Hemophilia APhase 3NCT06297655
A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A
A Phase 3 study testing Recombinant human activated coagulation factor VIII for injection in people with Hemophilia A, run by Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd. at 12 sites in China.
Trial summary
This study is a single arm, open, multicenter clinical trial in which the previous treated severe hemophilia A patients receive recombinant human coagulation factor VIII for injection to evaluate the efficacy and safety of on-demand treatment in the event of new bleeding events. 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 activated coagulation factor VIII for injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 12 sitesChina
- Study dates
- 12 Nov 2024to Jul 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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
- The subjects voluntarily joined this study, signed an informed consent form, and had good compliance;
- Age (calculated from the date of signing the informed consent form): ≥12 years and ≤65 years;
- Severe hemophilia A (coagulation factor VIII activity<1%), with exposure days (ED) of no less than 150 days prior to factor VIII (recombinant coagulation factor VIII or plasma derived coagulation factor VIII) treatment; Surgical patients need to have undergone at least 2 surgeries (including major surgeries) and plan to undergo elective surgery at the same time;
- The subjects must be HIV negative or have a viral load of < 200 particles/μ, HIV positive patients with l or < 400000 copies/ml.
- At baseline, no inhibitors were detected, and there was no history of Factor VIII inhibitor antibody formation (Bethesda method detection < 0.6 BU/ml, test result record required);
- Subjects of childbearing age who agree to take effective contraceptive measures throughout the entire trial period and continue until 28 days after the last medication.
Who cannot join
- Known congenital or acquired hemorrhagic diseases other than hemophilia A;
- The subjects have used or planned to receive immunosuppressive treatments such as prednisone, cyclophosphamide, and cyclosporine in the week prior to enrollment, and are allowed to use inhaled or topical corticosteroids;
- Those who are known or suspected to have hypersensitivity reactions to the investigational drug, including human embryonic kidney cell protein;
- Significant liver and kidney dysfunction, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 5 upper limit of normal (ULN), and serum creatinine >1.5 ULN;
- Hemoglobin < 60 g/L;
- Expected to receive other drugs for the treatment of hemophilia A within 72 hours before the first administration (such as recombinant factor VIII, blood derived factor VIII, desmopressin, cryoprecipitate, plasma, etc.), or those who have used long-acting hemophilia A drugs for less than 5 half-lives before the first administration;
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- Used within 14 days before the first administration or planned to use anticoagulants or antiplatelet drugs for treatment during clinical trials;
- Within 6 months prior to the first administration, use of Emicizumab;
- Serious cardiovascular and cerebrovascular diseases occurring within 6 months prior to the first administration, including but not limited to cerebral hemorrhage, cerebral infarction, unstable angina, malignant arrhythmia, acute myocardial infarction, congestive heart failure (New York Heart Association cardiac function classification ≥ III), and hypertension (systolic blood pressure ≥ 160 mmHg or diastolic blood pressure 100 mmHg) that is poorly controlled despite treatment with one or more antihypertensive drugs;
- Participated in clinical trials of other drugs within 30 days prior to screening, or last used investigational drug with less than 5 half-lives;
- Alcohol abuse, drug abuse, mental disorders, and intellectual disabilities.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
China12 sites
Xiangya Hospital Central South University
Changsha, Hunan, 410008
The Second Affiliated Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 400010
Fuyang Hospital Affiliated to Anhui Medical University
Fuyang, Anhui, 236000
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Anhui Provincial Hospital
Hefei, Anhui, 230002
The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650106
The First Hospital of Lanzhou University
Lanzhou, Gansu, 730013
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, 330006
Nanjing Drug Tower Hospital
Nanjing, Jiangsu, 210008
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021
Hematology Hospital of Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Henan Tumor Hospital
Zhengzhou, Henan, 450003
About this study
Official title: A Phase III Clinical Study Evaluating the Efficacy and Safety of On-demand Treatment and Perioperative Replacement Therapy With Recombinant Human Coagulation Factor VIII for Injection in Treated Severe Hemophilia A
This study is a single arm, open, multicenter clinical trial in which the previous treated severe hemophilia A patients receive recombinant human coagulation factor VIII for injection to evaluate the efficacy and safety of on-demand treatment in the event of new bleeding events. If evaluable surgical cases occur, the overall efficacy and safety of recombinant human coagulation factor VIII for injection as a replacement therapy for severe hemophilia A PTPs during the perioperative period will be evaluated.
- Conditions
- Hemophilia A
- Being tested
- Recombinant human activated coagulation factor VIII for injection
- 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 activated coagulation factor VIII for injectionDrug
Recombinant human coagulation factor VIII for injection is a recombinant DNA product with functional characteristics comparable to endogenous factor VIII.
| Study group | Role | What they receive |
|---|---|---|
| Recombinant human activated coagulation factor Ⅷ for injection | Experimental | Each subject in this study receive on-demand treatment with recombinant human coagulation factor VIII for injection for 180 days, with an increase in medication frequency based on the relief after medication. Receives: Drug: Recombinant human activated coagulation factor VIII for injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of on-demand treatment improvement for newly occurring bleeding events
Evaluate the hemostatic efficacy of the patient after each new bleeding event (including new bleeding events during visits and new bleeding events during home treatment), and calculate the proportion of improvement as excellent+good to the total number of on-demand treatment evaluations.
Measured: For each new bleeding event, assessed within 72 hours after initial treatment
Activity recovery
The activity of Recombinant human coagulation factor VIII measured at 15 minutes after infusion minus the baseline Recombinant human coagulation factor VIII activity divided by the expected improvement in Recombinant human coagulation factor VIII activity, which reflects the improvement in Recombinant human coagulation factor VIII levels after infusion.
Measured: 15 minutes after infusion
Secondary outcomes (13)
The proportion of effective surgical hemostasis
Assess the hemostatic efficacy of injecting recombinant human coagulation factor VIII during and after surgery. The effective ratio is the proportion of excellent or good to the total times of the surgical evaluation of hemostatic efficacy
Measured: Within 72 hours after surgery
Annualized bleeding rate (ABR)
Including spontaneous and traumatic bleeding, as well as total bleeding; ABR can be calculated using the following formula: bleeding frequency/(treatment period days/365.25).
Measured: Up to 180 days.
The incidence of Less Han Expected Thermal Effect
Without the influence of confounding factors, continuously infuse Recombinant human coagulation factor VIII twice within 24 hours for on-demand treatment, with no response to the same bleeding event.
Measured: For each new bleeding event, assessed within 24 hours after initial treatment
The injection times of recombinant human coagulation factor VIII
The injection times of recombinant human coagulation factor VIII for each new bleeding event.
Measured: Up to 180 days.
The dosage of recombinant human coagulation factor VIII
The dosage of recombinant human coagulation factor VIII (including average and total doses) for each new bleeding event
Measured: Up to 180 days.
Changes of Haemophilia Joint Health Score before and after on-demand treatment
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: Up to 180 days.
Changes of Quality of Life Assessment before and after on-demand treatment
Quality of Life Assessment 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: Up to 180 days.
The incidence of recombinant human coagulation factor VIII inhibitors
The incidence of recombinant human coagulation factor VIII inhibitors
Measured: Up to 180 days
Incremental recovery in non-bleeding state
Calculated from the peak FVIII activity (expressed as Cmax) measured within 1 hour after the end of infusion.
Measured: Within 1 hour after infusion
The incidence of adverse events was assessed by vital signs
The incidence of adverse events was assessed by vital signs.
Measured: Up to 180 days
The incidence of adverse events was assessed by physical examination
The incidence of adverse events was assessed by physical examination.
Measured: Up to 180 days
The incidence of adverse events was assessed by electrocardiogram
The incidence of adverse events was assessed by electrocardiogram.
Measured: Up to 180 days
The incidence of adverse events was assessed by laboratory tests
The incidence of adverse events was assessed by laboratory tests.
