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

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ClinicalTrials.gov Identifier: NCT05981274
Recruitment Status : Recruiting
First Posted : August 8, 2023
Last Update Posted : April 10, 2024
Sponsor:
Information provided by (Responsible Party):
Chen Yeu-Chin, Tri-Service General Hospital

Study Description
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Brief 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)

Condition or disease Intervention/treatment
Severe Hemophilia A Without Inhibitor 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 Design
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Study Type : Observational
Estimated Enrollment : 45 participants
Observational Model: Other
Time Perspective: Other
Official Title: 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)
Actual Study Start Date : July 14, 2023
Estimated Primary Completion Date : December 31, 2027
Estimated Study Completion Date : December 31, 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Groups and Cohorts
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Group/Cohort Intervention/treatment
Systematic joint and bone health assessments before and after enrollment
  1. Haemophilia Early Arthropathy Detection :HEAD-US, HJHS, and DEXA scan
  2. serum markers : CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN)
  3. 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)


Outcome Measures
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Primary Outcome Measures :
  1. Systematic joint and bone health assessments before and after enrollment [ Time Frame: up to 5 years ]
    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.


Secondary Outcome Measures :
  1. Serum markers [ Time Frame: up to 5 years ]
    CTX-II, COMP, HSCRP, TNF-ALPHA, CTX-I, SRANKL, OPG, OPN


Biospecimen Retention:   Samples Without DNA
Blood samples (serum) will be obtained for serum markers.

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Males with one affected X chromosome is defined hemophilia disease; a female with one affected X chromosome is a "carrier" of hemophilia.
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
severe hemophilia A patients or moderate hemophilia A patients with hemophilic arthropathy.
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05981274


Contacts
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Contact: Yeu-Chin Chen (+886)2-87923311 ext 13654 yeuchin99@gmail.com

Locations
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Taiwan
Tri-Service General Hospital Hemophilia Care Center Recruiting
Taipei, Neihu Dist., Taiwan, 114202
Contact: Yeu-Chin chen    (+886)2-87923311 ext 13654    yeuchin99@gmail.com   
Sponsors and Collaborators
Tri-Service General Hospital
More Information
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Responsible Party: Chen Yeu-Chin, Director of the Hemophilia Center, Tri-Service General Hospital
ClinicalTrials.gov Identifier: NCT05981274    
Other Study ID Numbers: A202305097
First Posted: August 8, 2023    Key Record Dates
Last Update Posted: April 10, 2024
Last Verified: July 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn