Long-term preventive KN057 treatment for Hemophilia A and B

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label, multicenter Phase 3 trial tests long-term prophylaxis with KN057 in males with Hemophilia A or B, including those with inhibitors, and compares pharmacokinetics between two manufacturing processes. In the PK part participants are randomized 1:1 to an old-process then new-process sequence or to continuous new-process KN057 for 52 weeks. A non-PK part enrolls participants non-randomly to receive new-process KN057 for 52 weeks. The trial prioritizes screening of participants who previously took part in KN057-A-301 or KN057-A-302.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. 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.
2. 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.
3. 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.

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 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.
4. Known or suspected hypersensitivity to any constituent of the trial product or related products.
5. Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study.
6. Used Emicizumab treatment within 6 months before screening.
7. Have received any gene therapy for hemophilia in the past.
8. 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 it's running

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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.