Hemophilia AHEADUS_SHA8NCT06816953
Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis
A study testing HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen and Inflammatory markers in people with Hemophilia A, run by Chulalongkorn University at 1 site in Thailand.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen and Inflammatory markers
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteThailand
- Study dates
- 1 Apr 2024to 28 Feb 2025
- Phase
- Interventional
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- patients with underlying condition affecting the joints such as osteoarthritis, metabolic bone disease, etc.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Thailand1 site
Pediatric Department, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital
Bangkok, Patumwan, 10330
About this study
Official title: Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving Low Dose FVIII Prophylaxis
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
- Being tested
- HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen, Inflammatory markers
- Keywords
- Hemophilic arthropathy, Diagnostic ultrasound, Severe hemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HEAD-USDiagnostic test
HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle)
Urinary carboxy-terminal cross linked telopeptide of type II collagenDiagnostic test
Cartilage biomarker represent cartilage degradation
Inflammatory markersDiagnostic test
Inflammatory markers included CBC, Ferritin, ESR, CRP
| Study group | Role | What they receive |
|---|---|---|
| Standard low dose FVIII prophylaxis | Experimental | Standard weight based low dose FVIII concentrates prophylaxis Receives: 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 Receives: Diagnostic Test: HEAD-US; Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen; Diagnostic Test: Inflammatory markers |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: From enrollment to the end of treatment at 32 weeks
Secondary outcomes (1)
Correlation between cartilage biomarkers and Inflammatory markers
To investigate the correlation between cartilage biomarkers (Urine CTX-II), Inflammatory markers (CBC, Ferritin, ESR, CRP) and ultrasound (HEAD-US score) comparing standard weight base low dose prophylaxis with pharmacokinetic guided extended half life FVIII concentrates
Measured: From enrollment to the end of treatment at 32 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2025
- Study start1 Apr 2024
- Primary completion30 Jan 2025 (estimated)
- Study completion28 Feb 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Chulalongkorn UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 19 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chulalongkorn University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06816953
- Sponsor's protocol ID
- 0736/67
- Other IDs
- Ratchadapisek Research Fund
- Acronym
- HEADUS_SHA8
- Status last verified
- Jan 2025
- First submitted
- 19 Jan 2025
- Last updated
- 10 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving Low Dose FVIII Prophylaxis
- ClinicalTrials.gov ID
- NCT06816953
- Acronym / sponsor ID
- HEADUS_SHA8 / 0736/67
- Conditions
- Hemophilia A
- Keywords
- Hemophilic arthropathy, Diagnostic ultrasound, Severe hemophilia A
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 19 participants (estimated)
- Lead sponsor
- Chulalongkorn UniversityUniversity, hospital or other
- Study start
- 1 Apr 2024 (actual)
- Primary completion
- 30 Jan 2025 (estimated)
- Study completion
- 28 Feb 2025 (estimated)
- First posted
- 10 Feb 2025 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Standard low dose FVIII prophylaxis | Experimental | Diagnostic Test: HEAD-US, Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen, Diagnostic Test: Inflammatory markersStandard weight based low dose FVIII concentrates prophylaxis |
| PK guided Extended half life FVIII prophylaxis | Active comparator | Diagnostic Test: HEAD-US, Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen, Diagnostic Test: Inflammatory markersPharmacokinetic guided extended half life FVIII prophylaxis |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- patients with underlying condition affecting the joints such as osteoarthritis, metabolic bone disease, etc.
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 |
|---|---|---|
| HEAD-USHEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle) | Diagnostic test | PK guided Extended half life FVIII prophylaxis, Standard low dose FVIII prophylaxis |
| Urinary carboxy-terminal cross linked telopeptide of type II collagenCartilage biomarker represent cartilage degradation | Diagnostic test | PK guided Extended half life FVIII prophylaxis, Standard low dose FVIII prophylaxis |
| Inflammatory markersInflammatory markers included CBC, Ferritin, ESR, CRP | Diagnostic test | PK guided Extended half life FVIII prophylaxis, Standard low dose FVIII prophylaxis |
Primary outcome
The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis
Time frame: From enrollment to the end of treatment at 32 weeks
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.
The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis
Time frame: From enrollment to the end of treatment at 32 weeks
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.
1 secondary outcome
Correlation between cartilage biomarkers and Inflammatory markers
Time frame: From enrollment to the end of treatment at 32 weeks
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 Feb 2025.
- Individual participant dataSharing planned: Yes
Related trials for Hemophilia A
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen and Inflammatory markers in people with Hemophilia A, run by Chulalongkorn University at 1 site in Thailand.
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 1 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: enrolling by invitation, as of the sponsor's last update on 10 Feb 2025. 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 Thailand. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis (time frame: From enrollment to the end of treatment at 32 weeks). The study lists 2 primary outcomes in all. 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 Feb 2025.