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

NCT06816953 | Enrolling By Invitation

• 2 other identifiers: 0736/67Ratchadapisek Research Fund
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hemophilia A

Keywords

Hemophilic arthropathy; Diagnostic ultrasound; Severe hemophilia A

Outcome Measures

Primary Outcomes (2)

• The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis

  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

  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 Outcomes (1)

• Correlation between cartilage biomarkers and Inflammatory markers

  From enrollment to the end of treatment at 32 weeks

Study Arms (2)

Standard low dose FVIII prophylaxis

EXPERIMENTAL

Standard weight based low dose FVIII concentrates prophylaxis

Diagnostic Test: HEAD-US; Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen; Diagnostic Test: Inflammatory markers

PK guided Extended half life FVIII prophylaxis

ACTIVE COMPARATOR

Pharmacokinetic guided extended half life FVIII prophylaxis

Diagnostic Test: HEAD-US; Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen; Diagnostic Test: Inflammatory markers

Interventions

HEAD-US DIAGNOSTIC_TEST

HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle)

PK guided Extended half life FVIII prophylaxis; Standard low dose FVIII prophylaxis

Urinary carboxy-terminal cross linked telopeptide of type II collagen DIAGNOSTIC_TEST

Cartilage biomarker represent cartilage degradation

PK guided Extended half life FVIII prophylaxis; Standard low dose FVIII prophylaxis

Inflammatory markers DIAGNOSTIC_TEST

Inflammatory markers included CBC, Ferritin, ESR, CRP

PK guided Extended half life FVIII prophylaxis; Standard low dose FVIII prophylaxis

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Chulalongkorn University: lead

Study Sites (1)

Pediatric Department, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital

Bangkok, Patumwan, 10330, Thailand

Location

MeSH Terms

Conditions

Hemophilia A

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

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: PwSHA receiving low-dose FVIII prophylaxis for ≥6 months were enrolled. Weight-based low-dose FVIII prophylaxis (10-15 IU/kg, 1-3 times/week) was continued for 4 months, then switched to PK-guided low-dose EHL FVIII prophylaxis, targeting a trough FVIII activity of 1 IU/dL, for another 4 months. ESR, CRP, ferritin, and CTX-II were measured at week 8 (during the weight-based low-dose regimen) and at week 24 (during the PK-guided regimen). HEAD-US scores were also compared at weeks 16 and 32. Bleeding rates and quality-of-life (QoL) score were analyzed parallelly.

Study Record Dates

• First Submitted: January 19, 2025
• First Posted: February 10, 2025
• Study Start: April 1, 2024
• Primary Completion: January 30, 2025
• Study Completion: February 28, 2025
• Last Updated: February 10, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: 1 year

Locations

TH (1)