Haemophilia APhase 3NCT07437404
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
A Phase 3 study testing SCT800 prophylaxis and treatment for bleeding events in people with Haemophilia A, run by Sinocelltech Ltd. at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 6 years, men only
- Conditions the study is for
- Haemophilia A
- What is being tested?
- SCT800 prophylaxis and treatment for bleeding events
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteChina
- Study dates
- 26 Apr 2024to 26 Dec 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Beijing children's hospital
Beijing, Beijing Municipality, 000000
Runhui Wu, 86-13512078851, wurunhuigcp@163.com
About this study
Official title: 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.
Read the 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.
- Conditions
- Haemophilia A
- Being tested
- SCT800 prophylaxis and treatment for bleeding events
- Keywords
- SCT800, haemophilia A, previously untreated patients
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SCT800 prophylaxis and treatment for bleeding eventsDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| SCT800 prophylaxis and control of bleeding events | Experimental | 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. Receives: Drug: SCT800 prophylaxis and treatment for bleeding events |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of FVIII inhibitor
The percentage of FVIII inhibitor measured by Nijmegen-Bethesda
Measured: up to 50-100 Exposure Days
Secondary outcomes (5)
The percentage of adverse events
Measured: 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)
Measured: 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).
Measured: up to 50-100 exposure days
consumption of SCT800
consumption of SCT800 per month and per year
Measured: 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).
Measured: up to 50-100 exposure days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start26 Apr 2024
- Primary completion26 Dec 2028 (estimated)
- Study completion26 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Model Description
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sinocelltech Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07437404
- Sponsor's protocol ID
- SCT800-A304
- Status last verified
- Feb 2026
- First submitted
- 9 Feb 2026
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No