HemophiliaPhase 42022PCC-ANCT07406139
PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A）
A Phase 4 study testing PCC in people with Hemophilia and Inhibitors, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This study is a multicenter, prospective, single-arm exploratory clinical trial designed to evaluate the efficacy and safety of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Hemophilia, Inhibitors
- What is being tested?
- PCC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2026to 20 Dec 2026
- Phase
- Phase 4
- 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
- Diagnosed with hemophilia A with inhibitors. For high-responding patients (those who have previously had an inhibitor titer >5 BU), the inhibitor titer at enrollment must be >0.6 BU;
- Age between 12 and 65 years;
- At least three joint bleeding episodes within the past six months;
- Signed informed consent form.
Who cannot join
- Presence of other congenital or acquired bleeding disorders;
- Liver function tests (ALT, AST) >2.5 times the upper limit of normal, or renal function tests (BUN, Cr) >1.5 times the upper limit of normal;
- Currently receiving immune tolerance induction (ITI) therapy with an inhibitor titer <5 BU;
- History of thrombotic events;
- Known history of drug allergy, asthma, urticaria, or other allergic conditions;
- Deemed unsuitable for study participation by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of haematology and Blood diseases hospital
Tianjin, Tianjin Municipality, 300020
Mankai Ju, +8615620988690, jumankai@ihcams.ac.cn
Lei Zhang, 2223608177, zhanglei1@ihcams.ac.cn
About this study
Official title: A Multicenter, Prospective, Single-arm Exploratory Study of Prothrombin Complex Concentrate in the Treatment of Bleeding Episodes in Patients With Hemophilia A With Inhibitors
This study is a multicenter, prospective, single-arm exploratory clinical trial designed to evaluate the efficacy and safety of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors. All participants received on-demand PCC therapy during bleeding episodes, with dosing adjusted by investigators according to the type of bleeding. The recommended dose was 50 IU/kg per infusion, administered every 8-12 hours, with a maximum total daily dose not exceeding 150 IU/kg. If no effective hemostasis was achieved within 24 hours, investigators could decide to add other hemostatic agents or switch to alternative treatments.
Read the detailed description
Hemophilia A is a severe and common hereditary bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII). Recurrent bleeding episodes, if not treated promptly, can lead to joint deformities or pseudotumor formation and may even be life-threatening in severe cases. In recent years, with the widespread use of plasma-derived and recombinant FVIII products, the management of hemophilia A has made significant progress. Increasing numbers of patients are now receiving prophylactic care and appropriate surgical interventions, resulting in substantial improvements in quality of life.
However, the incidence of inhibitors in hemophilia A has been rising. Approximately 30% of patients with severe hemophilia A and 3-13% of those with non-severe disease develop inhibitors. The presence of inhibitors neutralizes FVIII activity, rendering FVIII replacement therapy ineffective, thereby increasing the risk of life-threatening bleeding and contributing to severe joint damage. Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are two commonly used "bypassing agents" worldwide. These agents achieve hemostasis by generating thrombin through pathways that bypass the need for FVIII or FIX activation.
According to Cochrane systematic reviews, both aPCC and rFVIIa achieve hemostatic efficacy rates above 80%, demonstrate similar tolerability, and have a low incidence of thrombotic complications. However, since aPCC is not available in China and rFVIIa is costly, prothrombin complex concentrate (PCC) is generally used as an alternative. The 2020 Chinese Guidelines for the Management of Hemophilia recommend PCC and rFVIIa as bypassing options for patients with high-titer inhibitors (>5 BU/mL), those who fail immune tolerance induction (ITI), or those who experience bleeding during ITI therapy.
PCC is a plasma-derived concentrate obtained from pooled plasma of healthy donors, containing mainly vitamin K-dependent coagulation factors II, VII, IX, and X. It is categorized as either a four-factor PCC (containing therapeutic levels of FVII, as well as anticoagulant proteins C and S) or a three-factor PCC (with lower levels of FVII and anticoagulant proteins). Four-factor PCC can enhance thrombin generation directly or indirectly on platelet surfaces, with elevated FVII levels likely being a key contributor to its procoagulant effect. Meanwhile, the presence of balancing anticoagulant proteins C and S may help mitigate excessive coagulation and reduce thrombotic risk.
