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

NCT06312475 | Active Not Recruiting Phase 3

• 1 other identifier: KN057-A-301
• Study Type: interventional
• Target: 53
• 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 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

Outcome Measures

Primary Outcomes (1)

• Annualized bleeding rate (ABR) calculated based on treated spontaneous and traumatic bleeding episodes in Arm 1 and Arm 2.

  Treated bleeding refers to the use of bypass agents and/or coagulation factors for hemostatic treatment of the bleeding.

  From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

Secondary Outcomes (19)

• ABR calculated based on bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes, and treated target joint bleeding respectively in Arm 1 and Arm 2.

  From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

• ABR calculated based on bleeding episodes and treated bleeding episodes respectively in Arm 2.

  From Day 183 (the beginning of the extension period) to Day 365 (the end of the extension period), approximately 26 weeks in total

• ABR calculated based on bleeding episodes, treated bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes, and treated target joint bleeding respectively in Arm 1.

  From Day 1 (the beginning of the main trial) to Day 365 (the end of the extension period), approximately 52 weeks in total

• Change from baseline in Hemophilia Joint Health Score (HJHS) scores in Arm 1 and Arm 2.

  From Day 1 (the beginning of the main trial) to Day 183 (the end of the main trial), approximately 26 weeks in total

• Change in HJHS scores from baseline in Arm 1 and from the 26th week in Arm 2.

  From Day 1 (the beginning of the main trial) to Day 365 (the end of the extension period), approximately 52 weeks in total

Study Arms (2)

Arm 1: KN057 Prophylaxis

EXPERIMENTAL

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.

Drug: KN057

Arm 2: No Prophylaxis

EXPERIMENTAL

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.

Drug: KN057

Interventions

KN057 DRUG

KN057 will be administered subcutaneously once a week.

Arm 1: KN057 Prophylaxis; Arm 2: No Prophylaxis

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 ≥25 kg and BMI \<28 kg/m\^2 at screening;
• 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;
• ≥6 treated bleeding episodes within 26 weeks before screening;
• Have not used TFPI antibody drugs before;
• Be able and agree to elute prior drugs for the treatment of hemophilia.

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;
• 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);
• Ongoing or planned Immune Tolerance Induction treatment;
• When bleeding occurred in the past, rFVIIa was ineffective and PCC treatment must be used;
• 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.

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

• Ziqiang Yu, Doctor

  The First Affiliated Hospital of Soochow University

  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

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: March 15, 2024
• Study Start: January 9, 2024
• Primary Completion: October 15, 2025
• Study Completion: December 15, 2025
• Last Updated: September 17, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)