Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.

An Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.

This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.

Study Overview

• Status: Recruiting
• Conditions: Haemophilia A
• Intervention / Treatment: Drug: SCT800 prophylaxis and treatment for bleeding events

Detailed Description

SCT800 is a recombinant human coagulation factor VIII, which was on market in 2021 in China. The indication of SCT800 is prophylaxis and control of bleeding events in adult and children, and for the perioperative management of bleeding events. This study continued to evaluate the safety and efficacy of SCT800 in previously untreated patients with severe haemophilia A.

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Runhui Wu; Phone Number: +86-13370115037; Email: wurunhuigcp@163.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 000000
      • Recruiting
      • Beijing Children's Hospital
      • Contact: Runhui Wu; Phone Number: 86-13512078851; Email: wurunhuigcp@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male subjects with severe hemophilia A (FVIII:C < 1% in central laboratory tests)
• previously untreated with pdFVIII/rFVIII, including marketed SCT800 (those who have previously used ≤5EDs of blood products such as cryoprecipitate or fresh frozen plasma are acceptable).
• FVIII inhibitor negative
• informed consent.

Exclusion Criteria:

• Known allergy to recombinant coagulation factor VIII concentrate or any excipient; known allergy to bovine, rodent or hamster bovine;
• subjects with a history or family history of FVIII inhibitor formation;
• Clinical liver function test (ALT、AST) ≥ 5 ULN or clinical kidney function test (BUN，Cr) ≥2 ULN;
• Patients with other coagulation dysfunction diseases in addition to hemophilia A.
• International Normalized Ratio (INR) > 1.5.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SCT800 prophylaxis and control of bleeding events; For prophylaxis, 15-50 IU/kg SCT800 is intravenously. For control of bleeding events(BE), 10-20 IU/kg for mild BE, 15 -30IU/kg for moderate BE, 30-50IU/kg for severe BE.	Drug: SCT800 prophylaxis and treatment for bleeding events; For prophylaxis, 15-50 IU/kg SCT800 is intravenously. For control of bleeding events(BE), 10-20 IU/kg for mild BE, 15 -30IU/kg for moderate BE, 30-50IU/kg for severe BE.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of FVIII inhibitor	The percentage of FVIII inhibitor measured by Nijmegen-Bethesda	up to 50-100 Exposure Days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The percentage of adverse events		up to 50-100 exposure days
annualized bleeding rate	Annualized Bleeding Rate（ABR） can be calculated using the following formula: Number of bleeding events in efficacy evaluation period/(number of days in treatment period/365.25)	up to 50-100 exposure days
annualized joint bleeding rate	Annualized joint bleeding rate（AJBR） can be calculated using the following formula: Number of joint bleeding events during efficacy evaluation period/(number of days in treatment period/365.25).	up to 50-100 exposure days
consumption of SCT800	consumption of SCT800 per month and per year	up to 50-100 exposure days
hemostatic effect of SCT800	The investigator shall evaluate the hemostatic effect after the treatment of every bleeding event of subjects based on a four-point scale(excellent, good, moderate, not relieved).	up to 50-100 exposure days

Collaborators and Investigators

• Sponsor: Sinocelltech Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): April 26, 2024
• Primary Completion (Estimated): December 26, 2028
• Study Completion (Estimated): December 26, 2028

Study Registration Dates

• First Submitted: February 9, 2026
• First Submitted That Met QC Criteria: February 23, 2026
• First Posted (Actual): February 27, 2026

Study Record Updates

• Last Update Posted (Actual): February 27, 2026
• Last Update Submitted That Met QC Criteria: February 23, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: haemophilia A; SCT800; previously untreated patients
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A; Therapeutics
• Other Study ID Numbers: SCT800-A304

Drug and device information, study documents

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