Preventing bleeds in patients with Hemophilia A or B using KN057

A Randomized, Open-label Study to Evaluate the Efficacy and Safety of KN057 Injection Prophylaxis in Patients With Hemophilia A or B Without Inhibitors

This study is testing if a new treatment called KN057 can help prevent bleeding in boys and men with Hemophilia A or B who don't have inhibitors.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 111 (estimated)
• Ages: 12 Years to 70 Years
• Sex: Male
• Sponsor: Suzhou Alphamab Co., Ltd. (Industry-sponsored)
• Drugs / interventions: Emicizumab
• Locations: 1 site (Tianjin, Tianjin)
• Trial ID: NCT06569108 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of KN057 in preventing bleeds in patients with Hemophilia A or B who do not have inhibitors. Participants will be randomly assigned to receive either KN057 prophylaxis or on-demand treatment, with a portion also receiving standard prophylaxis before switching to KN057. The study aims to demonstrate that KN057 can effectively reduce bleeding episodes over a 52-week period. The trial includes male participants aged 12 to 70 years with severe or moderately severe hemophilia.

Who should consider this trial

Good fit: Ideal candidates are males aged 12 to 70 with severe or moderately severe Hemophilia A or B and no history of inhibitors.

Not a fit: Patients with a history of FVIII or FIX inhibitors or those who have not used coagulation factor replacement therapy for at least 100 exposure days may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce bleeding episodes for patients with Hemophilia A or B, improving their quality of life.

How similar studies have performed: Other studies have shown promise in using similar prophylactic approaches for hemophilia, indicating potential for success.

Eligibility criteria

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.

Where this trial is running

Tianjin, Tianjin

• Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences — Tianjin, Tianjin, China (Recruiting)

Study contacts

• Principal investigator: Renchi Yang, Doctor — Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
• Study coordinator: Yanrong Dong, Master

Conditions Hemophilia A Without Inhibitor; Hemophilia B Without Inhibitor