Brief Title
A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Official Title
An Open-Label, Multicenter Evaluation of the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW117) in Patients With Severe Hemophilia A.
Brief Summary
The primary objectives of the study are to evaluate the Pharmacokinetics，Safety and
tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
(FRSW117) in patients with severe hemophilia A.
The secondary objectives are to monitor anti-durg antibodies and anti-PEG antibodies levels
in patients with severe hemophilia A
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Cmax from time 0 to the last data point for FRSW117 Measured by One-Stage Clotting Assay.
Secondary Outcome
Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
Condition
Severe Hemophilia A
Intervention
ADVATE
Study Arms / Comparison Groups
Arm 1-ADVATE+FRSW117(25 IU/kg)
Description: Subjects received two treatments: 25 IU/kg ADVATE in the first period, followed by 25 IU/kg FRSW117 in the second period, with a washout period before each treatment.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
13
Start Date
June 22, 2021
Completion Date
May 30, 2022
Primary Completion Date
April 30, 2022
Eligibility Criteria
Inclusion Criteria:
- Patients with clinically confirmed hemophilia A (coagulation factor VIII <1%) and
previous medical records confirming exposure to coagulation factor VIII for ≥150 days
(EDs ≥150).
- Non-immunodeficient, with some immunity (CD4 > 200/μL).
- Platelet count >100×10^9/L.
- Normal prothrombin time (PT) or international normalized ratio (INR) <1.3.
- Negative lupus anticoagulant.
- Fully understand, informed about this study and sign the informed consent form,
voluntarily participate in the clinical study and have the ability to complete all
study procedures
Exclusion Criteria:
- Hypersensitivity to the test substance or its excipients (including rodent or hamster
protein).
- Pre-existing hypersensitivity or allergic reactions to FVIII or IgG2 injection
therapy.
- Positive FVIII inhibitor at screening (≥0.6 BU/mL), or previous history of FVIII
inhibitor Positive history, or family history of inhibitors.
- Patients with other coagulation disorders in addition to hemophilia A.
- The results of vWF antigen examination lower than normal.
- Severe anemia and need blood transfusion (hemoglobin < 60g/L).
- Patients who have received any standard half-lives FVIII formulations (e.g., Kogenate,
Kovaltry, Advate, Xyntha, etc.) or received any other half-life-extending FVIII
formulations within 4 days or 5 half-lives (whichever is longer) before
administration.
- Patients who had used emecizumab within 6 months prior to administration.
- Patients with fever, upper respiratory tract infection or allergy symptoms within the
previous 2 weeks before screening.
- Suffer from other diseases that may increase the risk of bleeding or the risk of
thrombosis.
- Severe cardiovascular and cerebrovascular diseases: such as cerebral hemorrhage,
stroke, myocardial infarction, unstable angina pectoris, congestive heart failure(the
current New York Heart Association cardiac function grade III, Hypertension that
cannot be controlled with drug treatment: systolic blood pressure> 160 mmHg or
diastolic blood pressure> 95 mmHg.
- Clinically significant of other systematic diseases: alcoholism, drug abuse, mental
disorders and mental retardation.
- Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level >
3 × upper limit of normal (ULN)).
- Abnormal kidney function: BUN > 2×ULN, Cr > 2.0mg/dL.
- One or more clinically significant tests for Hepatitis B Virus Surface Antigen, Human
Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus
(HCV) Antibody.
- Patients who received any anticoagulant or antiplatelet therapy within a week prior
screening or need to receive an anticoagulant or antiplatelet therapy during the
period of clinical trials.
- Systemic immunomodulators (e.g., corticosteroids [equivalent dose of 10 mg/ day
prednisone], A-interferon, immunoglobulin, cyclophosphamide, cyclosporine, etc.) were
used within 14 days prior to administration or during the study period.
- Patients having major surgery or receiving blood or blood 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 a 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.
Gender
Male
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Renchi Yang, PhD, ,
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT04864743
Organization ID
CTR20210830
Responsible Party
Sponsor
Study Sponsor
Jiangsu Gensciences lnc.
Study Sponsor
Renchi Yang, PhD, Principal Investigator, Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
Verification Date
April 2021