SCT800 for preventing and treating bleeds in previously untreated severe 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 test tries SCT800, a recombinant factor VIII, as regular prevention and to treat bleeds in male patients with severe hemophilia A who haven't had prior factor VIII treatment.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: N/A to 6 Years
• Sex: Male
• Sponsor: Sinocelltech Ltd. (Industry-sponsored)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07437404 on ClinicalTrials.gov

What this trial studies

This open-label, uncontrolled Phase 3 study gives SCT800 as prophylaxis and to treat bleeding episodes in males with severe hemophilia A who are previously untreated. Participants must have FVIII activity <1% and be inhibitor-negative, with limited prior exposure to plasma products allowed. The study monitors safety (including inhibitor development, liver and kidney tests, and allergic reactions) and measures bleeding control on prophylaxis and response to treatment of bleeds. The trial is sponsored by Sinocelltech and conducted at Beijing Children's Hospital to continue collecting data on SCT800 in this population after its 2021 approval in China.

Who should consider this trial

Good fit: Male patients with severe hemophilia A (FVIII:C <1%), no prior exposure to factor VIII products (acceptable up to ≤5 exposure days to certain plasma products), FVIII inhibitor-negative, and able to give informed consent are ideal candidates.

Not a fit: Patients with existing FVIII inhibitors, significant liver or kidney dysfunction, other coagulation disorders, or known allergy to recombinant FVIII or its excipients are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, SCT800 could offer an effective option to prevent bleeds and manage bleeding episodes in previously untreated severe hemophilia A patients.

How similar studies have performed: Recombinant factor VIII products have a history of successful use for prophylaxis and bleed control, and SCT800 was approved in China in 2021, but data specifically in previously untreated patients remain limited.

Eligibility criteria

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.

Where this trial is running

Beijing, Beijing Municipality

• Beijing children's hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Runhui Wu

Conditions Haemophilia A; SCT800; haemophilia A; previously untreated patients