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

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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia
• Intervention / Treatment: Drug: STSP-0601 for Injection

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Yanli Wang; Phone Number: 010-67519614; Email: wangyanli@staidson.com

Study Locations

• China
  • Chongqing
    • Chongqing, Chongqing, China, 400000
      • The Second Affiliated Hospital of Chongqing Medical University
  • Gansu
    • Lanzhou, Gansu, China, 730000
      • Lanzhou University First Hospita
  • Guangdong
    • Shenzhen, Guangdong, China, 511285
      • Shenzhen Second People's Hospital
  • Hebei
    • Tangshan, Hebei, China, 063000
      • North China University of Technology Affiliated Hospital
  • Henan
    • Nanyang, Henan, China, 473000
      • The First affiliated Hospital of Nanyang Medical College
    • Zhengzhou, Henan, China, 450000
      • Henan Cancer Hospital
  • Hunan
    • Changsha, Hunan, China, 410000
      • Xiangya Hospital of Central South University
  • Jiangsu
    • Nanjing, Jiangsu, China, 210000
      • Nanjing Gulou Hospital
  • Jiangxi
    • Nanchang, Jiangxi, China, 330038
      • The First Affiliated Hospital Of Nanchang University
  • Shanxi
    • Xi'an, Shanxi, China, 710000
      • Xi'an Central Hospital
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Hospital of Hematology, Chinese Academy of Medical Sciences
      • Contact: Lei Zhang; Phone Number: 022-23909095; Email: zhanglei1@ihcams.ac.cn
  • Yunnan
    • Kunming, Yunnan, China, 650000
      • The Second Affiliated hospital of Kunming Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Consecutive doses of low-dose of STSP-0601	Drug: STSP-0601 for Injection; Intravenous Injection
Experimental: Consecutive doses of high-dose of STSP-0601	Drug: STSP-0601 for Injection; Intravenous Injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Staidson (Beijing) Biopharmaceuticals Co., Ltd
• Collaborators: Beijing Novikang Medical Technology Co., LTD
• Investigators: Principal Investigator: Lei Zhang, MD, Affiliation:Hospital of Hematology, Chinese Academy of Medical Sciences

Study record dates

Study Major Dates

• Study Start (Actual): January 9, 2023
• Primary Completion (Actual): August 25, 2023
• Study Completion (Actual): August 25, 2023

Study Registration Dates

• First Submitted: November 9, 2022
• First Submitted That Met QC Criteria: November 9, 2022
• First Posted (Actual): November 17, 2022

Study Record Updates

• Last Update Posted (Estimated): January 1, 2024
• Last Update Submitted That Met QC Criteria: December 29, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: STSP-0601-03

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No