Clinical trial NCT05619926: Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hemophilia
Lead sponsor: Staidson (Beijing) Biopharmaceuticals Co., Ltd
Sponsor class: INDUSTRY
Collaborators: name: Beijing Novikang Medical Technology Co., LTD
First posted: 2022-11-17
Enrollment: 32
Sex: MALE
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of adverse events
timeFrame: From day 0 to up to day 4
measure: Proportion of successfully treated bleeding episodes
timeFrame: 8 hours after first administration of study drug
Secondary outcomes:
measure: Proportion of successfully treated bleeding episodes
timeFrame: 12 hours，24 hours after first administration of study drug
measure: Proportion of bleeding episodes received salvage treatment
timeFrame: Within 3 months after enrollment
Other outcomes:
Locations:
city: Chongqing
country: China
facility: The Second Affiliated Hospital of Chongqing Medical University
state: Chongqing
city: Lanzhou
country: China
facility: Lanzhou University First Hospita
state: Gansu
city: Shenzhen
country: China
facility: Shenzhen Second People's Hospital
state: Guangdong
city: Tangshan
country: China
facility: North China University of Technology Affiliated Hospital
state: Hebei
city: Nanyang
country: China
facility: The First Affiliated Hospital of Nanyang Medical College
state: Henan
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 Gulou Hospital
state: Jiangsu
city: Nanchang
country: China
facility: The First Affiliated Hospital of Nanchang University
state: Jiangxi
city: Xi'an
country: China
facility: Xi'an Central Hospital
state: Shanxi
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: Affiliation:Hospital of Hematology, Chinese Academy of Medical Sciences
name: Lei Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: