Safety of KN057 Prophylaxis in Patients With Haemophilia A or B

NCT07545395 | Recruiting Phase 3

• 1 other identifier: KN057-A-303
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

Brief Summary

The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the pharmacokinetic characteristics of the new and old processes KN057. The participants in Part PK will be randomly assigned to Old process Group or New process Group in a 1:1 ratio. The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks. The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks. The participants in Part non-PK will be non-randomized and treated with new process KN057 for 52 weeks prophylaxis after enrollment. Priority screening and enrollment of participants who have participated in the KN057-A-301 or KN057-A-302 study.

Conditions

Hemophilia A or B

Outcome Measures

Primary Outcomes (11)

• Incidence of TEAE.

  TEAE refers to 'treatment emergent adverse event'.

  Up to 12/26/56 weeks.

• Incidence of TEAE related to the experimental drug.

  Up to 12/26/56 weeks.

• Incidence of SAE.

  SAE refers to 'serious adverse event'.

  Up to 12/26/56 weeks.

• Incidence of thromboembolic events.

  Up to 12/26/56 weeks.

• Incidence of TMA and DIC.

  TMA refers to 'thrombotic microangiopathy'. DIC refers to 'disseminated intravascular coagulation'.

  Up to 12/26/56 weeks.

• Incidence of hypersensitivity type reactions.

  Up 12/26/56 weeks.

• Incidence of injection site reactions.

  Up to 12/26/56 weeks.

• Incidence of clinically significant laboratory value abnormalities.

  Up to 12/26/56 weeks.

• Number of participants with clinically significant changes from baseline in electrocardiograms.

  Up to 12/26/56 weeks.

• Number of participants with clinically significant changes from baseline in vital signs.

  Up to 12/26/56 weeks.

• Number of participants with clinically significant changes from baseline in physical exam.

  Up to 12/26/56 weeks.

Secondary Outcomes (10)

• The exposure levels of KN057 after the first administration in both the new and old processes.

  Up to 12 weeks.

• The steady-state trough concentrations of KN057 after the first administration in both the new and old processes.

  Up to 12 weeks.

• Incidence of anti-KN057 antibody (ADA) and neutralizing antibody (Nab).

  Up to 12/26/56 weeks.

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

  Up to 26/52 weeks.

• ABR calculated based on bleeding episodes, treated spontaneous bleeding episodes, treated joint bleeding episodes.

  Up to 26/52 weeks.

Study Arms (3)

Part PK # Old process Group

EXPERIMENTAL

The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks.

Drug: KN057

Part PK # New process Group

EXPERIMENTAL

The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks.

Drug: KN057

Part non-PK

EXPERIMENTAL

The participants in Part non-PK will be treated with new process KN057 for 52 weeks prophylaxis after enrollment.

Drug: KN057

Interventions

KN057 DRUG

KN057 will be administered subcutaneously once a week.

Part PK # New process Group; Part PK # Old process Group; Part non-PK

Eligibility Criteria

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

You may qualify if:

• Male, 12 to 65 years old at the time of signing informed consent, body weight ≥30 kg and BMI \<28 kg/m\^2 at screening.
• For participates with inhibitors: Tested positive for high-titer FVIII or FIX inhibitors (≥ 5 BU/mL) at screening; or tested positive for low-titer FVIII or FIX inhibitors (0.6 BU/mL or upper limit of normal \[ULN\] \< inhibitor titer \< 5 BU/mL) at screening, with ongoing treatment using bypassing agents (rFVIIa or PCC).
• For participates without inhibitors: 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 the inhibitor has turned negative for at least 5 years before screening and has not reappeared (no positive inhibitor was detected); Use coagulation factor replacement therapy for no less than 100 exposure days before screening.
• Participates with inhibitors agree to avoid using PCC for treatment when breakthrough bleeding occurred. Participates without inhibitors agree to be treated with standard half-life coagulation factors (FVIII or FIX) in the event of breakthrough bleeding.

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 atrial fibrillation, atherosclerotic diseases of important arteries, ischemic disease of important organs, vascular occlusive disease, autoimmune diseases with a high risk of thrombosis, or indwelling central venous catheter.
• 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

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Renchi Yang, Doctor

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

  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

  Xiangya Hospital of Central South University

  PRINCIPAL INVESTIGATOR

• Hongbo Cheng, Doctor

  Jiangxi Provincial People's Hopital

  PRINCIPAL INVESTIGATOR

• Yanping Song, Doctor

  Xi'an Central Hospital

  PRINCIPAL INVESTIGATOR

• Zeping Zhou, Doctor

  The Second Affiliated Hospital of Kunming Medical University

  PRINCIPAL INVESTIGATOR

• Jie Yin, Doctor

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

• Zhenyu Yan, Doctor

  North China University of Science and Technology

  PRINCIPAL INVESTIGATOR

• Yun Chen, Doctor

  Jinan Central Hospital

  PRINCIPAL INVESTIGATOR

• Yinsuo Zheng, Doctor

  Bao Ji Central Hospital

  PRINCIPAL INVESTIGATOR

• Shu Chen, Doctor

  The Second Affiliated Hospital of Chongqing Medical University

  PRINCIPAL INVESTIGATOR

• Ying Dong, Doctor

  Maoming City People's Hospital

  PRINCIPAL INVESTIGATOR

• Xiaoli Wu, Doctor

  The Second Hospital of Hebei Medical Hospital

  PRINCIPAL INVESTIGATOR

• Yanming Zhang, Doctor

  Huai'an Second People'Hospital

  PRINCIPAL INVESTIGATOR

• Miaoyong Zhu, Doctor

  Wenzhou People's Hospital

  PRINCIPAL INVESTIGATOR

• Haiping Yang, Doctor

  The First Affiliated Hospital of Henan University of Science and Technology

  PRINCIPAL INVESTIGATOR

• Qingyi Wang, Doctor

  The Fifth Affiliated Hospital of Anhui Medical

  PRINCIPAL INVESTIGATOR

• Wenqian Li, Doctor

  Qinghai People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yanrong Dong, Master

CONTACT

+86 18914005458; yanrongdong@alphamab.com

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: March 8, 2026
• First Posted: April 22, 2026
• Study Start: March 13, 2026
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): March 30, 2028
• Last Updated: April 22, 2026

Record last verified: 2026-04

Locations

CN (1)