To date, no prospective clinical study has been conducted in China to evaluate the efficacy and safety of PCC in the management or prophylaxis of bleeding in hemophilia A patients with inhibitors; available data are limited to retrospective analyses and case reports. Considering the current situation in China, a prospective evaluation of the efficacy and safety of four-factor PCC for hemostasis in hemophilia A patients with inhibitors is of great importance, as it can provide more objective and scientific evidence for clinical decision-making. In addition, since patient-specific factors, the mechanisms of bypassing agents, and pharmacokinetic variability can all lead to differing treatment responses, this study also incorporates thrombin generation assay (TGA) testing to assess individual patient responses and provide further objective data supporting the hemostatic efficacy of four-factor PCC.
- Conditions
- Hemophilia, Inhibitors
- Being tested
- PCC
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PCCDrug
On-demand PCC therapy during bleeding episodes. The recommended dose was 50 IU/kg per infusion, administered every 8-12 hours, with a maximum total daily dose not exceeding 150 IU/kg.
| Study group | Role | What they receive |
|---|---|---|
| PCC treatment | Experimental | Not described Receives: Drug: PCC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors.
Hemostatic Efficacy Rate:
Bleeding symptoms were evaluated at 8 hours and 24 hours after treatment. The assessment of hemostatic response included four categories: excellent, good, moderate, and ineffective.
Measured: From enrollment to the end of treatment at 24 hours
Secondary outcomes (1)
To evaluate the improvement score of bleeding symptoms and signs within 24 hours after the first infusion of prothrombin complex concentrate (PCC) for each bleeding episode in patients with hemophilia A with inhibitors.
Measured: From enrollment to the end of treatment at 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2026
- Study start1 Mar 2026 (estimated)
- Primary completion20 Dec 2026 (estimated)
- Study completion20 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- North China University of Science and Technology, The Second Affiliated Hospital of Kunming Medical University, The Second People's Hospital of Anhui Province, Children's Hospital of Chongqing Medical University, Union Hospital, Affiliated to Fujian Medical University, The First Affiliated Hospital of Xiamen University, First Affiliated Hospital of Guangxi Medical University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07406139
- Sponsor's protocol ID
- IIT2022048
- Acronym
- 2022PCC-A
- Status last verified
- Oct 2025
- First submitted
- 20 Nov 2025
- Last updated
- 12 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Prospective, Single-arm Exploratory Study of Prothrombin Complex Concentrate in the Treatment of Bleeding Episodes in Patients With Hemophilia A With Inhibitors
- ClinicalTrials.gov ID
- NCT07406139
- Acronym / sponsor ID
- 2022PCC-A / IIT2022048
- Conditions
- Hemophilia, Inhibitors
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- North China University of Science and Technology, The Second Affiliated Hospital of Kunming Medical University, The Second People's Hospital of Anhui Province, Children's Hospital of Chongqing Medical University, Union Hospital, Affiliated to Fujian Medical University, The First Affiliated Hospital of Xiamen University, First Affiliated Hospital of Guangxi Medical University
- Study start
- 1 Mar 2026 (estimated)
- Primary completion
- 20 Dec 2026 (estimated)
- Study completion
- 20 Dec 2026 (estimated)
- First posted
- 12 Feb 2026 (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 multicenter, prospective, single-arm exploratory clinical trial designed to evaluate the efficacy and safety of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors. All participants received on-demand PCC therapy during bleeding episodes, with dosing adjusted by investigators according to the type of bleeding. The recommended dose was 50 IU/kg per infusion, administered every 8-12 hours, with a maximum total daily dose not exceeding 150 IU/kg. If no effective hemostasis was achieved within 24 hours, investigators could decide to add other hemostatic agents or switch to alternative treatments.
Detailed description
Hemophilia A is a severe and common hereditary bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII). Recurrent bleeding episodes, if not treated promptly, can lead to joint deformities or pseudotumor formation and may even be life-threatening in severe cases. In recent years, with the widespread use of plasma-derived and recombinant FVIII products, the management of hemophilia A has made significant progress. Increasing numbers of patients are now receiving prophylactic care and appropriate surgical interventions, resulting in substantial improvements in quality of life.
