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

Alphamab

Status and phase: Active, not recruiting; Phase 3

Conditions: Hemophilia B With Inhibitor; Hemophilia A With Inhibitor

Treatments: Drug: KN057

Study type: Interventional

Funder types: Industry

Identifiers

NCT06312475

KN057-A-301

Details and patient eligibility

About

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.

Enrollment: 53 patients

Sex: Male

Ages: 12 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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53 participants in 2 patient groups

Arm 1: KN057 Prophylaxis

Experimental group

Description:

Successfully 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.

Treatment:

Drug: KN057

Arm 2: No Prophylaxis

Experimental group

Description:

Successfully 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.

Treatment:

Drug: KN057