Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A

NCT04845399 | Completed Phase 3

• 1 other identifier: CTR20210593
• Study Type: interventional
• Target: 101
• Locations: 1 country
• Sites: 16

Brief Summary

The primary objectives of the study are to further evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in adolescent and adult patients with hemophilia A.

Conditions

Hemophilia A

Keywords

Hemophilia A; Factor VIII-Fc Fusion Protein; Phase III Expansion Trial; Efficacy and Safety

Outcome Measures

Primary Outcomes (3)

• Annualized Bleeding Rates (ABR).

  Annualized bleeding rate = (number of bleeding episodes during the efficacy, period/total number of days during the efficacy period)\*365.25. The efficacy period begins with the first prophylactic dose of FRSW107 and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods are not included in the efficacy period. A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. Any injection to treat the bleeding episode taken more than 72 hours after the preceding one was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last injection.

  For the duration of study participation, 6 months.

• Number of target joints.

  Describe the number and percentage of cases with target joints ≥1 and their 95% confidence intervals before and after drug administration, and compare the change in the number of target joints from baseline after treatment.

  For the duration of study participation, 6 months.

• Annualized Joint Bleeding Rate (AJBR)

  Annualized joint bleeding rate(AJBR)can be calculated using the following formula: Number of joint bleeding episodesevents during efficacy evaluation period/(number of days in treatment period/365.25).

  For the duration of study participation, 6 months.

Secondary Outcomes (7)

• Total Dose Required for Resolution of a Bleeding Episode.

  For the duration of study participation, 6 months.

• Number of injections required to resolve a bleeding episode.

  For the duration of study participation, 6 months.

• Quality of life assessment.

  For the duration of study participation, 6 months.

• Score of bleeding symptoms and Vital signs.

  For the duration of study participation, 6 months.

• Number of participants with inhibitor development

  For the duration of study participation, 6 months.

Study Arms (1)

Arm 1

EXPERIMENTAL

Participants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months.

Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Interventions

Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection DRUG

Participants received prophylaxis treatment at 50 IU/ kg every three days.30-50 IU/kg is recommended to administration while bleeding occurs during the experiment.

Arm 1

Eligibility Criteria

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

You may qualify if:

• Patients Who have Completed trial of RH-107-001 (previously treated patients) Previously received Recombinant Human Coagulation Factor VIII-Fc prophylactic.
• The patient and/or guardian or his or her legal representative must be able to read, understand, and provide signed informed consent, And voluntarily signed the Informed Consent Form.
• The compliance of patients appeared quite well.
• Patient who is considered by the investigators suitable for ongoing to accept previously treated.

You may not qualify if:

• Subjects who have not completed trial of RH-107-001or who have completed the Phase III clinical trial but not willing to continue receiving treatment.
• Subjects who did not participate in the Phase III clinical trial of RH-107-001.

Sponsors & Collaborators

• Zhengzhou Gensciences Inc: lead
• Jiangsu Gensciences lnc.: collaborator

Study Sites (16)

Anhui Provincial Hospital

Hefei, Anhui, 230001, China

Location

Capital Medical University affiliated Beijing Children's Hospital

Beijing, Beijing Municipality, 100045, China

Location

Chongqing Three Gorges Central Hospital

Chongqing, Chongqing Municipality, 404000, China

Location

The First Hospital of Lanzhou University

Lanzhou, Gansu, 730000, China

Location

The Second Affiliated Hospital of Guangzhou Medical University

Guangzhou, Guangzhou, 510260, China

Location

Nanfang Hospital of Southern Medical University

Guangzhou, Guangzhou, 510515, China

Location

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, 550004, China

Location

Henan provincial People's Hospital

Zhengzhou, He'nan, 450003, China

Location

Henan Cancer Hospital

Zhengzhou, He'nan, 450008, China

Location

Xiangya Hospital of Central South University

Changsha, Hunan, 410013, China

Location

The Affiliated Hospital of Xuzhou Medical College

Xuzhou, Jiangsu, 221002, China

Location

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, 330006, China

Location

Jinan central hospital

Ji'nan, Shandong, 250013, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, 266000, China

Location

The Second Hospital of Shanxi Medical University

Taiyuan, Shanxi, 030001, 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

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

  PRINCIPAL INVESTIGATOR

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: April 9, 2021
• First Posted: April 15, 2021
• Study Start: April 16, 2021
• Primary Completion: June 26, 2021
• Study Completion: June 26, 2021
• Last Updated: August 29, 2023

Record last verified: 2021-04

Locations

CN (16)