Hemophilia APhase 3NCT04061109
Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.
A Phase 3 study testing Recombinant Human Coagulation FVIII in people with Hemophilia A, run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. at 11 sites in China.
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… Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Human Coagulation FVIII
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 11 sitesChina
- Study dates
- 15 May 2019to Mar 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
China11 sites, 11 recruiting
- Recruiting
Xiangya Hospital Central South University
Changsha, Hunan, 410013
Xielan Zhao, Doctor, 0731-84327564, zhaoxl9198@163.com
Xielan Zhao
- Recruiting
The Second Affiliated Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 400010
Shifeng Lou, Master, 023-62887783, 13508331213@163.com
Shifeng Lou
- Recruiting
FuJian Medical University Union Hospital
Fuzhou, Fujian, 350001
Meijuan Huang, D, 0591-86218041, huangmj163@163.com
Meijuan Huang
- Recruiting
AnHui Provincial Hospital
Hefei, Anhui, 230001
Zimin Sun, Doctor, 0551 -62283191, zmsun_vip@163.com
Zimin Sun
- Recruiting
The First Hospital of LanZhou University
Lanzhou, Gansu, 730000
Yaming Xi, Doctor, 0931-8356266, xiyaming@163.com
Yaming Xi
- Recruiting
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, 330006
Chenghao Jin, Doctor, 0791-86895612, Jinch227@aliyun.com
Chenghao Jin
- Recruiting
Nanjing Drum Tower Hospital
Nanjing, Jiangsu, 210008
Rongfu Zhou, Doctor, 83106666-61441, rfzhoucn@163.com
Rongfu Zhou
- Recruiting
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021
Peng Cheng, Master, 0771-12580, 13977166448@163.com
Peng Cheng
- Recruiting
Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Lei Zhang, Doctor, 022-20909240, zhanglei1@ihcams.ac.cn
Lei Zhang
- Recruiting
HeNan Provincial Peoples Hospital
Zhengzhou, Henan, 450003
Zhen Wang, Doctor, 0371-65896920, jenny1983218@126.com
Zhen Wang
- Recruiting
HeNan Cancer Provincial Hospital
Zhengzhou, Henan, 450008
Hu Zhou, Doctor, 0371-65587278, papertigerhu@163.com
Hu Zhou
About this study
Official title: Multicenter, Noncontrol Study of Recombinant Coagulation Factor VIII for Prophylactic Therapy in Subjects With Hemophilia A.
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.
- Conditions
- Hemophilia A
- Being tested
- Recombinant Human Coagulation FVIII
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human Coagulation FVIIIDrug
A kind of Recombinant Coagulation Factor VIII Injection produced by sponsor.
| Study group | Role | What they receive |
|---|---|---|
| Prophylactic therapy | Experimental | 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. Receives: Drug: Recombinant Human Coagulation FVIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: up to 24 weeks
Incremental Recovery of the First Dose
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection
Measured: After the first dose on day 1
Secondary outcomes (6)
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).
Measured: up to 24 weeks
Monthly Average Number of Bleedings
Number of bleeding events in each month.
Measured: up to 24 weeks
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
Measured: up to 24 weeks
Incremental Recovery of Duplicated Dose
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection.
Measured: up to 12 weeks, 24 weeks
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.
Measured: Baseline, week 24
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.
Measured: Baseline, week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2019
- Study start15 May 2019
- Primary completionMar 2020 (estimated)
- Study completionMar 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04061109
- Sponsor's protocol ID
- CTTQ-NXBYZ-02
- Status last verified
- Aug 2019
- First submitted
- 16 Jul 2019
- Last updated
- 19 Aug 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Noncontrol Study of Recombinant Coagulation Factor VIII for Prophylactic Therapy in Subjects With Hemophilia A.
- ClinicalTrials.gov ID
- NCT04061109
- Acronym / sponsor ID
- CTTQ-NXBYZ-02
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 May 2019 (actual)
- Primary completion
- Mar 2020 (estimated)
- Study completion
- Mar 2020 (estimated)
- First posted
- 19 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prophylactic therapy | Experimental | Drug: Recombinant Human Coagulation FVIIISubjects 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. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Human Coagulation FVIIIA kind of Recombinant Coagulation Factor VIII Injection produced by sponsor. | Drug | Prophylactic therapy |
Primary outcome
Annualized joint bleeding rate
Time frame: up to 24 weeks
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
Time frame: After the first dose on day 1
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection
6 secondary outcomes
Bleeding Event Treatment Efficacy
Time frame: up to 24 weeks
Monthly Average Number of Bleedings
Time frame: up to 24 weeks
The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode
Time frame: up to 24 weeks
Incremental Recovery of Duplicated Dose
Time frame: up to 12 weeks, 24 weeks
Hemophilia Joint Health Score (HJHS)
Time frame: Baseline, week 24
Assess the impact of Recombinant Human Coagulation FVIII following Patient Reported Outcome (PRO) EQ-5D
Time frame: Baseline, week 24
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Human Coagulation FVIII in people with Hemophilia A, run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. at 11 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lei Zhang, Doctor (022-20909240, zhanglei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized joint bleeding rate (time frame: up to 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2019.