Hemophilia A PatientPhase 2/3NCT07439055
Real-World Joint Outcomes After Selective Transition to Low-Dose Emicizumab in Pediatric Hemophilia A
A Phase 2/3 study testing Switching to low dose emicizumab in people with Hemophilia A Patient, run by Chulalongkorn University at 1 site in Thailand.
Trial summary
Recent studies have shown that pharmacokinetic (PK)-guided extended half-life (EHL) factor VIII can improve joint health in patients with hemophilia A. However, some patients experience suboptimal joint outcomes despite optimized PK-guided therapy. Read more about this study
- Who is it for?
- Ages 3 to 30 years, men only
- Conditions the study is for
- Hemophilia A Patient
- What is being tested?
- Switching to low dose emicizumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteThailand
- Study dates
- 1 Jun 2025to 1 Apr 2026
- Phase
- Phase 2/3
- 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 3 to 30 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- hemophilia A patients aged 3-30 years who received treatment with PK-guided EHL Factor VIII for more than 8 months
Who cannot join
- hemophilia A patients with presence of factor VIII inhibitors
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Study of Clinical Outcomes, Cartilage Biomarkers and Hemophilic Arthropathy (HEAD-US) in Hemophilia A Patients : Low Dose Emicizumab vs. Pharmacokinetic-Guided Extended Half-Life FVIII Concentrates
Recent studies have shown that pharmacokinetic (PK)-guided extended half-life (EHL) factor VIII can improve joint health in patients with hemophilia A. However, some patients experience suboptimal joint outcomes despite optimized PK-guided therapy. Low-dose emicizumab has emerged as a potential option for improving bleeding control and joint health in patients who do not respond adequately to PK-guided EHL factor VIII.
The objectives of the study is to compare clinical bleeding outcomes, joint health, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) in hemophilia A patients in Thailand during PK-guided EHL factor VIII therapy and after switching to low-dose emicizumab.
Eighteen patients with hemophilia A from King Chulalongkorn Memorial Hospital, Thailand, aged 8-28 years, who were receiving PK-guided EHL factor VIII were enrolled. Patients with a Hemophilia Joint Health Score (HJHS) ≥ 12 were switched to low-dose emicizumab. A loading dose of 2 mcg/kg was administered, followed by dosing every 2 weeks during the first month and every 4 weeks thereafter. Clinical bleeding, annual bleeding rate (ABR), annual joint bleeding rate (AJBR), HJHS, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) were evaluated every 4 months for up to 8 months after switching.
- Conditions
- Hemophilia A Patient
- Being tested
- Switching to low dose emicizumab
- Keywords
- Hemophilia A, Hemarthrosis, Extended half life factor VIII, Low dose Emicizumab, Cartilage biomarker, HEAD-US
- Medical subject headings
- Hemophilia A, Hemarthrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Switching to low dose emicizumabDrug
All Hemophilia A patients in the study had received treatment with PK-guided EHL factor VIII. The patients who had HJHS more than 12 were switched to treatment with low dose emicizumab
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A patients with HJHS more than 12 | Experimental | All Hemophilia A patients in the study had received treatment with PK-guided EHL factor VIII. The patients who had HJHS more than 12 were switched to treatment with low dose emicizumab Receives: Drug: Switching to low dose emicizumab |
| Hemophilia A patients with HJHS less than 12 | No intervention | Hemophilia A patients whose HJHS was less than 12 continue the same PK-guided EHL Factor VIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual bleeding rate
Annual bleeding rate report as times per year
Measured: 8 months
Annual joint bleeding rate
Annual joint bleeding rate report as times per year
Measured: 8 months
Hemophilia Joint Health Score (HJHS)
HJHS is the physical examination assessment tool for hemophilia patients evaluated most commonly 6 joints affected by bleeding (both elbows, both knees and both ankles). The scores range from 0 (minimum) to 125 (maximum). The lowest score means the best joint health.
Measured: 8 months
The quality of life assessment instrument for children and adolescents with haemophilia (Hae-QoL)
The assessment tools evaluate physical, psychological, family, social aspect of Hemophilia patients. There are 35 questions. Each questions has multiple choices (never, seldom, somtimes, often, always) represent score 1 (never) to 5 (always). The total raw score range from 35 to 175 and then must be transform to "transformed score" ranged from 0 to 100. 0 is the best and 100 is the worst score.
