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

NCT06297655 | Not Yet Recruiting Phase 3

• 1 other identifier: TQG202-III-01
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 11

Brief Summary

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (2)

• The proportion of on-demand treatment improvement for newly occurring bleeding events

  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

  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 Outcomes (7)

• The proportion of effective surgical hemostasis

  Within 72 hours after surgery

• Annualized bleeding rate (ABR)

  Up to 180 days.

• The incidence of Less Han Expected Thermal Effect

  For each new bleeding event, assessed within 24 hours after initial treatment

• The injection times of recombinant human coagulation factor VIII

  Up to 180 days.

• The dosage of recombinant human coagulation factor VIII

  Up to 180 days.

Study Arms (1)

Recombinant human activated coagulation factor Ⅷ for injection

EXPERIMENTAL

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

Interventions

Recombinant human activated coagulation factor VIII for injection DRUG

Recombinant human coagulation factor VIII for injection is a recombinant DNA product with functional characteristics comparable to endogenous factor VIII.

Recombinant human activated coagulation factor Ⅷ for injection

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.: lead

Study Sites (11)

Anhui Provincial Hospital

Hefei, Anhui, 230002, China

Location

The Second Affiliated Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, 400010, China

Location

The First Hospital of Lanzhou University

Lanzhou, Gansu, 730013, China

Location

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, 510515, China

Location

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, 530021, China

Location

Henan Tumor Hospital

Zhengzhou, Henan, 450003, China

Location

Xiangya Hospital Central South University

Changsha, Hunan, 410008, China

Location

Nanjing Drug Tower Hospital

Nanjing, Jiangsu, 210008, China

Location

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, 330006, China

Location

Hematology Hospital of Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

The Second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650106, 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

Central Study Contacts

Lei Zhang, Doctor

CONTACT

13502118379; zhanglei1@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2024
• First Posted: March 7, 2024
• Study Start: April 1, 2024
• Primary Completion: April 1, 2025
• Study Completion: April 1, 2025
• Last Updated: March 7, 2024

Record last verified: 2024-03

Locations

CN (11)