Safety and Efficacy of STSP-0601 in Adult Patients with Hemophilia a or B with Inhibitor

A Multi-center, Open-label, Phase Ⅱb Trial to Evaluate the Safety and Efficacy of STSP-0601 for Injection in Patients with Hemophilia with Inhibitor

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 inhibitor.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230000
      • Anhui Provincial Hospital
  • Chongqing
    • Chongqing, Chongqing, China, 400000
      • The Second Affiliated Hospital of Chongqing Medical University
  • Fujian
    • Fuzhou, Fujian, China, 350005
      • Fujian Medical University Affiliated Union Medical College Hospital
  • Gansu
    • Lanzhou, Gansu, China, 730000
      • Lanzhou University First Hospital
  • Guangdong
    • Guangzhou, Guangdong, China, 510000
      • Southern Medical University Southern Hospital
    • Shenzhen, Guangdong, China, 518000
      • Shenzhen Second People's Hospital
  • Guizhou
    • Guiyang, Guizhou, China, 550000
      • Guizhou Medical University Affiliated Hospital
  • Heilongjiang
    • Harbin, Heilongjiang, China, 150000
      • Harbin First Hospital Hematology Tumor Research Center
  • Henan
    • Zhengzhou, Henan, China, 450000
      • Henan Cancer Hospital
  • Hunan
    • Changsha, Hunan, China, 410000
      • Xiangya Hospital of Central South University
  • Jiangsu
    • Nanjing, Jiangsu, China, 210000
      • Nanjing University School of Medicine Affiliated Gulou Hospital
  • Jiangxi
    • Nanchang, Jiangxi, China, 330038
      • The First Affiliated Hospital of NanChang University
  • Jilin
    • Changchun, Jilin, China, 130000
      • The First Hospital of Jilin University
  • Liaoning
    • Shenyang, Liaoning, China, 110000
      • Shengjing Hospital affiliated to China Medical University
  • Shandong
    • Qingdao, Shandong, China, 266000
      • Affiliated Hospital of Qingdao University
  • Shanghai
    • Shanghai, Shanghai, China, 200000
      • Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
  • Shanxi
    • Xi'an, Shanxi, China, 710000
      • Xi'an Central Hospital
  • Sichuan
    • Chengdu, Sichuan, China, 610000
      • West China Hospital of Sichuan University
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Hospital of Hematology, Chinese Academy of Medical Sciences
  • Yunnan
    • Kunming, Yunnan, China, 650000
      • The Second Affiliated Hospital of Kunming Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 18 ≤age≤70 years of age,male.
2. Hemophilia A or B patients. (No less than 3 patients with hemophilia B)
3. Peak historical inhibitor titer ≥ 5 BU and apositive inhibitor test when enrolled.
4. Establish proper venous access.
5. There were at least 3 bleeding events that requiring treatment occurred in the past 6 months before screening (Only applicable to the on-demand treatment stage).
6. Agree to use adequate contraception to avoid pregnancy. Agree not to donate sperm or eggs.
7. Provide signed informed consent.

Exclusion Criteria:

1. Have any coagulation disorder other than hemophilia.
2. Plan to receive prophylactic treatment of coagulation factor during the trail.
3. Patients plan to receive Emicizumab during the trial.
4. 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.
5. Have a history of arterial and/or venous thrombotic events.
6. Platelet <100×109/L.
7. Hemoglobin<90g/L.
8. Severe liver or kidney disease.
9. Severe bleeding event occurred within 4 weeks before enrollment.
10. Accepted major operation or blood transfusion within 4 weeks before enrollment.
11. Have a known allergy to STSP-0601.
12. Pregnant, lactating, or blood pregnancy test positive female subjects
13. Participate in other clinical research within 4 weeks before enrollment(except for participating in prothrombin complex, FVII, FVIIa, FVIII, FIX trails).
14. Within 1 day prior to enrollment, FVII, FVIIa, tranexamic acid, and aminocaproic acid were used. Within 3 days prior to enrollment, prothrombin complex, FVIII, and FIX were used. Within 4 weeks prior to enrollment, treatment with amisulumab was received.
15. 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: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Consecutive doses of STSP-0601	Drug: STSP-0601 for Injection; A Multiple-dose Design to Evaluate the Safety, Tolerability and Efficacy of STSP-0601 for Injection in hemophilia A or B patients with inhibitor.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Proportion of successfully treated bleeding episodes	12 hours after first administration of study drug

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of successfully treated bleeding episodes		8 hours after first administration of study drug
Proportion of successfully treated first bleeding episodes		12 hours after first administration of study drug
Excellent + good rate of treated bleeding episodes	Excellent: Pain and Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had fully eased. No additional infusion of study drug was required. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely eased , but had not completely disappeared. No additional infusion of study drug was required.	12 hours after first administration of study drug
Excellent + good rate of treated bleeding episodes	Excellent: Pain and Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had fully eased. No additional infusion of study drug was required. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely eased , but had not completely disappeared. No additional infusion of study drug was required.	8 hours after first administration of study drug
Number of doses required for effective hemostasis		12 hours after first administration of study drug
Proportion of bleeding episodes received salvage treatment		24 hours after first administration of study drug
Time to complete/significant remission from first dose		72 hours after last administration of study drug
Time to complete/significant remission from haematogenesis		72 hours after last administration of study drug
Proportion of successfully treated Target joint bleeding episodes		12 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): March 18, 2024
• Primary Completion (Actual): October 17, 2024
• Study Completion (Actual): October 17, 2024

Study Registration Dates

• First Submitted: February 23, 2024
• First Submitted That Met QC Criteria: February 23, 2024
• First Posted (Actual): March 1, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 17, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A
• Other Study ID Numbers: STSP-0601-04

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
• product manufactured in and exported from the U.S.: No