Hemophilia A or BPhase 3NCT07545395
Safety of KN057 Prophylaxis in Patients With Haemophilia A or B
A Phase 3 study testing KN057 in people with Hemophilia A or B, run by Suzhou Alphamab Co., Ltd. at 1 site in China.
Trial summary
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the pharmacokinetic characteristics of the new and old processes KN057. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia A or B
- What is being tested?
- KN057
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteChina
- Study dates
- 13 Mar 2026to 30 Mar 2028
- Phase
- Phase 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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male, 12 to 65 years old at the time of signing informed consent, body weight ≥30 kg and BMI <28 kg/m^2 at screening.
- For participates with inhibitors: Tested positive for high-titer FVIII or FIX inhibitors (≥ 5 BU/mL) at screening; or tested positive for low-titer FVIII or FIX inhibitors (0.6 BU/mL or upper limit of normal [ULN] < inhibitor titer < 5 BU/mL) at screening, with ongoing treatment using bypassing agents (rFVIIa or PCC).
- For participates without inhibitors: Severe and moderately severe hemophilia A or hemophilia B (FVIII or FIX activity level ≤2%); FVIII or FIX inhibitor test is negative (<0.6 BU/ml) or lower than the lower limit of laboratory normal values during the screening period; There is no history of FVIII or FIX inhibitors in the past, or there has been an inhibitor, but the inhibitor has turned negative for at least 5 years before screening and has not reappeared (no positive inhibitor was detected); Use coagulation factor replacement therapy for no less than 100 exposure days before screening.
- Participates with inhibitors agree to avoid using PCC for treatment when breakthrough bleeding occurred. Participates without inhibitors agree to be treated with standard half-life coagulation factors (FVIII or FIX) in the event of breakthrough bleeding.
Who cannot join
- Have serious or poorly controlled chronic diseases or obvious systemic diseases.
- Have a history of thromboembolic disease, or currently have symptoms or signs related to thromboembolic disease or being treated with thrombolytic/antithrombotic therapy.
- Have high-risk factors for thrombosis: such as atrial fibrillation, atherosclerotic diseases of important arteries, ischemic disease of important organs, vascular occlusive disease, autoimmune diseases with a high risk of thrombosis, or indwelling central venous catheter.
- Known or suspected hypersensitivity to any constituent of the trial product or related products.
- Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study.
- Used Emicizumab treatment within 6 months before screening.
- Have received any gene therapy for hemophilia in the past.
- Other factors that the investigator deems inappropriate for participating in this trial, such as the presence of concomitant diseases, treatment or examination abnormalities that affect the subject's safety during the trial or affect the interpretation of trial results.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Renchi Yang, Doctor
About this study
Official title: An Open-label Study to Evaluate the Safety of KN057 Long-term Prophylaxis in Patients With Hemophilia A or Hemophilia B With or Without Inhibitors
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the pharmacokinetic characteristics of the new and old processes KN057.
The participants in Part PK will be randomly assigned to Old process Group or New process Group in a 1:1 ratio. The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks. The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks.
Read more
The participants in Part non-PK will be non-randomized and treated with new process KN057 for 52 weeks prophylaxis after enrollment.
Priority screening and enrollment of participants who have participated in the KN057-A-301 or KN057-A-302 study.
- Conditions
- Hemophilia A or B
- Being tested
- KN057
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KN057Drug
KN057 will be administered subcutaneously once a week.
| Study group | Role | What they receive |
|---|---|---|
| Part PK # Old process Group | Experimental | The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks. Receives: Drug: KN057 |
| Part PK # New process Group | Experimental | The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks. Receives: Drug: KN057 |
| Part non-PK | Experimental | The participants in Part non-PK will be treated with new process KN057 for 52 weeks prophylaxis after enrollment. Receives: Drug: KN057 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of TEAE.
TEAE refers to 'treatment emergent adverse event'.
Measured: Up to 12/26/56 weeks.
Incidence of TEAE related to the experimental drug.
Measured: Up to 12/26/56 weeks.
Incidence of SAE.
SAE refers to 'serious adverse event'.
Measured: Up to 12/26/56 weeks.
Incidence of thromboembolic events.
Measured: Up to 12/26/56 weeks.
Incidence of TMA and DIC.
TMA refers to 'thrombotic microangiopathy'. DIC refers to 'disseminated intravascular coagulation'.
Measured: Up to 12/26/56 weeks.
Incidence of hypersensitivity type reactions.
Measured: Up 12/26/56 weeks.
Incidence of injection site reactions.
Measured: Up to 12/26/56 weeks.
Incidence of clinically significant laboratory value abnormalities.
