New weekly shot aims to prevent bleeding in hemophilia patients
NCT ID NCT07545395
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 3 study is testing a drug called KN057, given as a weekly injection under the skin, to prevent bleeding in people with hemophilia A or B (with or without inhibitors). The trial will enroll 70 males aged 12 to 65 and will monitor safety and how well the drug works over 52 weeks. Some participants will also compare two different manufacturing processes of the drug.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- KN057 (a drug given as a weekly injection under the skin)
- What this could lead to
- If successful, this could provide a new, convenient once-weekly preventive treatment to reduce bleeding episodes in people with hemophilia A or B, including those with inhibitors.
- What could go wrong
- This is a Phase 3 trial, but it is still early to know if KN057 works better than existing options. Potential risks include allergic reactions, injection site issues, and rare but serious blood clotting events.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 70 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2026
- Expected to finish
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Mar 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 to 65 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. 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. 2. 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. 3. 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. Exclusion Criteria: 1. Have serious or poorly controlled chronic diseases or obvious systemic diseases. 2. Have a history of thromboembolic disease, or currently have symptoms or signs related to thromboembolic disease or being treated with thrombolytic/antithrombotic therapy. 3. 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. 4. Known or suspected hypersensitivity to any constituent of the trial product or related products. 5. Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study. 6. Used Emicizumab treatment within 6 months before screening. 7. Have received any gene therapy for hemophilia in the past. 8. 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
RECRUITINGTianjin, Tianjin Municipality, 300020, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a 12-Week exercise programme build strength safely in boys with hemophilia?
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Gene Editing's lasting impact: a 10-Year safety watch
- Can a single gene shot free hemophilia b patients from regular infusions?
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth