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Hemophilia A With InhibitorPhase 3NCT06312475

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

A Phase 3 study testing KN057 in people with Hemophilia A With Inhibitor and Hemophilia B With Inhibitor, run by Suzhou Alphamab Co., Ltd. at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use. Successfully screened participants will be randomly assigned to KN057 Prophylaxis (Arm 1) versus No Prophylaxis (Arm 2) at a ratio of 2:1. Read more about this study

Who is it for?
Ages 12 to 70 years, men only
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
53 participants
Where
1 siteChina
Study dates
9 Jan 2024to 15 Dec 2025
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 70 years
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male, 12 to 70 years old at the time of signing informed consent (including the cut-off value), body weight ≥25 kg and BMI <28 kg/m^2 at screening;
  • The FVIII or FIX inhibitor test is positive at a high titer (≥5 BU/ml) during the screening period; or the FVIII or FIX inhibitor is detected at a low titer (0.6 BU/ml or the upper limit of normal value < inhibitor titer < 5 BU/ml) during the screening period and treatment with bypass agents (rFVIIa or PCC) has been started;
  • ≥6 treated bleeding episodes within 26 weeks before screening;
  • Have not used TFPI antibody drugs before;
  • Be able and agree to elute prior drugs for the treatment of hemophilia.

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 a history of coronary atherosclerotic disease, ischemic disease of important organs, vascular occlusive disease, autoimmune diseases with a high risk of thrombosis, or indwelling central venous catheter;
  • The presence of other inherited or acquired bleeding disorders other than hemophilia A and hemophilia B;
  • Being on standard prophylaxis and maintaining it for more than 12 weeks (standard prophylaxis is defined as at least 80% compliance with a predetermined prophylaxis regimen);
  • Ongoing or planned Immune Tolerance Induction treatment;
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  • When bleeding occurred in the past, rFVIIa was ineffective and PCC treatment must be used;
  • 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.

Trial locations and contacts

1 site in 1 country.

China1 site
  • Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 300020

Other trials in China

About this study

Official title: A Randomized, Open-label Study to Evaluate the Efficacy and Safety of KN057 Injection Prophylaxis in Patients With Hemophilia A or B With Inhibitors

The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use. Successfully screened participants will be randomly assigned to KN057 Prophylaxis (Arm 1) versus No Prophylaxis (Arm 2) at a ratio of 2:1. Participants in KN057 Prophylaxis will receive KN057 prophylaxis for 52 weeks upon enrollment. Participants in No Prophylaxis will first receive on-demand treatment for 26 weeks, then switch to KN057 prophylaxis for 26 weeks.The trial period is 59 weeks, including a 3-week screening period, a 26-week main trial, a 26-week extension period, and a 4-week follow-up period after the last administration.

Being tested
KN057

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 groups in NCT06312475
Study groupRoleWhat they receive
Arm 1: KN057 ProphylaxisExperimentalSuccessfully screened participants will be randomly assigned to KN057 Prophylaxis versus No Prophylaxis at a ratio of 2:1. Participants in Arm 1 (KN057 Prophylaxis) will receive KN057 through the main trial (26 weeks) and extension period (26 weeks) for total of approximately 1 year.
Receives: Drug: KN057
Arm 2: No ProphylaxisExperimentalSuccessfully screened participants will be randomly assigned to KN057 Prophylaxis versus No Prophylaxis at a ratio of 2:1. Participants in Arm 2 (No Prophylaxis) will continue on-demand treatment with their usual bypass agents (rFVIIa or PCC) through the main trial for 26 weeks, in the extension period they will switch to prophylaxis treatment and receive KN057 for 26 weeks.
Receives: Drug: KN057

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Annualized bleeding rate (ABR) calculated based on treated spontaneous and traumatic bleeding episodes in Arm 1 and Arm 2.

    Treated bleeding refers to the use of bypass agents and/or coagulation factors for hemostatic treatment of the bleeding.

    Measured: From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

Secondary outcomes (19)
  1. ABR calculated based on bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes, and treated target joint bleeding respectively in Arm 1 and Arm 2.

    Bleeding episodes includes spontaneous and/or traumatic bleeding episodes, excluding surgery/procedure-related bleeding episodes. Treated bleeding refers to the use of bypass agents and/or coagulation factors for hemostatic treatment of the bleeding. Target joint are those with spontaneous bleeding ≥3 times in the 6 months prior to screening.

    Measured: From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

  2. ABR calculated based on bleeding episodes and treated bleeding episodes respectively in Arm 2.

    Bleeding episodes includes spontaneous and/or traumatic bleeding episodes, excluding surgery/procedure-related bleeding episodes. Treated bleeding refers to the use of bypass agents and/or coagulation factors for hemostatic treatment of the bleeding.

    Measured: From Day 183 (the beginning of the extension period) to Day 365 (the end of the extension period), approximately 26 weeks in total

  3. ABR calculated based on bleeding episodes, treated bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes, and treated target joint bleeding respectively in Arm 1.

    Bleeding episodes includes spontaneous and/or traumatic bleeding episodes, excluding surgery/procedure-related bleeding episodes. Treated bleeding refers to the use of bypass agents and/or coagulation factors for hemostatic treatment of the bleeding. Target joint are those with spontaneous bleeding ≥3 times in the 6 months prior to screening.

