A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

NCT05265286 | Completed Phase 2

• 1 other identifier: CTR20220283
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 5

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.

Conditions

Severe Hemophilia A

Keywords

Pharmacokinetics;; Safety;; immunogenicity;; Preliminary efficacy

Outcome Measures

Primary Outcomes (6)

• Maximum measured concentration of FVIII:C (Cmax)

  Measured by One-Stage Clotting Assay

  Pre-dose and post dose up to 10 days

• Time required for the concentration of the drug to reach half of its original value (T1/2)

  Measured by One-Stage Clotting Assay

  Pre-dose and post dose up to 10 days

• Area Under the Curve to Infinity (AUC)

  Measured by One-Stage Clotting Assay

  Pre-dose and post dose up to 10 days

• 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)

  Measured by One-Stage Clotting Assay

  Pre-dose and post dose up to 10 days

• Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants

  Pre-dose and post dose up to 36 days

• Evaluation of the level of anti-PEG antibody production in participants

  Pre-dose and post dose up to 36 days

Secondary Outcomes (3)

• Number of participants with treatment-related adverse events as assessed by CTCAE V5.0

  Pre-dose and post dose up to 36 days

• Development of Inhibitor

  Pre-dose and post dose up to 36 days

• score of bleeding symptoms and Vital signs

  Pre-dose and post dose up to 36 days

Study Arms (1)

Arm 1 prophylaxis treatment

EXPERIMENTAL

Subjects of high dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. Subjects of low dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. All subjects are being received PK assessment in ED1 and ED4.

Drug: FRSW117

Interventions

FRSW117 DRUG

Subjects of high dose group are being received four doses(50 IU/kg,once a week or every 7 days) of FRSW117. Subjects of low dose group (40 IU/kg,once a week or every 7 days)are being received four doses of FRSW117.

Also known as: PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Arm 1 prophylaxis treatment

Eligibility Criteria

• Age: 12 Years - 65 Years
• Gender Eligibility Details: Hemophilia A is an X-chromosome-linked recessive inherited bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII)
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Jiangsu Gensciences lnc.: lead

Study Sites (5)

People'S Hospital of Zhengzhou

Zhengzhou, Henan, 450000, China

Location

Affiliated Hospital of Jiangnan University

Wuxi, Jiangsu, 214100, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, 266000, China

Location

People'S Hospital of Rizhao

Rizhao, Shandong, 276800, China

Location

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Injections

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Renchi Yang, PhD

  Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2022
• First Posted: March 3, 2022
• Study Start: April 14, 2022
• Primary Completion: August 21, 2022
• Study Completion: August 21, 2022
• Last Updated: May 15, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (5)