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

NCT ID: NCT06312475

Last Updated: 2025-09-17

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 53 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-01-09
• Study Completion Date: 2025-12-15

Brief Summary

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.

Conditions

Hemophilia A With Inhibitor; Hemophilia B With Inhibitor

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm 1: KN057 Prophylaxis

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.

Group Type: EXPERIMENTAL

KN057

Intervention Type: DRUG

KN057 will be administered subcutaneously once a week.

Arm 2: No Prophylaxis

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.

Group Type: EXPERIMENTAL

KN057

Intervention Type: DRUG

KN057 will be administered subcutaneously once a week.

Interventions

KN057

KN057 will be administered subcutaneously once a week.

Intervention Type: DRUG

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 ≥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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Suzhou Alphamab Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Renchi Yang, Doctor

Role: PRINCIPAL_INVESTIGATOR

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

Jing Sun, Doctor

Role: PRINCIPAL_INVESTIGATOR

Nanfang Hospital, Southern Medical University

Hu Zhou, Doctor

Role: PRINCIPAL_INVESTIGATOR

Henan Cancer Hospital

Changcheng Zheng, Doctor

Role: PRINCIPAL_INVESTIGATOR

The First Affiliated Hospital of USTC (Anhui Provincial Hospital)

Xielan Zhao, Doctor

Role: PRINCIPAL_INVESTIGATOR

Xiangya Hospital of Central South University

Lili Chen, Doctor

Role: PRINCIPAL_INVESTIGATOR

Tai Zhou First People's Hospital

Chenghao Jin, Doctor

Role: PRINCIPAL_INVESTIGATOR

Jiangxi Provincial People's Hopital

Yanping Song, Doctor

Role: PRINCIPAL_INVESTIGATOR

Xi'an Central Hospital

Yaming Xi, Doctor

Role: PRINCIPAL_INVESTIGATOR

LanZhou University

Zeping Zhou, Doctor

Role: PRINCIPAL_INVESTIGATOR

The Second Affiliated Hospital of Kunming Medical University

Runhui Wu, Doctor

Role: PRINCIPAL_INVESTIGATOR

Beijing Children's Hospital

Ziqiang Yu, Doctor

Role: PRINCIPAL_INVESTIGATOR

The First Affiliated Hospital of Soochow University

Jingyu Yan, Doctor

Role: PRINCIPAL_INVESTIGATOR

North China University of Science and Technology

Sujun Gao, Doctor

Role: PRINCIPAL_INVESTIGATOR

The First Hospital of Jilin University

Wei Yang, Doctor

Role: PRINCIPAL_INVESTIGATOR

Shengjing Hospital of China University

Rong Zhou, Doctor

Role: PRINCIPAL_INVESTIGATOR

The Third People's Hospital of Chengdu

Locations

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

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

KN057-A-301

Identifier Type: -

Identifier Source: org_study_id