VPB0697 - Efficacy, Safety and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Children with Severe Hemophilia A.

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: Virtual

Presenting Author(s)

RW

Runhui Wu

Director and Physician in Chief
Beijing Children’s Hospital, Capital Medical University, Beijing, China
Beijing, Beijing, China (People's Republic)

Co-Author(s)

XW

Xiaoling Wang

Chief pharmacist
Beijing Children’s Hospital, Capital Medical University, Beijing, China
Beijing, Beijing, China (People's Republic)
XZ

Xielan Zhao

Chief physician
Xiangya Hospital Central South University
Changsha, Henan, China (People's Republic)
YC

Yanli Chen

Chief physician
Shanxi provincial children’s hospital
taiyuan, Shanxi, China (People's Republic)
ZZ

Zeping Zhou

Chief physician
The Second Affiliated Hospital of Kunming Medical University
kunming, Yunnan, China (People's Republic)
JS

Jing Sun

Chief Physician
Nanfang Hospital of Southern Medical University, Guangzhou, P.R. China
Tianjin, Tianjin, China (People's Republic)
MX

Ming Xu

Chief physician
Chengdu Women’s & Children’s Central Hospital
chengdu, Sichuan, China (People's Republic)
WL

Wenqian Li

Chief physician
Qinghai Provincial People’s Hospital
xining, Qinghai, China (People's Republic)
JX

Jianwen Xiao

Chief physician
The Affiliated Children’s Hospital of Chongqing Medical University
chongqing, Chongqing, China (People's Republic)
FY

Fenge Yang

Chief physician
Fujian Medical University Union Hospital
fuzhou, Fujian, China (People's Republic)
XC

Xun Chen

Chief physician
Jinan Central Hospital
Jinan, Shandong, China (People's Republic)
WX

Weiqun Xu

Chief physician
The Affiliated Children’s Hospital of Zhejiang Medical University
hangzhou, Zhejiang, China (People's Republic)
JH

Jing Huang

Chief physician
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, China (People's Republic)
CM

Chuanrong Ma

Clinical research physician
Sinocelltech Ltd.
Beijing, Beijing, China (People's Republic)
WG

Wenlin Gai

Clinical research director
Sinocelltech Ltd.
Beijing, Beijing, China (People's Republic)
LX

Liangzhi Xie

Chairman
Sinocelltech Ltd.
Beijing, Beijing, China (People's Republic)
RY

Renchi Yang

Tianjin
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases
Tianjin, Tianjin, China (People's Republic)

Background: SCT800 is the first domestic recombinant factor VIII (rFVIII) approved in China for treatment of hemophilia A in adult patients, and is currently under investigation for pediatric indications.

Aims: The phase III study was designed to investigate the efficacy, safety and pharmacokinetics (PK) of SCT800 for prophylaxis and treatment of bleeding episodes in severe pediatric hemophilia A patients who have been previously treated with factor VIII (FVIII)

Methods: Study patients include boys < 12yr with severe hemophilia A (FVIII:C < 1%) and no history of FVIII inhibitors. For patients 0-5yr, the previous FVIII treatment should be >50 exposure days (ED); for those aged 6-11yr, ED should be >150. Eligible patients received SCT800 25-50 IU/kg every other day or three times a week for approximately 24 weeks. PK was evaluated after single injections of 50 IU/kg. The primary efficacy endpoint was annualized bleeding rate (ABR).

Results: Sixty-eight (68) patients were enrolled (34 each for age group of 0-5yr and 6-11) and the mean exposure to SCT800 was 78.9 days. The median ABRs of the two groups were similar, 2.21 vs 2.17(0-5yr vs 6-11yr. The median ABRs for spontaneous bleeds were 0.00 for both groups. Hemostatic efficacy was rated as “excellent” or “good” for 92.1% of bleeds. 83% of 127 reported bleeds were treated with ≤2 injections. Detailed ABR data for each group were included in the table below. Drug-related adverse events (AEs) occurred in 2.9% of the patients. Only one 2-year-old patient developed inhibitors and was successfully treated with high dose SCT800 using an immune tolerance induction (ITI) scheme. The higher clearance and shorter t1/2 were observed in patients 0-5yr compared to those of 6-11yr.

Conclusion(s): The data indicate that SCT800 is effective and safe for the prophylaxis and treatment of bleeds in previously FVIII-treated severe pediatric hemophilia A patients.