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

NCT06569108 | Active Not Recruiting Phase 3

• 1 other identifier: KN057-A-302
• Study Type: interventional
• Target: 125
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (2)

• Part A: Annualized bleeding rate (ABR) calculated based on treated spontaneous and traumatic bleeding episodes in Experimental group and Control group.

  Treated bleeding refers to the use of coagulation factors for hemostatic treatment of the bleeding.

  From Week 1 to Week 26, through the main trial.

• Part B: ABR calculated based on treated spontaneous and traumatic bleeding episodes in Prophylaxis group.

  Treated bleeding refers to the use of coagulation factors for hemostatic treatment of the bleeding.

  From Week 1 to Week 26, through the factor period. From Week 27 to Week 52, through the KN057 period.

Secondary Outcomes (21)

• ABR calculated based on bleeding episodes, treated spontaneous and traumatic bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes, and treated target joint bleeding respectively in Experimental group.

  From Week 1 to Week 52, through the main trial and extension period.

• ABR calculated based on bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes, and treated target joint bleeding respectively in Experimental group, Control group and Prophylaxis group.

  From Week 1 to Week 26. From Week 27 to Week 52.

• Proportion of participants with untreated bleeding episodes in Experimental group, Control group and Prophylaxis group.

  From Week 1 to Week 26. From Week 27 to Week 52.

• The annual usage of on-demand treatment drugs (adjusted by body weight) in Experimental group and Control group.

  From Week 1 to Week 26. From Week 27 to Week 52.

• Change from baseline in Hemophilia Joint Health Score (HJHS) scores in Experimental group, Control group and Prophylaxis group.

  From Week 1 to Week 26.

Study Arms (3)

Part A：Experimental group

EXPERIMENTAL

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.

Drug: KN057

Part A：Control group

EXPERIMENTAL

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.

Drug: KN057

Part B：Prophylaxis group

EXPERIMENTAL

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).

Drug: KN057

Interventions

KN057 DRUG

KN057 will be administered subcutaneously once a week.

Part A：Control group; Part A：Experimental group; Part B：Prophylaxis group

Eligibility Criteria

• Age: 12 Years - 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• 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 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);
• Use coagulation factor replacement therapy for no less than 100 exposure days before screening;
• Have not used Anti-TFPI drugs before;
• Be able and agree to elute the original hemophilia drugs.
• Participants who are enrolled into Part A must also meet the following criteria:
• ≥6 treated bleeding episodes within 26 weeks before screening;
• 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).

You may not qualify if:

• Have serious or poorly controlled chronic diseases or obvious systemic diseases;
• Have a history of thromboembolic disease, or currently have symptoms or signs related to thromboembolic disease or being treated with thrombolytic/antithrombotic therapy;
• 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;
• The presence of other inherited or acquired bleeding disorders other than hemophilia A and hemophilia B;
• Known or suspected hypersensitivity to any constituent of the trial product or related products;
• Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study;
• Used Emicizumab treatment within 6 months before screening;
• Have received any gene therapy for hemophilia in the past;
• 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.

Sponsors & Collaborators

• Suzhou Alphamab Co., Ltd.: lead

Study Sites (1)

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

Tianjin, Tianjin Municipality, 300020, China

Location

Study Officials

• Renchi Yang, Doctor

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

  PRINCIPAL INVESTIGATOR

• Jing Sun, Doctor

  Nanfang Hospital, Southern Medical University

  PRINCIPAL INVESTIGATOR

• Hu Zhou, Doctor

  Henan Cancer Hospital

  PRINCIPAL INVESTIGATOR

• Changcheng Zheng, Doctor

  The First Affiliated Hospital of USTC (Anhui Provincial Hospital)

  PRINCIPAL INVESTIGATOR

• Xielan Zhao, Doctor

  Xiangya Hospital of Central South University

  PRINCIPAL INVESTIGATOR

• Lili Chen, Doctor

  Tai Zhou First People's Hospital

  PRINCIPAL INVESTIGATOR

• Chenghao Jin, Doctor

  Jiangxi Provincial People's Hopital

  PRINCIPAL INVESTIGATOR

• Yanping Song, Doctor

  Xi'an Central Hospital

  PRINCIPAL INVESTIGATOR

• Yaming Xi, Doctor

  LanZhou University

  PRINCIPAL INVESTIGATOR

• Zeping Zhou, Doctor

  The Second Affiliated Hospital of Kunming Medical University

  PRINCIPAL INVESTIGATOR

• Runhui Wu, Doctor

  Beijing Children's Hospital

  PRINCIPAL INVESTIGATOR

• Jingyu Yan, Doctor

  North China University of Science and Technology

  PRINCIPAL INVESTIGATOR

• Sujun Gao, Doctor

  Bethune First Hospital of Jilin University

  PRINCIPAL INVESTIGATOR

• Wei Yang, Doctor

  Shengjing Hospital of China University

  PRINCIPAL INVESTIGATOR

• Rong Zhou, Doctor

  The Third People's Hospital of Chengdu

  PRINCIPAL INVESTIGATOR

• Ziqiang Yu, Doctor

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

• Yun Chen, Doctor

  Qianfoshan Hospital

  PRINCIPAL INVESTIGATOR

• Pingchong Lei, Doctor

  Henan Provincial People's Hospital

  PRINCIPAL INVESTIGATOR

• Yinsuo Zheng, Doctor

  Bao Ji Central Hospital

  PRINCIPAL INVESTIGATOR

• Peng Cheng, Doctor

  First Affiliated Hospital of Guangxi Medical University

  PRINCIPAL INVESTIGATOR

• Jianwen Xiao, Doctor

  Children's Hospital of Chongqing Medical University

  PRINCIPAL INVESTIGATOR

• Ruibin Huang, Doctor

  The First Affiliated Hospital of Nanchang University

  PRINCIPAL INVESTIGATOR

• Hailiang Li, Doctor

  The first affiliated hospital of jiangxi medical college

  PRINCIPAL INVESTIGATOR

• Shu Chen, Doctor

  The Second Affiliated Hospital of Chongqing Medical University

  PRINCIPAL INVESTIGATOR

• Xiong Zhang, Doctor

  Maoming City People's Hospital

  PRINCIPAL INVESTIGATOR

• Jingyu Zhang, Doctor

  The Second Hospital of Hebei Medical Hospital

  PRINCIPAL INVESTIGATOR

• Baolai Hua, Doctor

  Beijing Shijitan Hospital, Capital Medical University

  PRINCIPAL INVESTIGATOR

• Yanming Zhang, Doctor

  Huai'an Second People'Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 30, 2023
• First Posted: August 23, 2024
• Study Start: April 1, 2024
• Primary Completion (Estimated): October 15, 2026
• Study Completion (Estimated): December 15, 2026
• Last Updated: September 17, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)