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New weekly shot aims to stop bleeds in hemophilia patients

NCT ID NCT06569108

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This Phase 3 trial tests a new drug called KN057, given as a weekly injection, to prevent bleeding in people with hemophilia A or B who do not have inhibitors. The study includes 125 males aged 12 to 70 with severe or moderately severe hemophilia. Participants are split into groups to compare KN057 against standard on-demand or preventive treatment over 52 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
KN057 (a weekly injection to prevent bleeding)
What this could lead to
If it works, this could offer a new, convenient option to prevent bleeding episodes in people with hemophilia A or B without inhibitors.
What could go wrong
This is a Phase 3 trial, but results are not yet available. The drug may not reduce bleeding as hoped, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

125 people

The number who actually took part.

Started

Apr 2024

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 70 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Male, 12 to 70 years old at the time of signing informed consent (including the cut-off value), body weight ≥30 kg and BMI \<28 kg/m\^2 at screening; 2. Severe and moderately severe hemophilia A or hemophilia B (FVIII or FIX activity level ≤2%); 3. 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; 4. There is no history of FVIII or FIX inhibitors in the past; or there has been an inhibitor, but it has been at least 5 years since successful immune tolerance induction therapy (ITI), and the inhibitor has not reappeared (a positive inhibitor was detected after successful ITI); 5. Use coagulation factor replacement therapy for no less than 100 exposure days before screening; 6. Have not used Anti-TFPI drugs before; 7. Be able and agree to elute the original hemophilia drugs. Participants who are enrolled into Part A must also meet the following criteria: 1. ≥6 treated bleeding episodes within 26 weeks before screening; 2. Receiving on-demand treatment, non-standard prophylaxis, or standard prophylaxis no more than 12 weeks before screening; Participants who are enrolled into Part B must also meet the following criteria: 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). Exclusion Criteria: 1. Have serious or poorly controlled chronic diseases or obvious systemic diseases; 2. Have a history of thromboembolic disease, or currently have symptoms or signs related to thromboembolic disease or being treated with thrombolytic/antithrombotic therapy; 3. 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; 4. The presence of other inherited or acquired bleeding disorders other than hemophilia A and hemophilia B; 5. Known or suspected hypersensitivity to any constituent of the trial product or related products; 6. Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study; 7. Used Emicizumab treatment within 6 months before screening; 8. Have received any gene therapy for hemophilia in the past; 9. 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

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

    Tianjin, Tianjin Municipality, 300020, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.