However, the incidence of inhibitors in hemophilia A has been rising. Approximately 30% of patients with severe hemophilia A and 3-13% of those with non-severe disease develop inhibitors. The presence of inhibitors neutralizes FVIII activity, rendering FVIII replacement therapy ineffective, thereby increasing the risk of life-threatening bleeding and contributing to severe joint damage. Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are two commonly used "bypassing agents" worldwide. These agents achieve hemostasis by generating thrombin through pathways that bypass the need for FVIII or FIX activation.
According to Cochrane systematic reviews, both aPCC and rFVIIa achieve hemostatic efficacy rates above 80%, demonstrate similar tolerability, and have a low incidence of thrombotic complications. However, since aPCC is not available in China and rFVIIa is costly, prothrombin complex concentrate (PCC) is generally used as an alternative. The 2020 Chinese Guidelines for the Management of Hemophilia recommend PCC and rFVIIa as bypassing options for patients with high-titer inhibitors (>5 BU/mL), those who fail immune tolerance induction (ITI), or those who experience bleeding during ITI therapy.
PCC is a plasma-derived concentrate obtained from pooled plasma of healthy donors, containing mainly vitamin K-dependent coagulation factors II, VII, IX, and X. It is categorized as either a four-factor PCC (containing therapeutic levels of FVII, as well as anticoagulant proteins C and S) or a three-factor PCC (with lower levels of FVII and anticoagulant proteins). Four-factor PCC can enhance thrombin generation directly or indirectly on platelet surfaces, with elevated FVII levels likely being a key contributor to its procoagulant effect. Meanwhile, the presence of balancing anticoagulant proteins C and S may help mitigate excessive coagulation and reduce thrombotic risk.
To date, no prospective clinical study has been conducted in China to evaluate the efficacy and safety of PCC in the management or prophylaxis of bleeding in hemophilia A patients with inhibitors; available data are limited to retrospective analyses and case reports. Considering the current situation in China, a prospective evaluation of the efficacy and safety of four-factor PCC for hemostasis in hemophilia A patients with inhibitors is of great importance, as it can provide more objective and scientific evidence for clinical decision-making. In addition, since patient-specific factors, the mechanisms of bypassing agents, and pharmacokinetic variability can all lead to differing treatment responses, this study also incorporates thrombin generation assay (TGA) testing to assess individual patient responses and provide further objective data supporting the hemostatic efficacy of four-factor PCC.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PCC treatment | Experimental | Drug: PCC |
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)
- Diagnosed with hemophilia A with inhibitors. For high-responding patients (those who have previously had an inhibitor titer >5 BU), the inhibitor titer at enrollment must be >0.6 BU;
- Age between 12 and 65 years;
- At least three joint bleeding episodes within the past six months;
- Signed informed consent form.
Key exclusion criteria (6 listed)
- Presence of other congenital or acquired bleeding disorders;
- Liver function tests (ALT, AST) >2.5 times the upper limit of normal, or renal function tests (BUN, Cr) >1.5 times the upper limit of normal;
- Currently receiving immune tolerance induction (ITI) therapy with an inhibitor titer <5 BU;
- History of thrombotic events;
- Known history of drug allergy, asthma, urticaria, or other allergic conditions;
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 |
|---|---|---|
| PCCOn-demand PCC therapy during bleeding episodes. The recommended dose was 50 IU/kg per infusion, administered every 8-12 hours, with a maximum total daily dose not exceeding 150 IU/kg. | Drug | PCC treatment |
Primary outcome
Efficacy of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors.
Time frame: From enrollment to the end of treatment at 24 hours
Hemostatic Efficacy Rate: Bleeding symptoms were evaluated at 8 hours and 24 hours after treatment. The assessment of hemostatic response included four categories: excellent, good, moderate, and ineffective.
1 secondary outcome
To evaluate the improvement score of bleeding symptoms and signs within 24 hours after the first infusion of prothrombin complex concentrate (PCC) for each bleeding episode in patients with hemophilia A with inhibitors.
Time frame: From enrollment to the end of treatment at 24 hours
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 Feb 2026.
- Individual participant dataSharing planned: Yes. Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing PCC in people with Hemophilia and Inhibitors, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site 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 6 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: looking for participants now, as of the sponsor's last update on 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Contact Mankai Ju (+8615620988690, jumankai@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors. (time frame: From enrollment to the end of treatment at 24 hours). 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 Feb 2026.