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)

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)

Study Overview

• Status: Recruiting
• Conditions: Severe Hemophilia A Without Inhibitor
• Intervention / Treatment: Diagnostic test: serum biomarker.

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.

• Study Type: Observational
• Enrollment (Estimated): 45

Contacts and Locations

• Study Contact: Name: Yeu-Chin chen; Phone Number: 13654 (+886)2-87923311; Email: yeuchin99@gmail.com

Study Locations

• Taiwan
  • Neihu Dist.
    • Taipei, Neihu Dist., Taiwan, 114202
      • Recruiting
      • Tri-Service General Hospital Hemophilia Care Center
      • Contact: Yeu-Chin chen; Phone Number: 13654 (+886)2-87923311; Email: yeuchin99@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

severe hemophilia A patients or moderate hemophilia A patients with hemophilic arthropathy.

Description

Inclusion Criteria:

1. Patients with severe hemophilia A (all ages).
2. Patients with moderate type hemophilia A with hemophilic arthropathy.
3. Currently on rFVIIIFc regular prophylaxis and have previous medical record with regular prophylaxis with standard half-life product for one year
4. Able and willing to undergo joint and bone examinations

Exclusion Criteria:

1. Participants of other interventional studies.
2. Patients with current inhibitors.
3. History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
4. History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
5. Significantly impaired vision/hearing

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Systematic joint and bone health assessments before and after enrollment; Haemophilia Early Arthropathy Detection :HEAD-US, HJHS, and DEXA scan; serum markers : CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN); Quality of life assessments (Haem-A-QoL, EQ-5D)	Diagnostic test: serum biomarker.; serum biomarker:CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Systematic joint and bone health assessments before and after enrollment	Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) on patients' ankles, knees, and elbows. Also evaluate the Hemophilia Joint Health Score (HJHS) and Dexa scan at the hip.	up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Serum markers	CTX-II, COMP, HSCRP, TNF-ALPHA, CTX-I, SRANKL, OPG, OPN	up to 5 years

Collaborators and Investigators

• Sponsor: Tri-Service General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 14, 2023
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: July 17, 2023
• First Submitted That Met QC Criteria: July 31, 2023
• First Posted (Actual): August 8, 2023

Study Record Updates

• Last Update Posted (Actual): April 10, 2024
• Last Update Submitted That Met QC Criteria: April 8, 2024
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: A202305097

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No