Preventing bleeds in patients with hemophilia using KN057
A Randomized, Open-label Study to Evaluate the Efficacy and Safety of KN057 Injection Prophylaxis in Patients With Hemophilia A or B With Inhibitors
This study is testing if a new treatment called KN057 can help prevent bleeding in people with hemophilia A or B who have inhibitors.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 51 (estimated) |
| Ages | 12 Years to 70 Years |
| Sex | Male |
| Sponsor | Suzhou Alphamab Co., Ltd. Industry-sponsored |
| Locations | 1 site (Tianjin, Tianjin) |
| Trial ID | NCT06312475 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the efficacy and safety of KN057 as a prophylactic treatment for patients with hemophilia A or B who have inhibitors. Participants will be randomly assigned to receive either KN057 prophylaxis or no prophylaxis for a duration of 52 weeks, with a follow-up period included. The study will assess the frequency of bleeding episodes and the overall safety profile of KN057 compared to standard treatment. The trial consists of a 59-week period, including screening, treatment, and follow-up phases.
Who should consider this trial
Good fit: Ideal candidates are males aged 12 to 70 years with hemophilia A or B and a positive inhibitor test.
Not a fit: Patients with serious chronic diseases or a history of thromboembolic disease may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce bleeding episodes in patients with hemophilia A or B with inhibitors.
How similar studies have performed: Other studies have explored similar prophylactic approaches in hemophilia, but the specific use of KN057 is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. 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; 2. 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; 3. ≥6 treated bleeding episodes within 26 weeks before screening; 4. Have not used TFPI antibody drugs before; 5. Be able and agree to elute prior drugs for the treatment of hemophilia. 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. 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); 6. Ongoing or planned Immune Tolerance Induction treatment; 7. When bleeding occurred in the past, rFVIIa was ineffective and PCC treatment must be used; 8. Known or suspected hypersensitivity to any constituent of the trial product or related products; 9. Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study.
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
- Email: yanrongdong@alphamab.com
- Phone: +86 18914005458
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.