Severe Hemophilia A Without InhibitorPhase 2/3NCT06155955
Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A
A Phase 2/3 study testing "Emicizumab", "HEMLIBRA®" in people with Severe Hemophilia A Without Inhibitor and Joint Bleed, run by Chulalongkorn University at 1 site in Thailand.
Trial summary
To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration To study pharmacokinetic, side effect of low dose Emicizumab Read more about this study
- Who is it for?
- Ages 3 to 30 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Severe Hemophilia A Without Inhibitor, Joint Bleed
- What is being tested?
- "Emicizumab", "HEMLIBRA®"
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteThailand
- Study dates
- 22 Jun 2023to 21 Jun 2024
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Severe or moderate Hemophilia A with baseline FVIII:C of <3 IU/dl or severe bleeding phenotype
- Receiving low dose FVIII prophylaxis for at least 6 months
Who cannot join
- Detectable FVIII inhibitor at screening
- Detectable FVIII inhibitor at screening Having other underlying diseases: Juvenile rheumatoid arthritis, metabolic bone diseases, or other conditions mimicking or causing joint diseases
Trial locations and contacts
1 site in 1 country.
Thailand1 site, 1 recruiting
- Recruiting
King Chulalongkorn Memorial hospital
Bangkok, 10330
NUCHANUN KESSAKORN, MD, 66825054639, nuchanun.ks@gmail.com
Darintr Sosothikul, Diplomate, dsosothikul@hotmail.com
About this study
Official title: Comparison of Outcomes Between Low Dose Emicizumab and Extended Half-life Factor VIII With Pharmacokinetic-guided Prophylaxis in Clinically Severe Hemophilia A
- To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration
- To study pharmacokinetic, side effect of low dose Emicizumab
Read the detailed description
- To compare outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration using ABR, AJBR and HJHS, HaemoQoL
- To study pharmacokinetic, side effect and effectiveness of low dose Emicizumab
- Conditions
- Severe Hemophilia A Without Inhibitor, Joint Bleed
- Being tested
- "Emicizumab", "HEMLIBRA®"
- Keywords
- emicizumab, hemophilia A
- Medical subject headings
- Hemarthrosis, Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
"Emicizumab", "HEMLIBRA®"Drug
Also called emicizumab-kxwh
low dose
| Study group | Role | What they receive |
|---|---|---|
| low dose emicizumab | Experimental | 2 mg per kilograms per dose in first month, loading every 2 weeks then every 4 weeks Receives: Drug: "Emicizumab", "HEMLIBRA®" |
| low dose factor VIII prophylaxis | No intervention | factor VIII prophylaxis |
What is being measured
The primary outcome is the main question the study is designed to answer.
spontaneous and traumatic bleeding rate
Using "Annualized bleeding rate" (ABR)
Measured: 6 months after start low dose emicizumab as secondary prophylaxis treatment
treated spontaneous bleeds, treated joint bleeds
Using "Annualized joint bleeding rate" (AJBR)
Measured: 6 months after start low dose emicizumab as secondary prophylaxis treatment
The function of the knee, elbow, and ankle joints
Using "Hemophilia Joint Health Score" (HJHS)
Measured: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2023
- Study start22 Jun 2023
- Primary completion21 Mar 2024 (estimated)
- Study completion21 Jun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Severe Hemophilia A Without InhibitorStudies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chulalongkorn University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nuchanun Kessakorn, MD, Chulalongkorn University
- Investigators
- NUCHANUN KESSAKORN, Principal investigator, King Chulalongkorn Memorial Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06155955
- Sponsor's protocol ID
- KingChulalongkorn
- Status last verified
- Mar 2024
- First submitted
- 11 Nov 2023
- Last updated
- 5 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kessakorn N, Gosriwatana I, Sasipong N, Srichumpuang C, Moonla C, Sosothikul D. Low-Dose Emicizumab Versus Low-/Intermediate-Dose Factor VIII Secondary Prophylaxis for Noninhibitor Haemophilia A Patients With Severe Bleeding Phenotype. Haemophilia. 2025 Jan;31(1):122-131. doi: 10.1111/hae.15146. Epub 2024 Dec 31.
PubMed 39737816
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of Outcomes Between Low Dose Emicizumab and Extended Half-life Factor VIII With Pharmacokinetic-guided Prophylaxis in Clinically Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT06155955
- Acronym / sponsor ID
- KingChulalongkorn
- Conditions
- Severe Hemophilia A Without Inhibitor, Joint Bleed
- Keywords
- emicizumab, hemophilia A
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Chulalongkorn UniversityUniversity, hospital or other
- Study start
- 22 Jun 2023 (actual)
- Primary completion
- 21 Mar 2024 (estimated)
- Study completion
- 21 Jun 2024 (estimated)
- First posted
- 5 Dec 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.
- To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration
- To study pharmacokinetic, side effect of low dose Emicizumab
Detailed description
- To compare outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration using ABR, AJBR and HJHS, HaemoQoL
- To study pharmacokinetic, side effect and effectiveness of low dose Emicizumab
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| low dose emicizumab | Experimental | Drug: "Emicizumab", "HEMLIBRA®"2 mg per kilograms per dose in first month, loading every 2 weeks then every 4 weeks |
| low dose factor VIII prophylaxis | No intervention | –factor VIII prophylaxis |
Who may qualify
- Ages
- 3 to 30 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Severe or moderate Hemophilia A with baseline FVIII:C of <3 IU/dl or severe bleeding phenotype
- Receiving low dose FVIII prophylaxis for at least 6 months
Key exclusion criteria (2 listed)
- Detectable FVIII inhibitor at screening
- Detectable FVIII inhibitor at screening Having other underlying diseases: Juvenile rheumatoid arthritis, metabolic bone diseases, or other conditions mimicking or causing joint diseases
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 |
|---|---|---|
| "Emicizumab", "HEMLIBRA®"Also: emicizumab-kxwhlow dose | Drug | low dose emicizumab |
Primary outcome
spontaneous and traumatic bleeding rate
Time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Using "Annualized bleeding rate" (ABR)
treated spontaneous bleeds, treated joint bleeds
Time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Using "Annualized joint bleeding rate" (AJBR)
The function of the knee, elbow, and ankle joints
Time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Using "Hemophilia Joint Health Score" (HJHS)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2024.
- Derived from this study: PubMed 39737816Kessakorn N, Gosriwatana I, Sasipong N, Srichumpuang C, Moonla C, Sosothikul D. Low-Dose Emicizumab Versus Low-/Intermediate-Dose Factor VIII Secondary Prophylaxis for Noninhibitor Haemophilia A Patients With Severe Bleeding Phenotype. Haemophilia. 2025 Jan;31(1):122-131. doi: 10.1111/hae.15146. Epub 2024 Dec 31.
- 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. This study is not recruiting at the moment.
NUCHANUN KESSAKORN principal investigator
King Chulalongkorn Memorial Hospital
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 "Emicizumab", "HEMLIBRA®" in people with Severe Hemophilia A Without Inhibitor and Joint Bleed, 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 2 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 5 Mar 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact NUCHANUN KESSAKORN, MD (662-5054639, nuchanun.ks@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
spontaneous and traumatic bleeding rate (time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06155955, which also lists 1 linked publication.
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 5 Mar 2024.