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The goal of this clinical trial is to evaluate the safety and efficacy of KN057 in adult patients with severe Hemophilia A (coagulation factor FVIII activity <1%) or moderate-to-severe Hemophilia B (FIX activity ≤2%). Participants will be administered subcutaneously with KN057 once a week for 20 weeks. KN057 works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly subcutaneous administration provides better convenience and compliance.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| KN057 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KN057 | Drug | KN057 will be administered subcutaneously once a week. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of treatment emergent adverse events(TEAEs) | TEAEs are adverse events occurred following the start of treatment or adverse events increasing in severity during treatment. | Week 0 up to Week 26 |
| Number of participants with abnormal laboratory findings in Hematology | Including white blood cells, red blood cells, lymphocyte count, neutrophil count, monocyte count, eosinophil count, basophil count, lymphocyte percentage, neutrophil percentage, monocyte percentage, eosinophil percentage, basophil percentage, hemoglobin, hematocrit, platelet count. | Week 0 up to Week 26 |
| Number of participants with abnormal laboratory findings in Coagulation Function | Including prothrombin time (PT), international normalized ratio (INR), activated partial thrombin time (APTT), thrombin time (TT), fibrinogen (FBG/FIB), D-dimer (D-Di), fibrin(ogen) degradation product (FDP), antithrombin-â…¢ (AT-â…¢) | Week 0 up to Week 26 |
| Number of participants with abnormal laboratory findings in Blood Biochemistry | Including total bilirubin (TBIL), indirect bilirubin (IBIL), direct bilirubin (DBIL), alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transpeptidase (GGT), alkaline phosphatase (ALP), total protein (TP), albumin (ALB), globulin, white globulin ratio , urea and/or urea nitrogen (BUN) (collected according to the specific test item at the clinical site), creatinine (Cr), uric acid, glucose, total cholesterol (TC), triglycerides (TG), low-density lipoprotein (LDL), high-density lipoprotein (HDL), lactate dehydrogenase (LDH), creatine kinase, sodium , potassium, chloride, calcium, C- reactive protein. | Week 0 up to Week 26 |
| Number of participants with clinically significant changes in 12-lead electrocardiograms |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Concentration of KN057 | Week 0 up to Week 26 | |
| Maximum Plasma Concentration (Cmax) of KN057 | Week 0 up to Week 26 | |
| Time to Reach Maximum Plasma Concentration (Tmax) of KN057 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yanrong Dong Master | Contact | +86 18914005458 | yanrongdong@alphamab.com |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| Week 0 up to Week 26 |
| Number of participants with clinically significant changes in vital signs | Heart Rate, Respirations, Temperature, Blood Pressure | Week 0 up to Week 26 |
| Number of participants with clinically significant changes in physical examination Findings | Week 0 up to Week 26 |
| Incidence and severity of injection site reaction | Week 0 up to Week 26 |
| Week 0 up to Week 26 |
| Maximum observed KN057 concentration at steady-state | Week 0 up to Week 26 |
| Area Under the Concentration-time Profile From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) of KN057 | Week 0 up to Week 26 |
| Apparent Clearance (CL/F) of KN057 | Week 0 up to Week 26 |
| Changes of Free Tissue factor pathway inhibitor (TFPI) from baseline | Tissue factor pathway inhibitor (TFPI) is an anticoagulant protein that inhibits early phases of the procoagulant response. | Week 0 up to Week 26 |
| Changes of Total TFPI from baseline | TFPI: Tissue factor pathway inhibitor | Week 0 up to Week 26 |
| Changes of Prothrombin fragment 1+2 (PF1+2) from baseline | Week 0 up to Week 26 |
| The number and proportion of participants who produce anti-KN057 antibody | Week 0 up to Week 26 |
| The number and proportion of participants who produce anti-KN057 neutralizing antibodies | Week 0 up to Week 26 |
| Annualized Bleeding Rate (ABR) | ABR=number of bleeding episodes reported in visit days÷visit days×365.25 | Week 0 up to Week 20 |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |