Back to resultsSafety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.
Primary Purpose
Hemophilia A
Status
Unknown status
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Recombinant Human Coagulation FVIII
Sponsored by
About this trial
This is an interventional prevention trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria:
- Hemophilia A.
- 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:
- Has a history or family history of blood coagulation factor VIII inhibitor.
- Has other coagulation dysfunction diseases in addition to hemophilia A.
- HIV positive.
- Plan to receive surgery during the trial.
- Has used immunomodulator within one weeks before the first dose，and less than 7 half-life periods.
- Known to be allergic to experimental drugs or any excipients.
- Severe anemia and need blood transfusion.
- Serious liver or kidney damage.
- Serious heart disease.
- Uncontrollable hypertension.
- Has participated in other clinical studies within one month before the first dose.
- The researchers believe that it is not suitable for participants.
Sites / Locations
- AnHui Provincial HospitalRecruiting
- The Second Affiliated Hospital of Chongqing Medical UniversityRecruiting
- FuJian Medical University Union HospitalRecruiting
- The First Hospital of LanZhou UniversityRecruiting
- The First Affiliated Hospital of Guangxi Medical UniversityRecruiting
- HeNan Provincial Peoples HospitalRecruiting
- HeNan Cancer Provincial HospitalRecruiting
- Xiangya Hospital Central South UniversityRecruiting
- Nanjing Drum Tower HospitalRecruiting
- Jiangxi Provincial People's HospitalRecruiting
- Hematology and Blood Diseases Hospital, Chinese Academy of Medical SciencesRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Prophylactic therapy
Arm Description
Subjects received Recombinant Human Coagulation FVIII for prophylactic therapy with 25 - 35 IU/kg injection once every other day or three times per week for 6 months.
Outcomes
Primary Outcome Measures
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).
Incremental Recovery of the First Dose
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection
Secondary Outcome Measures
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).
Monthly Average Number of Bleedings
Number of bleeding events in each month.
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
Incremental Recovery of Duplicated Dose
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection.
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.
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.
Full Information
NCT ID
NCT04061109
First Posted
July 16, 2019
Last Updated
August 16, 2019
Sponsor
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT04061109
Brief Title
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
2. Study Status
Record Verification Date
August 2019
Overall Recruitment Status
Unknown status
Study Start Date
May 15, 2019 (Actual)
Primary Completion Date
March 2020 (Anticipated)
Study Completion Date
March 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
64 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Prophylactic therapy
Arm Type
Experimental
Arm Description
Subjects received Recombinant Human Coagulation FVIII for prophylactic therapy with 25 - 35 IU/kg injection once every other day or three times per week for 6 months.
Intervention Type
Drug
Intervention Name(s)
Recombinant Human Coagulation FVIII
Intervention Description
A kind of Recombinant Coagulation Factor VIII Injection produced by sponsor.
Primary Outcome Measure Information:
Title
Annualized joint bleeding rate
Description
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).
Time Frame
up to 24 weeks
Title
Incremental Recovery of the First Dose
Description
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection
Time Frame
After the first dose on day 1
Secondary Outcome Measure Information:
Title
Bleeding Event Treatment Efficacy
Description
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).
Time Frame
up to 24 weeks
Title
Monthly Average Number of Bleedings
Description
Number of bleeding events in each month.
Time Frame
up to 24 weeks
Title
The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode
Description
The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode
Time Frame
up to 24 weeks
Title
Incremental Recovery of Duplicated Dose
Description
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection.
Time Frame
up to 12 weeks, 24 weeks
Title
Hemophilia Joint Health Score (HJHS)
Description
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.
Time Frame
Baseline, week 24
Title
Assess the impact of Recombinant Human Coagulation FVIII following Patient Reported Outcome (PRO) EQ-5D
Description
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.
Time Frame
Baseline, week 24
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Hemophilia A.
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:
Has a history or family history of blood coagulation factor VIII inhibitor.
