A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection

NCT05251090 | Completed Phase 1

• 1 other identifier: CTR20220279
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 6

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.

Conditions

Hemophilia A

Keywords

Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability

Outcome Measures

Primary Outcomes (4)

• Maximum measured concentration of FVIII:C (Cmax)

  Measured by aPTT Clotting Assay.

  Pre-dose and post dose up to 8 days.

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

  Measured by aPTT Clotting Assay.

  Pre-dose and post dose up to 8 days

• Area Under the Curve to Infinity (AUC)

  Measured by aPTT Clotting Assay.

  Pre-dose and post dose up to 8 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 aPTT Clotting Assay.

  Pre-dose and post dose up to 8 days.

Secondary Outcomes (2)

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

  Post dose up to 32 days.

• Development of Inhibitor

  Pre-dose and post dose up to 32 days.

Study Arms (1)

Arm 1

EXPERIMENTAL

Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period.

Drug: ADVATE; Drug: FRSW107

Interventions

ADVATE DRUG

50 international units (IU)/kg, a single dose.

Arm 1

FRSW107 DRUG

50 IU/kg, a single dose.

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

Arm 1

Eligibility Criteria

• Age: Up to 12 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)

You may qualify if:

• 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.

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.
• 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.

Sponsors & Collaborators

• Jiangsu Gensciences lnc.: lead

Study Sites (6)

Beijing Children's Hospital,Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Shenzhen Children's Hospita

Shenzhen, Guangdong, 518000, China

Location

Nanfang Hospital of Southern Medical University

Guangzhou, Guangzhou, 510515, China

Location

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, 430000, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, 266000, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 610000, China

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII; 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)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors; Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Runhui Wu, PhD

  Beijing Children's Hospital

  PRINCIPAL INVESTIGATOR

• Xiaoling Wang, MA.Sc

  Beijing Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2022
• First Posted: February 22, 2022
• Study Start: June 16, 2021
• Primary Completion: November 15, 2021
• Study Completion: May 9, 2022
• Last Updated: August 22, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (6)