A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

March 1, 2024 updated by: Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.

A Phase III Clinical Study Evaluating the Efficacy and Safety of On-demand Treatment and Perioperative Replacement Therapy With Recombinant Human Coagulation Factor VIII for Injection in Treated Severe Hemophilia A

This study is a single arm, open, multicenter clinical trial in which the previous treated severe hemophilia A patients receive recombinant human coagulation factor VIII for injection to evaluate the efficacy and safety of on-demand treatment in the event of new bleeding events. If evaluable surgical cases occur, the overall efficacy and safety of recombinant human coagulation factor VIII for injection as a replacement therapy for severe hemophilia A PTPs during the perioperative period will be evaluated.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 3

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 Locations

  • China
    • Anhui
      • Hefei, Anhui, China, 230002
        • Anhui Provincial Hospital
        • Contact:
    • Chongqing
      • Chongqing, Chongqing, China, 400010
        • The Second Affiliated Hospital of Chongqing Medical University
        • Contact:
    • Gansu
      • Lanzhou, Gansu, China, 730013
        • The First Hospital of Lanzhou University
        • Contact:
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Nanfang Hospital, Southern Medical University
        • Contact:
    • Guangxi
      • Nanning, Guangxi, China, 530021
        • The First Affiliated Hospital of Guangxi Medical University
        • Contact:
    • Henan
      • Zhengzhou, Henan, China, 450003
        • Henan Tumor Hospital
        • Contact:
    • Hunan
      • Changsha, Hunan, China, 410008
        • Xiangya Hospital Central South University
        • Contact:
    • Jiangsu
      • Nanjing, Jiangsu, China, 210008
        • Nanjing Drug Tower Hospital
        • Contact:
    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Jiangxi Provincial People's Hospital
        • Contact:
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Hematology Hospital of Chinese Academy of Medical Sciences
        • Contact:
    • Yunnan
      • Kunming, Yunnan, China, 650106
        • The Second Affiliated Hospital of Kunming Medical University
        • Contact:

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

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • The subjects voluntarily joined this study, signed an informed consent form, and had good compliance;
  • Age: ≥ 12 years old and ≤ 65 years old (when signing the informed consent form, if it is a minor, parents should sign on behalf of it);
  • Severe hemophilia A (coagulation factor VIII activity<1%), with exposure days (ED) of no less than 150 days prior to factor VIII (recombinant coagulation factor VIII or plasma derived coagulation factor VIII) treatment; Surgical patients need to have undergone at least 2 surgeries (including major surgeries) and plan to undergo elective surgery at the same time;
  • The subjects must be HIV negative or have a viral load of < 200 particles/μ, HIV positive patients with l or < 400000 copies/ml.
  • At baseline, no inhibitors were detected, and there was no history of Factor VIII inhibitor antibody formation (Bethesda method detection < 0.6 BU/ml, test result record required);
  • Subjects of childbearing age who agree to take effective contraceptive measures throughout the entire trial period and continue until 28 days after the last medication.

Exclusion Criteria:

  • Known congenital or acquired hemorrhagic diseases other than hemophilia A;
  • The subjects have used or planned to receive immunosuppressive treatments such as prednisone, cyclophosphamide, and cyclosporine in the week prior to enrollment, and are allowed to use inhaled or topical corticosteroids;
  • Those who are known or suspected to have hypersensitivity reactions to the investigational drug, including human embryonic kidney cell protein;
  • Significant liver and kidney dysfunction, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 5 upper limit of normal (ULN), and serum creatinine >1.5 ULN;
  • Hemoglobin < 60 g/L;
  • Expected to receive other drugs for the treatment of hemophilia A within 72 hours before the first administration (such as recombinant factor VIII, blood derived factor VIII, desmopressin, cryoprecipitate, plasma, etc.), or those who have used long-acting hemophilia A drugs for less than 5 half-lives before the first administration;
  • Used within 14 days before the first administration or planned to use anticoagulants or antiplatelet drugs for treatment during clinical trials;
  • Within 6 months prior to the first administration, use of Emicizumab;
  • Serious cardiovascular and cerebrovascular diseases occurring within 6 months prior to the first administration, including but not limited to cerebral hemorrhage, cerebral infarction, unstable angina, malignant arrhythmia, acute myocardial infarction, congestive heart failure (New York Heart Association cardiac function classification ≥ III), and hypertension (systolic blood pressure ≥ 160 mmHg or diastolic blood pressure 100 mmHg) that is poorly controlled despite treatment with one or more antihypertensive drugs;
  • Participated in clinical trials of other drugs within 30 days prior to screening, or last used investigational drug with less than 5 half-lives;
  • Alcohol abuse, drug abuse, mental disorders, and intellectual disabilities.

