Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis (HEADUS_SHA8)

February 6, 2025 updated by: Chulalongkorn University

Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving Low Dose FVIII Prophylaxis

Patients with severe hemophilia A often develop joint complication requiring careful monitoring. This study aimed to compare the effectiveness of standard low dose weight-based FVIII concentrates (CFCs) prophylaxis with pharmacokinetic-guided extended half-life FVIII concentrates (eHLFVIII) using cartilage biomarkers and HEAD-US score.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

19

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Thailand
    • Patumwan
      • Bangkok, Patumwan, Thailand, 10330
        • Pediatric Department, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • patients with severe hemophilia A
  • previously receiving standard low dose FVIII prophylaxis 1-3 times per week for at least 6 months prior to enrollment in the project
  • no history of FVIII inhibitor (<0.6 Bethesda unit/ml)
  • obtained consent from both patients and their parents

Exclusion Criteria:

  • patients with underlying condition affecting the joints such as osteoarthritis, metabolic bone disease, etc.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Standard low dose FVIII prophylaxis
Standard weight based low dose FVIII concentrates prophylaxis
Diagnostic test: HEAD-US
HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle)
Diagnostic test: Urinary carboxy-terminal cross linked telopeptide of type II collagen
Cartilage biomarker represent cartilage degradation
Diagnostic test: Inflammatory markers
Inflammatory markers included CBC, Ferritin, ESR, CRP
Active Comparator: PK guided Extended half life FVIII prophylaxis
Pharmacokinetic guided extended half life FVIII prophylaxis
Diagnostic test: HEAD-US
HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle)
Diagnostic test: Urinary carboxy-terminal cross linked telopeptide of type II collagen
Cartilage biomarker represent cartilage degradation
Diagnostic test: Inflammatory markers
Inflammatory markers included CBC, Ferritin, ESR, CRP

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis
Time Frame: From enrollment to the end of treatment at 32 weeks
To compare the effectiveness of standard weight base FVIII concentrate prophylaxis and pharmacokinetic guided extended half life FVIII prophylaxis in managing hemophilic arthropathy using cartilage biomarkers.
From enrollment to the end of treatment at 32 weeks
The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis
Time Frame: From enrollment to the end of treatment at 32 weeks
To compare the effectiveness of standard weight base FVIII concentrate prophylaxis and pharmacokinetic guided extended half life FVIII prophylaxis in managing hemophilic arthropathy using HEAD-US score.
From enrollment to the end of treatment at 32 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Correlation between cartilage biomarkers and Inflammatory markers
Time Frame: From enrollment to the end of treatment at 32 weeks
To investigate the correlation between cartilage biomarkers (Urine CTX-II), Inflammatory markers (CBC, Ferritin, ESR, CRP) and ultrasound (HEAD-US score) comparing standard weight base low dose prophylaxis with pharmacokinetic guided extended half life FVIII concentrates
From enrollment to the end of treatment at 32 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chulalongkorn University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2024

Primary Completion (Estimated)

January 30, 2025

Study Completion (Estimated)

February 28, 2025

Study Registration Dates

First Submitted

January 19, 2025

First Submitted That Met QC Criteria

February 6, 2025

First Posted (Actual)

March 25, 2025

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 6, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 0736/67
  • Ratchadapisek Research Fund (Other Grant/Funding Number: Chulalongkorn University)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Sharing Time Frame

1 year

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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