Clinical trial NCT05251090: A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection
Official title: A Phase I, Multicentre, Open-label Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection in Children With Severe Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hemophilia A
Keywords: Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability
Lead sponsor: Jiangsu Gensciences lnc.
Sponsor class: INDUSTRY
First posted: 2022-02-22
Enrollment: 13
Sex: MALE
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A.


Eligibility criteria: Key Inclusion Criteria:

* The activity of the coagulation factor VIII (FVIII:C) \< 1%. Less than 6 years old Patients previously treated with FVIII concentrate (s) for a minimum of 50 exposure days (EDs) prior to study entry. 6 years old to 12 years old Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
* Normal prothrombin time or INR \< 1.3.
* Negative lupus anticoagulant.

Key Exclusion Criteria:

* Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins).
* History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration.
* Current FVIII inhibitor-positive or history of FVIII inhibitor-positive.
* Other coagulation disorder(s) in addition to hemophilia A.
* Infusion of any products containing FVIII within 72 h prior to administration.
* Significant hepatic or renal impairment (ALT and AST \> 2×ULN; serum bilirubin level \> 2 × upper limit of normal (ULN), BUN \> 2×ULN, Cr \> 2.0 ULN).
* One or more clinically significant tests for Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody.
* Patients who received any anticoagulant or antiplatelet therapy within one week prior screening or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials.
* Patients having major surgery or receiving blood or bood components transfusion within 4 weeks prior screening or having planned major surgery schedule during the study.
* Patients who previously participated in the other clinical trials within one month prior to administration.
* Any life-threatening disease or condition which, according to the investigator's judgment, could not benefit from the trial participation.
* Patient who is considered by the other investigators not suitable for clinical study.

Primary outcomes:
measure: Maximum measured concentration of FVIII:C (Cmax)
timeFrame: Pre-dose and post dose up to 8 days.
measure: Time required for the concentration of the drug to reach half of its original value (T1/2)
timeFrame: Pre-dose and post dose up to 8 days
measure: Area Under the Curve to Infinity (AUC)
timeFrame: Pre-dose and post dose up to 8 days.
measure: The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL).
timeFrame: Pre-dose and post dose up to 8 days.
Secondary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
timeFrame: Post dose up to 32 days.
measure: Development of Inhibitor
timeFrame: Pre-dose and post dose up to 32 days.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Children's Hospital,Capital Medical University
state: Beijing
city: Shenzhen
country: China
facility: Shenzhen Children's Hospita
state: Guangdong
city: Guangzhou
country: China
facility: Nanfang Hospital of Southern Medical University
state: Guangzhou
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
state: Hubei
city: Qingdao
country: China
facility: The Affiliated Hospital of Qingdao University
state: Shandong
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
state: Sichuan
Study officials:
affiliation: Beijing Children's Hospital
name: Runhui Wu, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Beijing Children's Hospital
name: Xiaoling Wang, MA.Sc
role: PRINCIPAL_INVESTIGATOR
References: