Hemophilia a and BPhase 2NCT06747416
KN057 Multiple Dose Study in Patients with Hemophilia a or Hemophilia B with or Without Inhibitors
A Phase 2 study testing KN057 in people with Hemophilia a and B, run by Suzhou Alphamab Co., Ltd..
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years, men only
- Conditions the study is for
- Hemophilia a and B
- What is being tested?
- KN057
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- 2 Sep 2025to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male, 18-70 years old (including thresholds), body weight ≥40 kg and BMI <30 kg/m^2.
- Severe Hemophilia A or moderate-to-severe Hemophilia B (coagulation factor FVIII activity <1% or FIX activity ≤2%).
- Have ≥ 6 treated bleeding episodes within 24 weeks prior to screening (spontaneous and/or traumatic, excluding bleeding episodes related to surgery or traumatic operation).
- Patients without inhibitors must meet the following criteria: FVIII or FIX inhibitor test is negative during the screening period. Use coagulation factor replacement therapy for no less than 100 exposure days before screening.
- Patients with inhibitors must meet the following criteria: FVIII or FIX inhibitor test is positive during the screening period. Inhibitors level with high titer positive (≥5 BU/ml) or current low titer positive (<5 BU/ml) refractory to FVIII or FIX replacement and with FVIII or FIX recovery < 60% of expected within previous 12 months prior to screening.
Who cannot join
- Those with serious or poorly controlled chronic diseases or obvious systemic diseases: such as cardiovascular system, respiratory system, endocrine and metabolic system, urinary system, digestive system, autoimmune diseases, neurological diseases or psychiatric diseases, bacterial or viral infection, etc.; have previously received lipid-lowering therapy for hypertriglyceridemia or are currently receiving lipid-lowering therapy for hypertriglyceridemia.
- Have a history of other hereditary or acquired bleeding disorders other than Hemophilia A and Hemophilia B.
- Have symptoms and signs related to thromboembolic disease or are receiving thrombolytic/antithrombotic treatment; have a history of coronary atherosclerotic disease, arterial or venous thrombosis, or ischemic disease of important organs.
- Have high risk factors for thrombosis, including reduced activity of antithrombin III, protein S or protein C.
- When bleeding occurred in the past, rFVIIa was ineffective and (activated) prothrombin complex concentrate (PCC/aPCC) treatment must be used.
- Are undergoing or planning to undergo immune tolerance induction therapy.
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- Regular use of immunomodulatory therapy, such as regular infusion of immune globulin or regular use of hormones, is required.
- Those with allergies; those who are allergic to test drugs/similar drugs or excipients; those with a history of multiple allergies (two categories or more); those with a history of specific reactions, such as being allergic to heparin or having experienced heparin-induced thrombocytopenia.
- Hematological abnormalities: platelet count ≤ 100 × 10^9 /L; hemoglobin < 100g/L; fibrinogen level < lower limit of normal (LLN); prothrombin time > 1.5 times upper limit of normal (ULN).
- Abnormal liver and kidney function: alanine aminotransferase and/or aspartate aminotransferase >3 times ULN; lactate dehydrogenase >1.5 times ULN; total bilirubin >1.5 times ULN; serum creatinine, triglyceride > ULN; albumin <0.8 times LLN.
- Chronic active hepatitis B/C virus (HBV/HCV, HBV-DNA or HCV-RNA quantitative detection indicates viral activity); Human immunodeficiency virus (HIV) antibody positive; syphilis antibody positive; Have received antiviral treatment (only for HBV, HCV, and HIV) in the past 3 month, or have plans to undergo antiviral treatment within 28 weeks after the first dose.
- Have had major surgery within the past 3 months (determined by the investigator), or have elective surgery planned within 28 weeks after the first dose.
- Due to treatment needs, anti-fibrinolytic or platelet function-affecting drugs need to be used within 5 days before administration or within 28 weeks after the first administration, including medicines, such as aspirin, COX-1 and non-selective non-steroidal anti-inflammatory drugs (NSAIDs, except for acetaminophen), traditional herbal medicine or health supplements at investigator's discretion.
- Participated in clinical trials related to coagulation factors and received investigational drug treatment within the past 1 month; participated in any other drug clinical trials and received investigational drug treatment within the past 3 months.
- Have been vaccinated in the past month, or have a vaccination plan within 28 weeks after the first dose, including inactivated vaccine, live attenuated vaccine, recombinant protein vaccine, recombinant adenovirus vaccine, RNA vaccine, DNA vaccine, etc.
- Subjects with fertile mates are unable to use effective contraception during the study period and within 3 months after the last dose; effective contraceptive methods include: vasectomy, scientific use of male condoms, etc.
- Have had treatment of Emicizumab within 6 months prior to screening;
- Have had any prior treatment of genetic therapy for hemophilia.
- Those participants who drink heavily or are alcoholic within 24 weeks prior to screening or can't avoid heavy drinking during the trial. Heavy drinking means more than 7 units of alcohol per week, 1 unit=360ml of beer, or 45ml of liquor with 40% alc/vol, or 150ml of wine.
- Other factors that the investigator considers inappropriate for participation in this trial.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase II Multicenter, Open Label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics and PK/PD Profile of Multiple Subcutaneous Injection of KN057 in Male Patients with Severe Hemophilia a or Moderate-to-Severe Hemophilia B with or Without Inhibitors
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.
- Conditions
- Hemophilia a and B
- Being tested
- KN057
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KN057Drug
KN057 will be administered subcutaneously once a week.
| Study group | Role | What they receive |
|---|---|---|
| KN057 | Experimental | Not described Receives: Drug: KN057 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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-Ⅲ (AT-Ⅲ)
Measured: 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.
Measured: Week 0 up to Week 26
Number of participants with clinically significant changes in 12-lead electrocardiograms
Measured: Week 0 up to Week 26
Number of participants with clinically significant changes in vital signs
Heart Rate, Respirations, Temperature, Blood Pressure
Measured: Week 0 up to Week 26
Number of participants with clinically significant changes in physical examination Findings
Measured: Week 0 up to Week 26
Incidence and severity of injection site reaction
Measured: Week 0 up to Week 26
Secondary outcomes (12)
Plasma Concentration of KN057
Measured: Week 0 up to Week 26
Maximum Plasma Concentration (Cmax) of KN057
Measured: Week 0 up to Week 26
Time to Reach Maximum Plasma Concentration (Tmax) of KN057
Measured: Week 0 up to Week 26
Maximum observed KN057 concentration at steady-state
Measured: 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
Measured: Week 0 up to Week 26
Apparent Clearance (CL/F) of KN057
Measured: 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.
Measured: Week 0 up to Week 26
Changes of Total TFPI from baseline
TFPI: Tissue factor pathway inhibitor
Measured: Week 0 up to Week 26
Changes of Prothrombin fragment 1+2 (PF1+2) from baseline
Measured: Week 0 up to Week 26
The number and proportion of participants who produce anti-KN057 antibody
Measured: Week 0 up to Week 26
The number and proportion of participants who produce anti-KN057 neutralizing antibodies
Measured: Week 0 up to Week 26
Annualized Bleeding Rate (ABR)
ABR=number of bleeding episodes reported in visit days÷visit days×365.25
Measured: Week 0 up to Week 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2024
- Study start2 Sep 2025 (estimated)
- Primary completion2 Sep 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia a and BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Suzhou Alphamab Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06747416
- Sponsor's protocol ID
- KN057-A-202
- Status last verified
- Dec 2024
- First submitted
- 11 Dec 2024
- Last updated
- 24 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Multicenter, Open Label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics and PK/PD Profile of Multiple Subcutaneous Injection of KN057 in Male Patients with Severe Hemophilia a or Moderate-to-Severe Hemophilia B with or Without Inhibitors
- ClinicalTrials.gov ID
- NCT06747416
- Acronym / sponsor ID
- KN057-A-202
- Conditions
- Hemophilia a and B
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Suzhou Alphamab Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 2 Sep 2025 (estimated)
- Primary completion
- 2 Sep 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 24 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KN057 | Experimental | Drug: KN057 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male, 18-70 years old (including thresholds), body weight ≥40 kg and BMI <30 kg/m^2.
- Severe Hemophilia A or moderate-to-severe Hemophilia B (coagulation factor FVIII activity <1% or FIX activity ≤2%).
