Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor

Study Overview
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Brief Summary
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This study will assess the safety,efficacy,pharmacokinetics and pharmacodynamics of multiple-dose of STSP-0601 at two dose levels for the treatment of bleeding episodes in hemophilia A or B patients without inhibitor.

Official Title
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A Multi-center, Open-label, Multiple-dose Design, Phase 2 Trial to Evaluate the Safety and Efficacy of STSP-0601 for Injection in Patients With Hemophilia Without Inhibitor

Conditions
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Hemophilia

Intervention / Treatment
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* Drug: STSP-0601 for Injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 ≤age≤70 years of age,male; Hemophilia A or B patients; A bleeding event requiring treatment occurred (Only applicable to the on-demand treatment stage); Factor VIII or IX level <2%, inhibitor titer < 0.6 BU; There were at least 2 bleeding events that requiring treatment occurred in the past 6 months before screening (Only the second stage is applicable); Establish proper venous access; Agree to use adequate contraception to avoid pregnancy; Provide signed informed consent. Exclusion Criteria: Have any coagulation disorder other than hemophilia; Plan to receive prophylactic treatment of coagulation factor during the trail; 4 weeks before screening, receive prophylactic treatment of amesetuzumab or plan to receive prophylactic treatment of amesetuzumab during the trial; Patients received anticoagulant or antifibrinolytic therapy 7 days before enrollment or plan to receive these drugs during the trial; Patients received anticoagulation therapy (such as coagulation factor replacement therapy, prothrombin complex, plasma, etc.) 7 days before enrollment; Have a history of arterial and/or venous thrombotic events; Platelet <100×109/L; Hemoglobin<90g/L; Severe liver or kidney disease; Severe bleeding event occurred within 4 weeks before enrollment; Accepted major operation or blood transfusion within 4 weeks before enrollment; HIV positive; Have a known allergy to STSP-0601; Participate in other clinical research within 4 weeks before enrollment(except for participating in prothrombin complex, FVII, FVIIa, FVIII, FIX trails); Patients not suitable for the trail according to the judgment of the investigators.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Consecutive doses of low-dose of STSP-0601<br> | Drug: STSP-0601 for Injection<br>* Intravenous Injection<br>|
| Experimental: Consecutive doses of high-dose of STSP-0601<br> | Drug: STSP-0601 for Injection<br>* Intravenous Injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events |  | From day 0 to up to day 4 | 
| Proportion of successfully treated bleeding episodes |  | 8 hours after first administration of study drug | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of successfully treated bleeding episodes |  | 12 hours，24 hours after first administration of study drug | 
| Proportion of bleeding episodes received salvage treatment |  | Within 3 months after enrollment |