Measured: 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start1 Jun 2025
- Primary completion1 Mar 2026 (estimated)
- Study completion1 Apr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A PatientStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chulalongkorn University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07439055
- Sponsor's protocol ID
- 0415/67
- Status last verified
- Jan 2026
- First submitted
- 20 Jan 2026
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Study of Clinical Outcomes, Cartilage Biomarkers and Hemophilic Arthropathy (HEAD-US) in Hemophilia A Patients : Low Dose Emicizumab vs. Pharmacokinetic-Guided Extended Half-Life FVIII Concentrates
- ClinicalTrials.gov ID
- NCT07439055
- Acronym / sponsor ID
- 0415/67
- Conditions
- Hemophilia A Patient
- Keywords
- Hemophilia A, Hemarthrosis, Extended half life factor VIII, Low dose Emicizumab, Cartilage biomarker, HEAD-US
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Chulalongkorn UniversityUniversity, hospital or other
- Study start
- 1 Jun 2025 (actual)
- Primary completion
- 1 Mar 2026 (estimated)
- Study completion
- 1 Apr 2026 (estimated)
- First posted
- 27 Feb 2026 (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.
Recent studies have shown that pharmacokinetic (PK)-guided extended half-life (EHL) factor VIII can improve joint health in patients with hemophilia A. However, some patients experience suboptimal joint outcomes despite optimized PK-guided therapy. Low-dose emicizumab has emerged as a potential option for improving bleeding control and joint health in patients who do not respond adequately to PK-guided EHL factor VIII.
The objectives of the study is to compare clinical bleeding outcomes, joint health, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) in hemophilia A patients in Thailand during PK-guided EHL factor VIII therapy and after switching to low-dose emicizumab.
Eighteen patients with hemophilia A from King Chulalongkorn Memorial Hospital, Thailand, aged 8-28 years, who were receiving PK-guided EHL factor VIII were enrolled. Patients with a Hemophilia Joint Health Score (HJHS) ≥ 12 were switched to low-dose emicizumab. A loading dose of 2 mcg/kg was administered, followed by dosing every 2 weeks during the first month and every 4 weeks thereafter. Clinical bleeding, annual bleeding rate (ABR), annual joint bleeding rate (AJBR), HJHS, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) were evaluated every 4 months for up to 8 months after switching.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A patients with HJHS more than 12 | Experimental | Drug: Switching to low dose emicizumabAll Hemophilia A patients in the study had received treatment with PK-guided EHL factor VIII. The patients who had HJHS more than 12 were switched to treatment with low dose emicizumab |
| Hemophilia A patients with HJHS less than 12 | No intervention | –Hemophilia A patients whose HJHS was less than 12 continue the same PK-guided EHL Factor VIII |
Who may qualify
- Ages
- 3 to 30 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- hemophilia A patients aged 3-30 years who received treatment with PK-guided EHL Factor VIII for more than 8 months
Key exclusion criteria (1 listed)
- hemophilia A patients with presence of factor VIII inhibitors
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 |
|---|---|---|
| Switching to low dose emicizumabAll Hemophilia A patients in the study had received treatment with PK-guided EHL factor VIII. The patients who had HJHS more than 12 were switched to treatment with low dose emicizumab | Drug | Hemophilia A patients with HJHS more than 12 |
Primary outcome
Annual bleeding rate
Time frame: 8 months
Annual bleeding rate report as times per year
Annual joint bleeding rate
Time frame: 8 months
Annual joint bleeding rate report as times per year
Hemophilia Joint Health Score (HJHS)
Time frame: 8 months
HJHS is the physical examination assessment tool for hemophilia patients evaluated most commonly 6 joints affected by bleeding (both elbows, both knees and both ankles). The scores range from 0 (minimum) to 125 (maximum). The lowest score means the best joint health.
The quality of life assessment instrument for children and adolescents with haemophilia (Hae-QoL)
Time frame: 8 months
The assessment tools evaluate physical, psychological, family, social aspect of Hemophilia patients. There are 35 questions. Each questions has multiple choices (never, seldom, somtimes, often, always) represent score 1 (never) to 5 (always). The total raw score range from 35 to 175 and then must be transform to "transformed score" ranged from 0 to 100. 0 is the best and 100 is the worst score.
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 27 Feb 2026.
- Individual participant dataSharing planned: Undecided
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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. This study is not recruiting at the moment.
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 Phase 2/3 study testing Switching to low dose emicizumab in people with Hemophilia A Patient, 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 1 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: under way, not looking for participants, as of the sponsor's last update on 27 Feb 2026. 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.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Annual bleeding rate (time frame: 8 months). The study lists 4 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 27 Feb 2026.