Clinical trial NCT06289166: Safety and Efficacy of STSP-0601 in Adult Patients with Hemophilia a or B with Inhibitor
Official title: A Multi-center, Open-label, Phase Ⅱb Trial to Evaluate the Safety and Efficacy of STSP-0601 for Injection in Patients with Hemophilia with Inhibitor
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hemophilia
Lead sponsor: Staidson (Beijing) Biopharmaceuticals Co., Ltd
Sponsor class: INDUSTRY
First posted: 2024-03-01
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Proportion of successfully treated bleeding episodes
timeFrame: 12 hours after first administration of study drug
Secondary outcomes:
measure: Proportion of successfully treated first bleeding episodes
timeFrame: 12 hours after first administration of study drug
measure: Proportion of successfully treated bleeding episodes
timeFrame: 8 hours after first administration of study drug
measure: Excellent + good rate of treated bleeding episodes
timeFrame: 12 hours after first administration of study drug
measure: Excellent + good rate of treated bleeding episodes
timeFrame: 8 hours after first administration of study drug
measure: Number of doses required for effective hemostasis
timeFrame: 12 hours after first administration of study drug
measure: Proportion of bleeding episodes received salvage treatment
timeFrame: 24 hours after first administration of study drug
measure: Time to complete/significant remission from first dose
timeFrame: 72 hours after last administration of study drug
measure: Time to complete/significant remission from haematogenesis
timeFrame: 72 hours after last administration of study drug
measure: Proportion of successfully treated Target joint bleeding episodes
timeFrame: 12 hours after first administration of study drug
Other outcomes:
Locations:
city: Hefei
country: China
facility: Anhui Provincial Hospital
state: Anhui
city: Chongqing
country: China
facility: The Second Affiliated Hospital of Chongqing Medical University
state: Chongqing
city: Fuzhou
country: China
facility: Fujian Medical University Affiliated Union Medical College Hospital
state: Fujian
city: Lanzhou
country: China
facility: Lanzhou University First Hospital
state: Gansu
city: Guangzhou
country: China
facility: Southern Medical University Southern Hospital
state: Guangdong
city: Shenzhen
country: China
facility: Shenzhen Second People's Hospital
state: Guangdong
city: Guiyang
country: China
facility: Guizhou Medical University Affiliated Hospital
state: Guizhou
city: Harbin
country: China
facility: Harbin First Hospital Hematology Tumor Research Center
state: Heilongjiang
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
city: Changsha
country: China
facility: Xiangya Hospital of Central South University
state: Hunan
city: Nanjing
country: China
facility: Nanjing University School of Medicine Affiliated Gulou Hospital
state: Jiangsu
city: Nanchang
country: China
facility: The First Affiliated Hospital of Nanchang University
state: Jiangxi
city: Changchun
country: China
facility: The first hospital of Jilin University
state: Jilin
city: Shenyang
country: China
facility: Shengjing Hospital Affiliated to China Medical University
state: Liaoning
city: Qingdao
country: China
facility: Affiliated Hospital of Qingdao University
state: Shandong
city: Shanghai
country: China
facility: Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
state: Shanghai
city: Xi'an
country: China
facility: Xi'an Central Hospital
state: Shanxi
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
state: Sichuan
city: Tianjin
country: China
facility: Hospital of Hematology, Chinese Academy of Medical Sciences
state: Tianjin
city: Kunming
country: China
facility: The Second Affiliated Hospital of Kunming Medical University
state: Yunnan
Study officials:
affiliation: Hospital of Hematology, Chinese Academy of Medical Sciences
name: Lei Zhang, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: