Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B Without Inhibitors

NCT ID: NCT06569108

Last Updated: 2025-09-17

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 125 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-04-01
• Study Completion Date: 2026-12-15

Brief Summary

The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B without inhibitors and is safe to use. Participants receiving on-demand treatment prior to screening will be randomly assigned to Experimental group or Control group at a ratio of 2:1 in Part A. Participants receiving prophylaxis prior to screening will be nonrandomly assigned to Prophylaxis group in Part B. Participants in Experimental group will receive KN057 prophylaxis for 52 weeks upon enrollment. Participants in Control group will first receive on-demand treatment for 26 weeks, then switch to KN057 prophylaxis for 26 weeks. Participants in Prophylaxis group will first receive prophylaxis with coagulation factor for 26 weeks, then switch to KN057 prophylaxis for 26 weeks.

Conditions

Hemophilia A Without Inhibitor; Hemophilia B Without Inhibitor

Study Design

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

Study Groups

Part A：Experimental group

Successfully screened participants in Part A will be randomly assigned to Experimental group versus Control group at a ratio of 2:1. Participants in Experimental group will receive KN057 prophylaxis 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.

Part A：Control group

Successfully screened participants in Part A will be randomly assigned to Experimental group versus Control group at a ratio of 2:1. Participants in Control group will continue on-demand treatment with coagulation factor 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.

Part B：Prophylaxis group

Successfully screened participants in Part B will be nonrandomly assigned to Prophylaxis group. Participants in Prophylaxis group will continue prophylaxis with coagulation factor for the first 26 weeks (the factor period), then they will switch to prophylaxis with KN057 for the last 26 weeks (the KN057 period).

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 ≥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.

• 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

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

Ziqiang Yu, Doctor

Role: PRINCIPAL_INVESTIGATOR

The First Affiliated Hospital of Soochow University

Yun Chen, Doctor

Role: PRINCIPAL_INVESTIGATOR

Qianfoshan Hospital

Pingchong Lei, Doctor

Role: PRINCIPAL_INVESTIGATOR

Henan Provincial People's Hospital

Yinsuo Zheng, Doctor

Role: PRINCIPAL_INVESTIGATOR

Bao Ji Central Hospital

Peng Cheng, Doctor

Role: PRINCIPAL_INVESTIGATOR

First Affiliated Hospital of Guangxi Medical University

Jianwen Xiao, Doctor

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Chongqing Medical University

Ruibin Huang, Doctor

Role: PRINCIPAL_INVESTIGATOR

The First Affiliated Hospital of Nanchang University

Hailiang Li, Doctor

Role: PRINCIPAL_INVESTIGATOR

The first affiliated hospital of jiangxi medical college

Shu Chen, Doctor

Role: PRINCIPAL_INVESTIGATOR

The Second Affiliated Hospital of Chongqing Medical University

Xiong Zhang, Doctor

Role: PRINCIPAL_INVESTIGATOR

Maoming City People's Hospital

Jingyu Zhang, Doctor

Role: PRINCIPAL_INVESTIGATOR

The Second Hospital of Hebei Medical Hospital

Baolai Hua, Doctor

Role: PRINCIPAL_INVESTIGATOR

Beijing Shijitan Hospital, Capital Medical University

Yanming Zhang, Doctor

Role: PRINCIPAL_INVESTIGATOR

Huai'an Second People'Hospital

Locations

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

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

KN057-A-302

Identifier Type: -

Identifier Source: org_study_id