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

NCT05027230 | Completed Phase 1

• 1 other identifier: STSP-0601-02
• Study Type: interventional
• Target: 77
• Locations: 1 country
• Sites: 1

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 inhibitors.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (1)

• Proportion of bleeding episodes with clinical relief of signs and symptoms

  4 hours、8 hours、24 hours after first administration of study drug

Study Arms (2)

Consecutive doses of low-dose of STSP-0601

EXPERIMENTAL

Drug: STSP-0601 for Injection

Consecutive doses of high-dose of STSP-0601

EXPERIMENTAL

Drug: STSP-0601 for Injection

Interventions

STSP-0601 for Injection DRUG

A multiple-dose design to evaluate the safety,tolerability and efficacy of two doses of STSP-0601 for injection in patients with inhibitory hemophilia

Consecutive doses of low-dose of STSP-0601

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Staidson (Beijing) Biopharmaceuticals Co., Ltd: lead

Study Sites (1)

Hospital of Hematology, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

Related Publications (1)

• Liu W, Xue F, Fu R, Ding B, Li M, Sun T, Chen Y, Liu X, Ju M, Dai X, Wu Q, Zhou Z, Yu J, Wang X, Zhu Q, Zhou H, Yang R, Zhang L. Preclinical studies of a factor X activator and a phase 1 trial for hemophilia patients with inhibitors. J Thromb Haemost. 2023 Jun;21(6):1453-1465. doi: 10.1016/j.jtha.2023.01.040. Epub 2023 Feb 14.

  PMID: 36796484 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Injections

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Lei Zhang, Ph.D

  Hospital of Hematology, Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2021
• First Posted: August 30, 2021
• Study Start: October 12, 2021
• Primary Completion: July 15, 2023
• Study Completion: July 29, 2023
• Last Updated: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)