    Measured: From Day 1 (the beginning of the main trial) to Day 365 (the end of the extension period), approximately 52 weeks in total

  4. Change from baseline in Hemophilia Joint Health Score (HJHS) scores in Arm 1 and Arm 2.

    Hemophilia Joint Health Scores (HJHS) is a validated 11-item scoring tool developed for the assessment of joint health in participants with hemophilia. It comprised an evaluation of the elbows, knee and ankle joints: swelling (0 to 3), duration of swelling (0 and 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2) and strength (0 to 4), in each item 0 = none and higher score = severe damage and global gait (walking, stairs, running, hopping on 1 leg) scored on scale ranged from 0 to 4, where 0 = all skills in normal limit and 4 = no skills within normal limits). Total HJHS score = sum of joint totals (0 to 120) + general gait (1 to 4) and ranged from 0 (no joint damage) to 124 (severe joint damage), where higher score indicated severe joint damage.

    Measured: From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

  5. Change in HJHS scores from baseline in Arm 1 and from the 26th week in Arm 2.

    Hemophilia Joint Health Scores (HJHS) is a validated 11-item scoring tool developed for the assessment of joint health in participants with hemophilia. It comprised an evaluation of the elbows, knee and ankle joints: swelling (0 to 3), duration of swelling (0 and 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2) and strength (0 to 4), in each item 0 = none and higher score = severe damage and global gait (walking, stairs, running, hopping on 1 leg) scored on scale ranged from 0 to 4, where 0 = all skills in normal limit and 4 = no skills within normal limits). Total HJHS score = sum of joint totals (0 to 120) + general gait (1 to 4) and ranged from 0 (no joint damage) to 124 (severe joint damage), where higher score indicated severe joint damage.

    Measured: From Day 1 (the beginning of the main trial) to Day 365 (the end of the extension period), approximately 52 weeks in total

  6. Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L) in Arm 1 and Arm 2.

    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: From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

  7. Change in EQ-5D-5L from baseline in Arm 1 and from the 26th week in Arm 2.

    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: From Day 1 (the beginning of the main trial) to Day 365 (the end of the extension period), approximately 52 weeks in total

  8. Incidence of TEAE, TEAE related to the experimental drug and SAE.

    TEAE refers to 'treatment emergent adverse event'. SAE refers to 'serious adverse event'.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  9. Incidence of thromboembolic events, TMA and DIC.

    TMA refers to 'thrombotic microangiopathy'. DIC refers to 'disseminated intravascular coagulation'.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  10. Incidence of hypersensitivity type reactions.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  11. Incidence of injection site reactions.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  12. Incidence of clinically significant laboratory value abnormalities.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  13. Number of participants with clinically significant changes from baseline in physical exam.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  14. Number of participants with clinically significant changes from baseline in electrocardiograms.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  15. Number of participants with clinically significant changes from baseline in vital signs.

    Measured: From the signing of informed consent to 4 weeks after the last dose of KN057 (the end of the trial), approximately 59 weeks in total

  16. KN057 plasma trough concentration.

    pharmacokinetics

    Measured: From start of KN057 treatment (Day 1 for KN057 prophylaxis group, Day 183 for no prophylaxis group) to 4 weeks after the last dose of KN057, approximately 56 weeks for KN057 prophylaxis group and 30 weeks for no prophylaxis group in total

  17. Levels of Free TFPI.

    pharmacodynamics

    Measured: From start of KN057 treatment (Day 1 for KN057 prophylaxis group, Day 183 for no prophylaxis group) to Day 365, approximately 52 weeks for KN057 prophylaxis group and 26 weeks for no prophylaxis group in total

  18. Levels of prothrombin fragment 1+2 (PF1+2).

    pharmacodynamics

    Measured: From start of KN057 treatment (Day 1 for KN057 prophylaxis group, Day 183 for no prophylaxis group) to Day 365, approximately 52 weeks for KN057 prophylaxis group and 26 weeks for no prophylaxis group in total

  19. Incidence of anti-KN057 antibody (ADA) and neutralizing antibody (Nab).

    immunogenicity

    Measured: From start of KN057 treatment (Day 1 for KN057 prophylaxis group, Day 183 for no prophylaxis group) to 4 weeks after the last dose of KN057, approximately 56 weeks for KN057 prophylaxis group and 30 weeks for no prophylaxis group in total

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Mar 2024
  2. Study start9 Jan 2024
  3. Primary completion15 Oct 2025 (estimated)
  4. Study completion15 Dec 2025 (estimated)

Resources

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
53 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06312475
Sponsor's protocol ID
KN057-A-301
Status last verified
Sep 2025
First submitted
30 Dec 2023
Last updated
17 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing KN057 in people with Hemophilia A With Inhibitor and Hemophilia B With Inhibitor, 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 5 inclusion and 9 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 17 Sep 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 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?

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?

Annualized bleeding rate (ABR) calculated based on treated spontaneous and traumatic bleeding episodes in Arm 1 and Arm 2. (time frame: From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total). 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 17 Sep 2025.

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