Has other coagulation dysfunction diseases in addition to hemophilia A.
HIV positive.
Plan to receive surgery during the trial.
Has used immunomodulator within one weeks before the first dose，and less than 7 half-life periods.
Known to be allergic to experimental drugs or any excipients.
Severe anemia and need blood transfusion.
Serious liver or kidney damage.
Serious heart disease.
Uncontrollable hypertension.
Has participated in other clinical studies within one month before the first dose.
The researchers believe that it is not suitable for participants.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lei Zhang, Doctor
Phone
022-20909240
Email
zhanglei1@ihcams.ac.cn
Facility Information:
Facility Name
AnHui Provincial Hospital
City
Hefei
State/Province
Anhui
ZIP/Postal Code
230001
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zimin Sun, Doctor
Phone
0551 -62283191
Email
zmsun_vip@163.com
First Name & Middle Initial & Last Name & Degree
Zimin Sun
Facility Name
The Second Affiliated Hospital of Chongqing Medical University
City
Chongqing
State/Province
Chongqing
ZIP/Postal Code
400010
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shifeng Lou, Master
Phone
023-62887783
Email
13508331213@163.com
First Name & Middle Initial & Last Name & Degree
Shifeng Lou
Facility Name
FuJian Medical University Union Hospital
City
Fuzhou
State/Province
Fujian
ZIP/Postal Code
350001
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Meijuan Huang, D
Phone
0591-86218041
Email
huangmj163@163.com
First Name & Middle Initial & Last Name & Degree
Meijuan Huang
Facility Name
The First Hospital of LanZhou University
City
Lanzhou
State/Province
Gansu
ZIP/Postal Code
730000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yaming Xi, Doctor
Phone
0931-8356266
Email
xiyaming@163.com
First Name & Middle Initial & Last Name & Degree
Yaming Xi
Facility Name
The First Affiliated Hospital of Guangxi Medical University
City
Nanning
State/Province
Guangxi
ZIP/Postal Code
530021
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Peng Cheng, Master
Phone
0771-12580
Email
13977166448@163.com
First Name & Middle Initial & Last Name & Degree
Peng Cheng
Facility Name
HeNan Provincial Peoples Hospital
City
Zhengzhou
State/Province
Henan
ZIP/Postal Code
450003
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhen Wang, Doctor
Phone
0371-65896920
Email
jenny1983218@126.com
First Name & Middle Initial & Last Name & Degree
Zhen Wang
Facility Name
HeNan Cancer Provincial Hospital
City
Zhengzhou
State/Province
Henan
ZIP/Postal Code
450008
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hu Zhou, Doctor
Phone
0371-65587278
Email
papertigerhu@163.com
First Name & Middle Initial & Last Name & Degree
Hu Zhou
Facility Name
Xiangya Hospital Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410013
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xielan Zhao, Doctor
Phone
0731-84327564
Email
zhaoxl9198@163.com
First Name & Middle Initial & Last Name & Degree
Xielan Zhao
Facility Name
Nanjing Drum Tower Hospital
City
Nanjing
State/Province
Jiangsu
ZIP/Postal Code
210008
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rongfu Zhou, Doctor
Phone
83106666-61441
Email
rfzhoucn@163.com
First Name & Middle Initial & Last Name & Degree
Rongfu Zhou
Facility Name
Jiangxi Provincial People's Hospital
City
Nanchang
State/Province
Jiangxi
ZIP/Postal Code
330006
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Chenghao Jin, Doctor
Phone
0791-86895612
Email
Jinch227@aliyun.com
First Name & Middle Initial & Last Name & Degree
Chenghao Jin
Facility Name
Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lei Zhang, Doctor
Phone
022-20909240
Email
zhanglei1@ihcams.ac.cn
First Name & Middle Initial & Last Name & Degree
Lei Zhang
12. IPD Sharing Statement
Learn more about this trial
Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.
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