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

A Phase 1,Multi-center, Randomized, Open, Single-dose Escalation Design to Evaluate the Safety, Tolerability and Pharmacodynamics of STSP-0601 for Injection in Patients With Inhibitory Hemophilia

This study will assess the pharmacokinetics and pharmacodynamics of STSP-0601 at six dose levels. The results will help identify the most optimal doses to treat bleedings in hemophilia patients with inhibitors.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Tianjin, China
    • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 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.
• Peak historical inhibitor titer ≥ 5 BU and a positive inhibitor test when enrolled.
• Establish proper venous access.
• 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 an active, ongoing bleeding for which the patient is being treated, or treatment for a bleeding was stopped within 7d of the time of study drug administration.
• Have a history of arterial and/or venous thromboembolic events.
• Have platelet count <100,000/mL.
• Severe liver or kidney disease.
• Accept major operation or blood transfusion within 1 month of the time of screening.
• HIV antibody positive.
• Have a known allergy to Blood product.
• Participate in other clinical research within 1 month 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

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: A single lowest dose of treatment group	Drug: STSP-0601 for Injection; Subjects will receive one single lowest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single intermediate dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single high dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single highest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low-intermediate dose of STSP-0601 for Injection following protocol requirements
Experimental: A single low dose of treatment group	Drug: STSP-0601 for Injection; Subjects will receive one single lowest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single intermediate dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single high dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single highest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low-intermediate dose of STSP-0601 for Injection following protocol requirements
Experimental: A single low-intermediate dose of treatment group	Drug: STSP-0601 for Injection; Subjects will receive one single lowest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single intermediate dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single high dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single highest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low-intermediate dose of STSP-0601 for Injection following protocol requirements
Experimental: A single intermediate dose of treatment group	Drug: STSP-0601 for Injection; Subjects will receive one single lowest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single intermediate dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single high dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single highest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low-intermediate dose of STSP-0601 for Injection following protocol requirements
Experimental: A single high dose of treatment group	Drug: STSP-0601 for Injection; Subjects will receive one single lowest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single intermediate dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single high dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single highest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low-intermediate dose of STSP-0601 for Injection following protocol requirements
Experimental: A single highest dose of treatment group	Drug: STSP-0601 for Injection; Subjects will receive one single lowest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single intermediate dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single high dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single highest dose of STSP-0601 for Injection following protocol requirements; Drug: STSP-0601 for Injection; Subjects will receive one single low-intermediate dose of STSP-0601 for Injection following protocol requirements

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of subjects of adverse events	Baseline to Day 8
Number of subjects With Significant Abnormal Physical Examination	Baseline to Day 8
Number of subjects of Significant Abnormal Vital Signs Findings	Baseline to Day 8
Number of Participants With Significant Abnormal Laboratory Values	Baseline to Day 8
Number of subjects With Significant Abnormal Electrocardiography (ECG) Findings	Baseline,1 hour,4 hour,48 hour,Day 8

Secondary Outcome Measures

Outcome Measure	Time Frame
Activated partial thromboplastin time	Baseline to 24 hours
Thrombin production (TG) peak	Baseline to 24 hours

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): January 15, 2020
• Primary Completion (Actual): April 19, 2021
• Study Completion (Actual): April 19, 2021

Study Registration Dates

• First Submitted: February 5, 2021
• First Submitted That Met QC Criteria: February 5, 2021
• First Posted (Actual): February 10, 2021

Study Record Updates

• Last Update Posted (Actual): September 19, 2024
• Last Update Submitted That Met QC Criteria: September 12, 2024
• Last Verified: July 1, 2021

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-0601-01

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