- Have ≥ 6 treated bleeding episodes within 24 weeks prior to screening (spontaneous and/or traumatic, excluding bleeding episodes related to surgery or traumatic operation).
- Patients without inhibitors must meet the following criteria: FVIII or FIX inhibitor test is negative during the screening period. Use coagulation factor replacement therapy for no less than 100 exposure days before screening.
- Patients with inhibitors must meet the following criteria: FVIII or FIX inhibitor test is positive during the screening period. Inhibitors level with high titer positive (≥5 BU/ml) or current low titer positive (<5 BU/ml) refractory to FVIII or FIX replacement and with FVIII or FIX recovery < 60% of expected within previous 12 months prior to screening.
Key exclusion criteria (20 listed)
- Those with serious or poorly controlled chronic diseases or obvious systemic diseases: such as cardiovascular system, respiratory system, endocrine and metabolic system, urinary system, digestive system, autoimmune diseases, neurological diseases or psychiatric diseases, bacterial or viral infection, etc.; have previously received lipid-lowering therapy for hypertriglyceridemia or are currently receiving lipid-lowering therapy for hypertriglyceridemia.
- Have a history of other hereditary or acquired bleeding disorders other than Hemophilia A and Hemophilia B.
- Have symptoms and signs related to thromboembolic disease or are receiving thrombolytic/antithrombotic treatment; have a history of coronary atherosclerotic disease, arterial or venous thrombosis, or ischemic disease of important organs.
- Have high risk factors for thrombosis, including reduced activity of antithrombin III, protein S or protein C.
- When bleeding occurred in the past, rFVIIa was ineffective and (activated) prothrombin complex concentrate (PCC/aPCC) treatment must be used.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KN057KN057 will be administered subcutaneously once a week. | Drug | KN057 |
Primary outcome
Incidence and severity of treatment emergent adverse events(TEAEs)
Time frame: Week 0 up to Week 26
TEAEs are adverse events occurred following the start of treatment or adverse events increasing in severity during treatment.
Number of participants with abnormal laboratory findings in Hematology
Time frame: Week 0 up to Week 26
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.
Number of participants with abnormal laboratory findings in Coagulation Function
Time frame: Week 0 up to Week 26
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-Ⅲ (AT-Ⅲ)
Number of participants with abnormal laboratory findings in Blood Biochemistry
Time frame: Week 0 up to Week 26
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.
Number of participants with clinically significant changes in 12-lead electrocardiograms
Time frame: Week 0 up to Week 26
Number of participants with clinically significant changes in vital signs
Time frame: Week 0 up to Week 26
Heart Rate, Respirations, Temperature, Blood Pressure
Number of participants with clinically significant changes in physical examination Findings
Time frame: Week 0 up to Week 26
Incidence and severity of injection site reaction
Time frame: Week 0 up to Week 26
12 secondary outcomes
Plasma Concentration of KN057
Time frame: Week 0 up to Week 26
Maximum Plasma Concentration (Cmax) of KN057
Time frame: Week 0 up to Week 26
Time to Reach Maximum Plasma Concentration (Tmax) of KN057
Time frame: Week 0 up to Week 26
Maximum observed KN057 concentration at steady-state
Time frame: 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
Time frame: Week 0 up to Week 26
Apparent Clearance (CL/F) of KN057
Time frame: Week 0 up to Week 26
Changes of Free Tissue factor pathway inhibitor (TFPI) from baseline
Time frame: Week 0 up to Week 26
Changes of Total TFPI from baseline
Time frame: Week 0 up to Week 26
Changes of Prothrombin fragment 1+2 (PF1+2) from baseline
Time frame: Week 0 up to Week 26
The number and proportion of participants who produce anti-KN057 antibody
Time frame: Week 0 up to Week 26
The number and proportion of participants who produce anti-KN057 neutralizing antibodies
Time frame: Week 0 up to Week 26
Annualized Bleeding Rate (ABR)
Time frame: Week 0 up to Week 20
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing KN057 in people with Hemophilia a and B, run by Suzhou Alphamab Co., Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 24 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yanrong Dong Master (+86 18914005458, yanrongdong@alphamab.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of treatment emergent adverse events(TEAEs) (time frame: Week 0 up to Week 26). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Dec 2024.