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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

1Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China, 2Xiangya Hospital Central South University, Changsha, China, 3Nanfang Hospital Affiliated to Southern Medical University, Guangzhou, China, 4The Affiliated Hospital of Guizhou Medical University, Guizhou, China, 5Fujian Medical University Union Hospital, Fuzhou, China, 6The First Affiliated Hospital of Soochou University, Suzhou, China, 7Chengdu Women's & Children's Central Hospital, Chengdu, China, 8Qinghai Provincial People's Hospital, Qinghai, China, 9The First People's Hospital of Changzhou, Changzhou, China, 10The Second Affiliated Hospital of Guangzhou Medical University, Guangzhou, China, 11Anhui Provincial Hospital, Anhui, China, 12The Second Affiliated Hospital of Kunming Medical University, Kunmin, China

Abstract Number: PB1034

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

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
Total N 20 19 39
  Median (IQR) 2.190(4.260) 0.000(4.250) 2.080(4.250)
  Estimated mean (95% CI） 3.087(2.0533-4.6418) 2.855(1.7184-4.7437) 2.957(2.1321-4.1004)
Spontaneous bleeds N 13 10 23
  Median (IQR) 0.000(2.295) 0.000(2.110) 0.000(2.1659)
  Estimated mean (95% CI) 1.531(0.9475-2.4724) 1.152(0.6377-2.0817) 1.316(0.8964-1.9331)
Traumatic bleeds N 13 12 15
  Median (IQR) 0.000(2.160) 0.000(2.050) 0.000(2.140)
  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 2, 2023.

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