Clinical trial NCT05265286: A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Official title: A Phase II, Multicentre, Open-label Study to Evaluate the Pharmacokinetic, Safety and Preliminary Efficacy of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Subjects With Severe Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Hemophilia A
Keywords: Pharmacokinetics;
Safety;
immunogenicity;
Preliminary efficacy
Lead sponsor: Jiangsu Gensciences lnc.
Sponsor class: INDUSTRY
First posted: 2022-03-03
Enrollment: 15
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A.


Eligibility criteria: Key Inclusion Criteria:

* The activity of the coagulation factor VIII (FVIII:C) \< 1%.
* 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.• Significant hepatic or renal impairment (ALT and AST \> 2×ULN; serum bilirubin level \> 2 × upper limit of normal (ULN), Urea /BUN \> 2×ULN, Cr \> 176.8 µmol/L)
* 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 screening
* 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

Other protocol-defined inclusion/exclusion Criteria May Apply.

Primary outcomes:
measure: Maximum measured concentration of FVIII:C (Cmax)
timeFrame: Pre-dose and post dose up to 10 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 10 days
measure: Area Under the Curve to Infinity (AUC)
timeFrame: Pre-dose and post dose up to 10 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 10 days
measure: Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
timeFrame: Pre-dose and post dose up to 36 days
measure: Evaluation of the level of anti-PEG antibody production in participants
timeFrame: Pre-dose and post dose up to 36 days
Secondary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE V5.0
timeFrame: Pre-dose and post dose up to 36 days
measure: Development of Inhibitor
timeFrame: Pre-dose and post dose up to 36 days
measure: score of bleeding symptoms and Vital signs
timeFrame: Pre-dose and post dose up to 36 days
Other outcomes:
Locations:
city: Zhengzhou
country: China
facility: People'S Hospital of Zhengzhou
state: Henan
city: Wuxi
country: China
facility: Affiliated Hospital of Jiangnan University
state: Jiangsu
city: Qingdao
country: China
facility: The Affiliated Hospital of Qingdao University
state: Shandong
city: Rizhao
country: China
facility: People'S Hospital of Rizhao
state: Shandong
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
state: Tianjin
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
name: Renchi Yang, PhD
role: PRINCIPAL_INVESTIGATOR
References: