Clinical trial NCT04061109: Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.
Official title: Multicenter, Noncontrol Study of Recombinant Coagulation Factor VIII for Prophylactic Therapy in Subjects With Hemophilia A.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Hemophilia A
Lead sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2019-08-19
Enrollment: 64
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Annualized joint bleeding rate
timeFrame: up to 24 weeks
measure: Incremental Recovery of the First Dose
timeFrame: After the first dose on day 1
Secondary outcomes:
measure: Bleeding Event Treatment Efficacy
timeFrame: up to 24 weeks
measure: Monthly Average Number of Bleedings
timeFrame: up to 24 weeks
measure: The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode
timeFrame: up to 24 weeks
measure: Incremental Recovery of Duplicated Dose
timeFrame: up to 12 weeks, 24 weeks
measure: Hemophilia Joint Health Score (HJHS)
timeFrame: Baseline, week 24
measure: Assess the impact of Recombinant Human Coagulation FVIII following Patient Reported Outcome (PRO) EQ-5D
timeFrame: Baseline, week 24
Other outcomes:
Locations:
city: Hefei
country: China
facility: AnHui Provincial Hospital
state: Anhui
city: Chongqing
country: China
facility: The Second Affiliated Hospital of Chongqing Medical University
state: Chongqing
city: Fuzhou
country: China
facility: FuJian Medical University Union Hospital
state: Fujian
city: Lanzhou
country: China
facility: The First Hospital of LanZhou University
state: Gansu
city: Nanning
country: China
facility: The First Affiliated Hospital of Guangxi Medical University
state: Guangxi
city: Zhengzhou
country: China
facility: HeNan Provincial Peoples Hospital
state: Henan
city: Zhengzhou
country: China
facility: HeNan Cancer Provincial Hospital
state: Henan
city: Changsha
country: China
facility: Xiangya Hospital Central South University
state: Hunan
city: Nanjing
country: China
facility: Nanjing Drum Tower Hospital
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial People's Hospital
state: Jiangxi
city: Tianjin
country: China
facility: Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
state: Tianjin
Study officials:
References: