Severe Hemophilia A Without InhibitorNCT05981274
A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
An observational study of Severe Hemophilia A Without Inhibitor, run by Tri-Service General Hospital (TSGH) at 1 site in Taiwan.
Trial 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. Read more about this study
- Who is it for?
- Any age, men onlyHealthy volunteers can take part
- Conditions the study is for
- Severe Hemophilia A Without Inhibitor
- What is being tested?
- serum biomarker.
- How many people
- 45 participants
- Where
- 1 siteTaiwan
- Study dates
- 14 Jul 2023to 31 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: severe hemophilia A patients or moderate hemophilia A patients with hemophilic arthropathy.
Who may be able to join
- Patients with severe hemophilia A (all ages).
- Patients with moderate type hemophilia A with hemophilic arthropathy.
- Currently on rFVIIIFc regular prophylaxis and have previous medical record with regular prophylaxis with standard half-life product for one year
- Able and willing to undergo joint and bone examinations
Who cannot join
- Participants of other interventional studies.
- Patients with current inhibitors.
- History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
- History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
- Significantly impaired vision/hearing
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Taiwan1 site, 1 recruiting
- Recruiting
Tri-Service General Hospital Hemophilia Care Center
Taipei, Neihu Dist., 114202
Yeu-Chin chen, (+886)2-87923311 ext. 13654, yeuchin99@gmail.com
About this study
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)
Study Objectives*
- Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A
- Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health.
- Compare the clinical outcomes from 1 year before and after switching to EHL.
- Exploratory: Identify biomarkers that could provide more useful and convenient evaluations of joint and bone health. (Time-saving and easy to monitor)
Read the 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
- Being tested
- serum biomarker.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
serum biomarker.Diagnostic test
serum biomarker:CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN)
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Diagnostic Test: serum biomarker. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: up to 5 years
Secondary outcomes (1)
Serum markers
CTX-II, COMP, HSCRP, TNF-ALPHA, CTX-I, SRANKL, OPG, OPN
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2023
- Study start14 Jul 2023
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Hemophilia A Without InhibitorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tri-Service General Hospital (TSGH)
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Yeu-Chin, Tri-Service General Hospital (TSGH)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05981274
- Sponsor's protocol ID
- A202305097
- Status last verified
- Jul 2023
- First submitted
- 17 Jul 2023
- Last updated
- 10 Apr 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05981274
- Acronym / sponsor ID
- A202305097
- Conditions
- Severe Hemophilia A Without Inhibitor
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Tri-Service General Hospital (TSGH)University, hospital or other
- Study start
- 14 Jul 2023 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 8 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study Objectives*
- Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A
- Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health.
- Compare the clinical outcomes from 1 year before and after switching to EHL.
- 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.
Study design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Systematic joint and bone health assessments before and after enrollment | – | Diagnostic Test: serum biomarker.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) |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- severe hemophilia A patients or moderate hemophilia A patients with hemophilic arthropathy.
Key inclusion criteria (4 listed)
- Patients with severe hemophilia A (all ages).
- Patients with moderate type hemophilia A with hemophilic arthropathy.
- Currently on rFVIIIFc regular prophylaxis and have previous medical record with regular prophylaxis with standard half-life product for one year
- Able and willing to undergo joint and bone examinations
Key exclusion criteria (5 listed)
- Participants of other interventional studies.
- Patients with current inhibitors.
- History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
- History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
- Significantly impaired vision/hearing
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| serum biomarker.serum biomarker:CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN) | Diagnostic test | Systematic joint and bone health assessments before and after enrollment |
Primary outcome
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.
1 secondary outcome
Serum markers
Time frame: up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Severe Hemophilia A Without Inhibitor, run by Tri-Service General Hospital (TSGH) at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yeu-Chin Chen ((+886)2-87923311 ext. 13654, yeuchin99@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Systematic joint and bone health assessments before and after enrollment (time frame: up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2024.