A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

A Phase Ib/II,Multi-center, Open, Multiple-dose Design to Evaluate the Safety,Tolerability and Efficacy of STSP-0601 for Injection in Patients With Inhibitory Hemophilia

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 inhibitors.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 77
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Hospital of Hematology, Chinese Academy of Medical Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 61 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 18 years old ≤age≤65 years of age,male.
• Hemophilia A or B patients with inhibitors.
• Received hemostatic treatment with investigational drug, or to undergo minor surgical or other invasive procedures (Only for phase 2. Minor surgical procedures: procedures without general anesthesia and the wounds are easy to observe).
• Peak historical inhibitor titer ≥ 5 BU and a positive inhibitor test when enrolled.
• 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 A or B.
• Treat with prophylactic treatment of coagulation factor.
• Treat with anticoagulant within 7d of the time of study drug administration.
• Have a history of arterial and/or venous thromboembolic events.
• Have platelet <100,000/mL,hemoglobin<90g/L.
• Severe liver or kidney disease.
• Bleeding in the central nervous system or throat before screening.
• Accept major operation or blood transfusion within 1 month of the time of screening.
• HIV positive with current CD4+ count of less than 200/μl.
• Have a known allergy to Blood product.
• Participate in other clinical research within 1 month of the time of study drug administration.
• Treat with coagulant within 7d of the time of study drug administration.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: 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; A multiple-dose design to evaluate the safety,tolerability and efficacy of two doses of STSP-0601 for injection in patients with inhibitory hemophilia
Experimental: Consecutive doses of high-dose of STSP-0601	Drug: STSP-0601 for Injection; A multiple-dose design to evaluate the safety,tolerability and efficacy of two doses of STSP-0601 for injection in patients with inhibitory hemophilia

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events	From day 0 to up to day 8
Proportion of successfully treated bleeding episodes	24 hours after administration of study drug

Secondary Outcome Measures

Outcome Measure	Time Frame
Proportion of bleeding episodes with clinical relief of signs and symptoms	4 hours、8 hours、24 hours after first administration of study drug

Collaborators and Investigators

• Sponsor: Staidson (Beijing) Biopharmaceuticals Co., Ltd
• Investigators: Principal Investigator: Lei Zhang, Ph.D, Hospital of Hematology, Chinese Academy of Medical Sciences

Study record dates

Study Major Dates

• Study Start (Actual): October 12, 2021
• Primary Completion (Actual): July 15, 2023
• Study Completion (Actual): July 15, 2023

Study Registration Dates

• First Submitted: August 25, 2021
• First Submitted That Met QC Criteria: August 25, 2021
• First Posted (Actual): August 30, 2021

Study Record Updates

• Last Update Posted (Estimated): November 13, 2023
• Last Update Submitted That Met QC Criteria: November 10, 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-0601-02

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