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

August 21, 2023 updated by: Jiangsu Gensciences lnc.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
13

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100045
        • Beijing Children's Hospital,Capital Medical University
    • Guangdong
      • Shenzhen, Guangdong, China, 518000
        • Shenzhen Children's Hospita
    • Guangzhou
      • Guangzhou, Guangzhou, China, 510515
        • Nanfang Hospital of Southern Medical University
    • Hubei
      • Wuhan, Hubei, China, 430000
        • Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
    • Shandong
      • Qingdao, Shandong, China, 266000
        • The Affiliated Hospital of Qingdao University
    • Sichuan
      • Chengdu, Sichuan, China, 610000
        • Chengdu Women's and Children's Central Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

No older than 12 years (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Arm 1
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
50 international units (IU)/kg, a single dose.
Drug: FRSW107
50 IU/kg, a single dose.
Other Names:
  • Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Maximum measured concentration of FVIII:C (Cmax)
Time Frame: Pre-dose and post dose up to 8 days.
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)
Time Frame: Pre-dose and post dose up to 8 days
Measured by aPTT Clotting Assay.
Pre-dose and post dose up to 8 days
Area Under the Curve to Infinity (AUC)
Time Frame: Pre-dose and post dose up to 8 days.
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).
Time Frame: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
Pre-dose and post dose up to 8 days.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
Time Frame: Post dose up to 32 days.
Adverse events related to Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0.
Post dose up to 32 days.
Development of Inhibitor
Time Frame: Pre-dose and post dose up to 32 days.
Measured by the Nijmegen-Modified Bethesda Assay.
Pre-dose and post dose up to 32 days.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jiangsu Gensciences lnc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Runhui Wu, PhD, Beijing Children's Hospital
  • Principal Investigator: Xiaoling Wang, MA.Sc, Beijing Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 16, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 15, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 9, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 11, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 11, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 22, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 22, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 21, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CTR20220279

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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