Measured: Up to 180 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2024
- Study start12 Nov 2024
- Primary completionJul 2026 (estimated)
- Study completionJul 2026 (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 Nanjing Shunxin Pharmaceutical 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06297655
- Sponsor's protocol ID
- TQG202-III-01
- Status last verified
- Jun 2026
- First submitted
- 1 Mar 2024
- Last updated
- 12 Jun 2026
- 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 12 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Clinical Study Evaluating the Efficacy and Safety of On-demand Treatment and Perioperative Replacement Therapy With Recombinant Human Coagulation Factor VIII for Injection in Treated Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT06297655
- Acronym / sponsor ID
- TQG202-III-01
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 12 Nov 2024 (actual)
- Primary completion
- Jul 2026 (estimated)
- Study completion
- Jul 2026 (estimated)
- First posted
- 7 Mar 2024 (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 study is a single arm, open, multicenter clinical trial in which the previous treated severe hemophilia A patients receive recombinant human coagulation factor VIII for injection to evaluate the efficacy and safety of on-demand treatment in the event of new bleeding events. If evaluable surgical cases occur, the overall efficacy and safety of recombinant human coagulation factor VIII for injection as a replacement therapy for severe hemophilia A PTPs during the perioperative period will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Recombinant human activated coagulation factor Ⅷ for injection | Experimental | Drug: Recombinant human activated coagulation factor VIII for injectionEach subject in this study receive on-demand treatment with recombinant human coagulation factor VIII for injection for 180 days, with an increase in medication frequency based on the relief after medication. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- The subjects voluntarily joined this study, signed an informed consent form, and had good compliance;
- Age (calculated from the date of signing the informed consent form): ≥12 years and ≤65 years;
- Severe hemophilia A (coagulation factor VIII activity<1%), with exposure days (ED) of no less than 150 days prior to factor VIII (recombinant coagulation factor VIII or plasma derived coagulation factor VIII) treatment; Surgical patients need to have undergone at least 2 surgeries (including major surgeries) and plan to undergo elective surgery at the same time;
- The subjects must be HIV negative or have a viral load of < 200 particles/μ, HIV positive patients with l or < 400000 copies/ml.
- At baseline, no inhibitors were detected, and there was no history of Factor VIII inhibitor antibody formation (Bethesda method detection < 0.6 BU/ml, test result record required);
Key exclusion criteria (11 listed)
- Known congenital or acquired hemorrhagic diseases other than hemophilia A;
- The subjects have used or planned to receive immunosuppressive treatments such as prednisone, cyclophosphamide, and cyclosporine in the week prior to enrollment, and are allowed to use inhaled or topical corticosteroids;
- Those who are known or suspected to have hypersensitivity reactions to the investigational drug, including human embryonic kidney cell protein;
- Significant liver and kidney dysfunction, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 5 upper limit of normal (ULN), and serum creatinine >1.5 ULN;
- Hemoglobin < 60 g/L;
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 activated coagulation factor VIII for injectionRecombinant human coagulation factor VIII for injection is a recombinant DNA product with functional characteristics comparable to endogenous factor VIII. | Drug | Recombinant human activated coagulation factor Ⅷ for injection |
Primary outcome
The proportion of on-demand treatment improvement for newly occurring bleeding events
Time frame: For each new bleeding event, assessed within 72 hours after initial treatment
Evaluate the hemostatic efficacy of the patient after each new bleeding event (including new bleeding events during visits and new bleeding events during home treatment), and calculate the proportion of improvement as excellent+good to the total number of on-demand treatment evaluations.
Activity recovery
Time frame: 15 minutes after infusion
The activity of Recombinant human coagulation factor VIII measured at 15 minutes after infusion minus the baseline Recombinant human coagulation factor VIII activity divided by the expected improvement in Recombinant human coagulation factor VIII activity, which reflects the improvement in Recombinant human coagulation factor VIII levels after infusion.
13 secondary outcomes
The proportion of effective surgical hemostasis
Time frame: Within 72 hours after surgery
Annualized bleeding rate (ABR)
Time frame: Up to 180 days.
The incidence of Less Han Expected Thermal Effect
Time frame: For each new bleeding event, assessed within 24 hours after initial treatment
The injection times of recombinant human coagulation factor VIII
Time frame: Up to 180 days.
The dosage of recombinant human coagulation factor VIII
Time frame: Up to 180 days.
Changes of Haemophilia Joint Health Score before and after on-demand treatment
Time frame: Up to 180 days.
Changes of Quality of Life Assessment before and after on-demand treatment
Time frame: Up to 180 days.
The incidence of recombinant human coagulation factor VIII inhibitors
Time frame: Up to 180 days
Incremental recovery in non-bleeding state
Time frame: Within 1 hour after infusion
The incidence of adverse events was assessed by vital signs
Time frame: Up to 180 days
The incidence of adverse events was assessed by physical examination
Time frame: Up to 180 days
The incidence of adverse events was assessed by electrocardiogram
Time frame: Up to 180 days
The incidence of adverse events was assessed by laboratory tests
Time frame: Up to 180 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
12 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 activated coagulation factor VIII for injection in people with Hemophilia A, run by Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd. at 12 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 6 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 12 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of on-demand treatment improvement for newly occurring bleeding events (time frame: For each new bleeding event, assessed within 72 hours after initial treatment). 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 12 Jun 2026.