Measured: Up to 12/26/56 weeks.
Number of participants with clinically significant changes from baseline in electrocardiograms.
Measured: Up to 12/26/56 weeks.
Number of participants with clinically significant changes from baseline in vital signs.
Measured: Up to 12/26/56 weeks.
Number of participants with clinically significant changes from baseline in physical exam.
Measured: Up to 12/26/56 weeks.
Secondary outcomes (10)
The exposure levels of KN057 after the first administration in both the new and old processes.
Measured: Up to 12 weeks.
The steady-state trough concentrations of KN057 after the first administration in both the new and old processes.
Measured: Up to 12 weeks.
Incidence of anti-KN057 antibody (ADA) and neutralizing antibody (Nab).
immunogenicity
Measured: Up to 12/26/56 weeks.
Annualized bleeding rate (ABR) calculated based on treated spontaneous and traumatic bleeding episodes.
Measured: Up to 26/52 weeks.
ABR calculated based on bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes.
Measured: Up to 26/52 weeks.
The correlation between the steady-state trough concentrations of KN057 and the incidence of TEAE related to the experimental drug.
Measured: Up to 26 weeks.
The correlation between the steady-state trough concentrations of KN057 and ABR calculated based on treated spontaneous and traumatic bleeding episodes.
Measured: Up to 26 weeks.
Levels of Free TFPI.
pharmacodynamics
Measured: Up to 12/26/56 weeks.
Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L).
The EQ-5D-5L questionnaire is made up for 2 components, health state description and evaluation. In description part, health status is measured in terms of 5 dimensions (5D): mobility, self-care, usual activities, pain/discomfort, and anxiety/depression; every dimension contains 5 levels (5L): no difficulty, a little difficulty, moderate difficulty, severe difficulty, very severe difficulty/inability to perform. In evaluation part, the respondents evaluate their overall health status using the visual analogue scale (EQ-VAS) ranging from 0 (worst imaginable health) to 100 (best imaginable health).
Measured: Up to 26/52 weeks.
The annual usage of on-demand treatment drugs (adjusted by body weight).
Measured: Up to 26/52 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2026
- Study start13 Mar 2026
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A or BStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Suzhou Alphamab Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Renchi Yang, Doctor, Principal investigator, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical SciencesHu Zhou, Doctor, Principal investigator, Henan Cancer HospitalChangcheng Zheng, Doctor, Principal investigator, The First Affiliated Hospital of China University of Science and Technology (Anhui Provincial)Xielan Zhao, Principal investigator, Xiangya Hospital of Central South UniversityHongbo Cheng, Doctor, Principal investigator, Jiangxi Provincial People's HopitalYanping Song, Doctor, Principal investigator, Xi'an Central HospitalZeping Zhou, Doctor, Principal investigator, The Second Affiliated Hospital of Kunming Medical UniversityJie Yin, Doctor, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07545395
- Sponsor's protocol ID
- KN057-A-303
- Status last verified
- Apr 2026
- First submitted
- 8 Mar 2026
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study to Evaluate the Safety of KN057 Long-term Prophylaxis in Patients With Hemophilia A or Hemophilia B With or Without Inhibitors
- ClinicalTrials.gov ID
- NCT07545395
- Acronym / sponsor ID
- KN057-A-303
- Conditions
- Hemophilia A or B
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Suzhou Alphamab Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Mar 2026 (actual)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 30 Mar 2028 (estimated)
- First posted
- 22 Apr 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.
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the pharmacokinetic characteristics of the new and old processes KN057.
The participants in Part PK will be randomly assigned to Old process Group or New process Group in a 1:1 ratio. The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks. The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks.
The participants in Part non-PK will be non-randomized and treated with new process KN057 for 52 weeks prophylaxis after enrollment.
Priority screening and enrollment of participants who have participated in the KN057-A-301 or KN057-A-302 study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Part PK # Old process Group | Experimental | Drug: KN057The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks. |
| Part PK # New process Group | Experimental | Drug: KN057The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks. |
| Part non-PK | Experimental | Drug: KN057The participants in Part non-PK will be treated with new process KN057 for 52 weeks prophylaxis after enrollment. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male, 12 to 65 years old at the time of signing informed consent, body weight ≥30 kg and BMI <28 kg/m^2 at screening.
- For participates with inhibitors: Tested positive for high-titer FVIII or FIX inhibitors (≥ 5 BU/mL) at screening; or tested positive for low-titer FVIII or FIX inhibitors (0.6 BU/mL or upper limit of normal [ULN] < inhibitor titer < 5 BU/mL) at screening, with ongoing treatment using bypassing agents (rFVIIa or PCC).
- For participates without inhibitors: Severe and moderately severe hemophilia A or hemophilia B (FVIII or FIX activity level ≤2%); FVIII or FIX inhibitor test is negative (<0.6 BU/ml) or lower than the lower limit of laboratory normal values during the screening period; There is no history of FVIII or FIX inhibitors in the past, or there has been an inhibitor, but the inhibitor has turned negative for at least 5 years before screening and has not reappeared (no positive inhibitor was detected); Use coagulation factor replacement therapy for no less than 100 exposure days before screening.
- Participates with inhibitors agree to avoid using PCC for treatment when breakthrough bleeding occurred. Participates without inhibitors agree to be treated with standard half-life coagulation factors (FVIII or FIX) in the event of breakthrough bleeding.
Key exclusion criteria (8 listed)
- Have serious or poorly controlled chronic diseases or obvious systemic diseases.
- Have a history of thromboembolic disease, or currently have symptoms or signs related to thromboembolic disease or being treated with thrombolytic/antithrombotic therapy.
- Have high-risk factors for thrombosis: such as atrial fibrillation, atherosclerotic diseases of important arteries, ischemic disease of important organs, vascular occlusive disease, autoimmune diseases with a high risk of thrombosis, or indwelling central venous catheter.
- Known or suspected hypersensitivity to any constituent of the trial product or related products.
- Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study.
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 |
|---|---|---|
| KN057KN057 will be administered subcutaneously once a week. | Drug | Part PK # New process Group, Part PK # Old process Group, Part non-PK |
Primary outcome
Incidence of TEAE.
Time frame: Up to 12/26/56 weeks.
TEAE refers to 'treatment emergent adverse event'.
Incidence of TEAE related to the experimental drug.
Time frame: Up to 12/26/56 weeks.
Incidence of SAE.
Time frame: Up to 12/26/56 weeks.
SAE refers to 'serious adverse event'.
Incidence of thromboembolic events.
Time frame: Up to 12/26/56 weeks.
Incidence of TMA and DIC.
Time frame: Up to 12/26/56 weeks.
TMA refers to 'thrombotic microangiopathy'. DIC refers to 'disseminated intravascular coagulation'.
Incidence of hypersensitivity type reactions.
Time frame: Up 12/26/56 weeks.
Incidence of injection site reactions.
Time frame: Up to 12/26/56 weeks.
Incidence of clinically significant laboratory value abnormalities.
Time frame: Up to 12/26/56 weeks.
Number of participants with clinically significant changes from baseline in electrocardiograms.
Time frame: Up to 12/26/56 weeks.
Number of participants with clinically significant changes from baseline in vital signs.
Time frame: Up to 12/26/56 weeks.
Number of participants with clinically significant changes from baseline in physical exam.
Time frame: Up to 12/26/56 weeks.
10 secondary outcomes
The exposure levels of KN057 after the first administration in both the new and old processes.
Time frame: Up to 12 weeks.
The steady-state trough concentrations of KN057 after the first administration in both the new and old processes.
Time frame: Up to 12 weeks.
Incidence of anti-KN057 antibody (ADA) and neutralizing antibody (Nab).
Time frame: Up to 12/26/56 weeks.
Annualized bleeding rate (ABR) calculated based on treated spontaneous and traumatic bleeding episodes.
Time frame: Up to 26/52 weeks.
ABR calculated based on bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes.
Time frame: Up to 26/52 weeks.
The correlation between the steady-state trough concentrations of KN057 and the incidence of TEAE related to the experimental drug.
Time frame: Up to 26 weeks.
The correlation between the steady-state trough concentrations of KN057 and ABR calculated based on treated spontaneous and traumatic bleeding episodes.
Time frame: Up to 26 weeks.
Levels of Free TFPI.
Time frame: Up to 12/26/56 weeks.
Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L).
Time frame: Up to 26/52 weeks.
The annual usage of on-demand treatment drugs (adjusted by body weight).
Time frame: Up to 26/52 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 22 Apr 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Hemophilia A or B
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.
Renchi Yang, Doctor principal investigator
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Hu Zhou, Doctor principal investigator
Henan Cancer Hospital
Changcheng Zheng, Doctor principal investigator
The First Affiliated Hospital of China University of Science and Technology (Anhui Provincial)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing KN057 in people with Hemophilia A or B, run by Suzhou Alphamab Co., Ltd. at 1 site in China.
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 8 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 22 Apr 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 China. 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 phase mean?
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?
Contact Yanrong Dong, Master (+86 18914005458, yanrongdong@alphamab.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of TEAE. (time frame: Up to 12/26/56 weeks.). The study lists 11 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 22 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.