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
Intervention / Treatment
Experimental: Recombinant human activated coagulation factor Ⅷ for injection
Each subject in this study receive on-demand treatment with recombinant human coagulation factor VIII for injection for 180 days, with an increase in medication frequency based on the relief after medication.
Drug: Recombinant human activated coagulation factor VIII for injection
Recombinant human coagulation factor VIII for injection is a recombinant DNA product with functional characteristics comparable to endogenous factor VIII.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The proportion of on-demand treatment improvement for newly occurring bleeding events
Time Frame: For each new bleeding event, assessed within 72 hours after initial treatment
Evaluate the hemostatic efficacy of the patient after each new bleeding event (including new bleeding events during visits and new bleeding events during home treatment), and calculate the proportion of improvement as excellent+good to the total number of on-demand treatment evaluations.
For each new bleeding event, assessed within 72 hours after initial treatment
Activity recovery
Time Frame: 15 minutes after infusion
The activity of Recombinant human coagulation factor VIII measured at 15 minutes after infusion minus the baseline Recombinant human coagulation factor VIII activity divided by the expected improvement in Recombinant human coagulation factor VIII activity, which reflects the improvement in Recombinant human coagulation factor VIII levels after infusion.
15 minutes after infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The proportion of effective surgical hemostasis
Time Frame: Within 72 hours after surgery
Assess the hemostatic efficacy of injecting recombinant human coagulation factor VIII during and after surgery. The effective ratio is the proportion of excellent or good to the total times of the surgical evaluation of hemostatic efficacy
Within 72 hours after surgery
Annualized bleeding rate (ABR)
Time Frame: Up to 180 days.
Including spontaneous and traumatic bleeding, as well as total bleeding; ABR can be calculated using the following formula: bleeding frequency/(treatment period days/365.25).
Up to 180 days.
The incidence of Less Han Expected Thermal Effect
Time Frame: For each new bleeding event, assessed within 24 hours after initial treatment
Without the influence of confounding factors, continuously infuse Recombinant human coagulation factor VIII twice within 24 hours for on-demand treatment, with no response to the same bleeding event.
For each new bleeding event, assessed within 24 hours after initial treatment
The injection times of recombinant human coagulation factor VIII
Time Frame: Up to 180 days.
The injection times of recombinant human coagulation factor VIII for each new bleeding event.
Up to 180 days.
The dosage of recombinant human coagulation factor VIII
Time Frame: Up to 180 days.
The dosage of recombinant human coagulation factor VIII (including average and total doses) for each new bleeding event
Up to 180 days.
Changes of Haemophilia Joint Health Score before and after on-demand treatment
Time Frame: Up to 180 days.
The scale assesses the functional status of the six major joints of the elbow, knee, and ankle, including: joint swelling, swelling duration, muscle atrophy, muscle strength, joint friction, joint pain, joint extension, joint flexion Degree decline, overall gait, etc.
Up to 180 days.
Changes of Quality of Life Assessment before and after on-demand treatment
Time Frame: Up to 180 days.
Quality of Life Assessment is a general questionnaire designed to measure health status on a scale of 0-100 with the higher value representing a better outcome and record the participants' current health state in 5 domains mobility, selfcare, usual activities, pain, anxiety.
Up to 180 days.

Collaborators and Investigators

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

Sponsor

Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.

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 (Estimated)

April 1, 2024

Primary Completion (Estimated)

April 1, 2025

Study Completion (Estimated)

April 1, 2025

Study Registration Dates

First Submitted

March 1, 2024

First Submitted That Met QC Criteria

March 1, 2024

First Posted (Actual)

March 7, 2024

Study Record Updates

Last Update Posted (Actual)

March 7, 2024

Last Update Submitted That Met QC Criteria

March 1, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TQG202-III-01

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.

Clinical Trials on Hemophilia A

Clinical Trials on Recombinant human activated coagulation factor VIII for injection

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Gabon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe