Hemophilia
Conditions
Brief summary
This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor.
Interventions
A Multiple-dose Design to Evaluate the Safety, Tolerability and Efficacy of STSP-0601 for Injection in hemophilia A or B patients with inhibitor.
Sponsors
Staidson (Beijing) Biopharmaceuticals Co., Ltd
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. 18 ≤age≤70 years of age,male. 2. Hemophilia A or B patients. (No less than 3 patients with hemophilia B) 3. Peak historical inhibitor titer ≥ 5 BU and apositive inhibitor test when enrolled. 4. Establish proper venous access. 5. There were at least 3 bleeding events that requiring treatment occurred in the past 6 months before screening (Only applicable to the on-demand treatment stage). 6. Agree to use adequate contraception to avoid pregnancy. Agree not to donate sperm or eggs. 7. Provide signed informed consent.
Exclusion criteria
1. Have any coagulation disorder other than hemophilia. 2. Plan to receive prophylactic treatment of coagulation factor during the trail. 3. Patients plan to receive Emicizumab during the trial. 4. 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. 5. Have a history of arterial and/or venous thrombotic events. 6. Platelet \<100×109/L. 7. Hemoglobin\<90g/L. 8. Severe liver or kidney disease. 9. Severe bleeding event occurred within 4 weeks before enrollment. 10. Accepted major operation or blood transfusion within 4 weeks before enrollment. 11. Have a known allergy to STSP-0601. 12. Pregnant, lactating, or blood pregnancy test positive female subjects 13. Participate in other clinical research within 4 weeks before enrollment(except for participating in prothrombin complex, FVII, FVIIa, FVIII, FIX trails). 14. Within 1 day prior to enrollment, FVII, FVIIa, tranexamic acid, and aminocaproic acid were used. Within 3 days prior to enrollment, prothrombin complex, FVIII, and FIX were used. Within 4 weeks prior to enrollment, treatment with amisulumab was received. 15. Patients not suitable for the trail according to the judgment of the investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of successfully treated bleeding episodes | 12 hours after first administration of study drug |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of successfully treated bleeding episodes | 8 hours after first administration of study drug | — |
| Excellent + good rate of treated bleeding episodes | 12 hours after first administration of study drug | Excellent: Pain and Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had fully eased. No additional infusion of study drug was required. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely eased , but had not completely disappeared. No additional infusion of study drug was required. |
| Number of doses required for effective hemostasis | 12 hours after first administration of study drug | — |
| Proportion of successfully treated first bleeding episodes | 12 hours after first administration of study drug | — |
| Time to complete/significant remission from first dose | 72 hours after last administration of study drug | — |
| Time to complete/significant remission from haematogenesis | 72 hours after last administration of study drug | — |
| Proportion of successfully treated Target joint bleeding episodes | 12 hours after first administration of study drug | — |
| Proportion of bleeding episodes received salvage treatment | 24 hours after first administration of study drug | — |
Countries
China
Outcome results
None listed