Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.

Hemophilia A Clinical Trial

Official title:

Multicenter, Noncontrol Study of Recombinant Coagulation Factor VIII for Prophylactic Therapy in Subjects With Hemophilia A.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04061109
• Other study ID #: CTTQ-NXBYZ-02
• Status: Recruiting
• Phase: Phase 3
• Start date: May 15, 2019
• Est. completion date: March 2020

Study information

• Verified date: August 2019
• Source: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
• Contact: Lei Zhang, Doctor
• Phone: 022-20909240
• Email: zhanglei1[@]ihcams.ac.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a multi-center, open-label, single-arm trial to evaluate the efficacy and safety of Recombinant Coagulation Factor VIII in patients with hemophilia A . Recombinant Coagulation Factor VIII is prophylactic administrated 25 - 35 IU/kg once every other day or three times per week which should be continuous for 24 weeks.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 64
• Est. completion date: March 2020
• Est. primary completion date: March 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

Inclusion Criteria:

1. Hemophilia A.

2. FVIII:C <1%. 3)12 and 65 years old.

4)Has received FVIII treatment and the treatment exposure days =100. 5)Has bleeding treatment records of at least 3 months before randomization. 6)FVIII inhibitor assay results is negative. 7) Subjects should agree to use an adequate method of contraception during the study.

8)Understood and Signed an informed consent form.

Exclusion Criteria:

1. Has a history or family history of blood coagulation factor VIII inhibitor.

2. Has other coagulation dysfunction diseases in addition to hemophilia A.

3. HIV positive.

4. Plan to receive surgery during the trial.

5. Has used immunomodulator within one weeks before the first dose,and less than 7 half-life periods.

6. Known to be allergic to experimental drugs or any excipients.

7. Severe anemia and need blood transfusion.

8. Serious liver or kidney damage.

9. Serious heart disease.

10. Uncontrollable hypertension.

11. Has participated in other clinical studies within one month before the first dose.

12. The researchers believe that it is not suitable for participants.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Recombinant Human Coagulation FVIII
A kind of Recombinant Coagulation Factor VIII Injection produced by sponsor.

Locations

Country	Name	City	State
China	Xiangya Hospital Central South University	Changsha	Hunan
China	The Second Affiliated Hospital of Chongqing Medical University	Chongqing	Chongqing
China	FuJian Medical University Union Hospital	Fuzhou	Fujian
China	AnHui Provincial Hospital	Hefei	Anhui
China	The First Hospital of LanZhou University	Lanzhou	Gansu
China	Jiangxi Provincial People's Hospital	Nanchang	Jiangxi
China	Nanjing Drum Tower Hospital	Nanjing	Jiangsu
China	The First Affiliated Hospital of Guangxi Medical University	Nanning	Guangxi
China	Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences	Tianjin	Tianjin
China	HeNan Cancer Provincial Hospital	Zhengzhou	Henan
China	HeNan Provincial Peoples Hospital	Zhengzhou	Henan

Sponsors (1)

Lead Sponsor	Collaborator
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized joint bleeding rate	Annualized joint bleeding rate(AJBR) can be calculated using the following formula: Number of joint bleeding events during efficacy evaluation period/(number of days in treatment period/365.25).	up to 24 weeks
Primary	Incremental Recovery of the First Dose	Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection	After the first dose on day 1
Secondary	Bleeding Event Treatment Efficacy	The investigator shall evaluate the hemostatic effect after the treatment of every bleeding event of subjects based on a four-point scale(excellent, good, moderate, not relieved).	up to 24 weeks
Secondary	Monthly Average Number of Bleedings	Number of bleeding events in each month.	up to 24 weeks
Secondary	The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode	The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode	up to 24 weeks
Secondary	Incremental Recovery of Duplicated Dose	Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection.	up to 12 weeks, 24 weeks
Secondary	Hemophilia Joint Health Score (HJHS)	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.	Baseline, week 24
Secondary	Assess the impact of Recombinant Human Coagulation FVIII following Patient Reported Outcome (PRO) EQ-5D	EQ-5D 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.	Baseline, week 24