NCT05981274

A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)

A Prospective and Retrospective Interventional Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 45 (estimated)
• Sponsor: Tri-Service General Hospital · Academic / Other
• Sex: Male
• Age: —
• Healthy volunteers: Accepted

Summary

Study Objectives\* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3. Compare the clinical outcomes from 1 year before and after switching to EHL. 4. Exploratory: Identify biomarkers that could provide more useful and convenient evaluations of joint and bone health. (Time-saving and easy to monitor)

Detailed description

In this study, we would like to focus on the evaluation of joint and bone health in persons with severe hemophilia A (PwHA) treated with rFVIIIFc prophylaxis. We also aim to identify potential biomarkers for assessing the joint and bone health of PwHA. Since the joint and bone health examinations are time-consuming, expensive, and require special equipment, the patients could only receive the examinations annually to realize the status of their joint health. The biomarkers testing will provide a more convenient and time-saving option on the joint and bone health evaluation for PwHA. Therefore, surrogate of biochemical marker(s) to represent the joint and BMD progression is an unmet need for clinical practice. Investigating different biomarkers and combining them with the joint and BMD results to provide a systematic approach for elucidating the treatment outcomes of PwHA treated with rFVIIIFc is major goal of this study.

Conditions

• Severe Hemophilia A Without Inhibitor

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	serum biomarker.	serum biomarker:CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN)

Timeline

• Start date: 2023-07-14
• Primary completion: 2027-12-31
• Completion: 2027-12-31
• First posted: 2023-08-08
• Last updated: 2024-04-10

Locations

1 site across 1 country: Taiwan