Abstract Number: PB1034
Meeting: ISTH 2020 Congress
Background: SCT800 is a B-domain deleted recombinant factor VIII (rFVIII) product for hemophilia A.
Aims: This study is to assess efficacy, safety and pharmacokinetics of SCT800 for prophylaxis and treatment of bleeds in patients with severe hemophilia A.
Methods: This is a multi-center, phase 3, open-label trial( clinicaltrials.gov: NCT03815318), which was approved by the clinical committee of Hospital of Blood Disease,CAMS&PUMC. This study was sponsored by Sinocelltech Inc. All subjects must sign the Informed Consent Form before screening. In this clinical trial, males aged 12-65 years with FVIII < 1% and ≥150 exposure days (EDs) to any FVIII containing product and no history of inhibitors. Eligible patients received SCT800 25-50 IU/kg every other day or three times a week for approximately 24 weeks. Primary efficacy outcome was annualized bleeding rate (ABR).
Results: The analysis showed that a total of 73 patients were enrolled, and 71 patients (97.3%) completed the trial. The patients received prophylaxis with SCT 800 had a mean of 77.4 EDs during the trial. The overall median annualized bleeding rate was 2.080 (interquartile range:4.250) bleeds/patient/year. Based on patient reports and investigator’s judgements, the success rate (defined as ‘excellent’ or ‘good’ hemostatic response) for treatment of bleeding episodes was 87%. 72% of bleeds resolved with one injection and 84% with up to two injections. Treatment-related adverse event (AE) incidence was 4.1% and there was one severe adverse event probably unrelated with study drug overall. No patient developed inhibitors. This is the preliminary analysis and the pharmacokinetic parameters are not obtained now.
|Adolescents 12-18 years 32 patients||Adult ≥18 years 41 patients||All patients 73 patients|
|Estimated mean (95% CI）||3.087(2.0533-4.6418)||2.855(1.7184-4.7437)||2.957(2.1321-4.1004)|
|Estimated mean (95% CI)||1.531(0.9475-2.4724)||1.152(0.6377-2.0817)||1.316(0.8964-1.9331)|
|Estimated mean (95% CI)||1.180(0.6577-2.1179)||1.599(0.8231-3.1048)||1.415(0.8971-2.2315)|
[Table1. Annualized bleeding rates.]
Conclusions: These results in adult Previous Treated Patients indicate that SCT800 is effective and safe for the prevention and treatment of bleeds in adults with severe hemophilia A.
To cite this abstract in AMA style:Liu W, Zhao X, Xue F, Sun J, Zeng X, Yang F, Yu Z, Xu M, Li W, Gu W, Feng Y, Zheng C, Bi H, Yang R. A Multicenter Open-label Single Arm Trial to Evaluate Safety and Efficacy and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Patients with Severe Hemophilia A under Prophylaxis Therapy [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-multicenter-open-label-single-arm-trial-to-evaluate-safety-and-efficacy-and-pharmacokinetics-of-recombinant-human-coagulation-factor-viii-sct800-in-previously-treated-patients-with-severe-hemophil/. Accessed October 26, 2020.
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