue score change

Description

Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)

Time Frame

Between baseline and 24 months

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

4 Years

Maximum Age & Unit of Time

11 Years

Accepts Healthy Volunteers

Eligibility 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).

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Andrea Doria, MD

Organizational Affiliation

The Hospital for Sick Children

Official's Role

Principal Investigator

Facility Information:

Facility Name

Beijing Children's Hospital

City

Beijing

Country

China

12. IPD Sharing Statement

Plan to Share IPD

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Imaging of Arthropathy in Boys With Hemophilia in China

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