PCC treatment for hemophilia A patients with inhibitors

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 trial tests whether giving prothrombin complex concentrate (PCC) during bleeding episodes can stop bleeds in people aged 12–65 with hemophilia A who have inhibitors.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 12 Years to 65 Years
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT07406139 on ClinicalTrials.gov

What this trial studies

This is a multicenter, prospective, single-arm study that gives on-demand PCC to patients with hemophilia A and inhibitors during bleeding episodes. Dosing was guided by investigators with a recommended 50 IU/kg per infusion every 8–12 hours and a maximum total daily dose of 150 IU/kg. If bleeding was not controlled within 24 hours, investigators could add other hemostatic agents or switch treatments. Safety and hemostatic effectiveness were recorded across participating centers.

Who should consider this trial

Good fit: Ideal candidates are people aged 12–65 with diagnosed hemophilia A and inhibitors who have had at least three joint bleeds in the past six months and can give informed consent.

Not a fit: Patients with other bleeding disorders, significant liver or kidney impairment, active immune tolerance induction with low-titer inhibitors, a history of thrombosis, or relevant allergies are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, PCC could provide an effective on-demand option to stop bleeding in patients with inhibitors when standard factor VIII replacement does not work.

How similar studies have performed: Bypassing agents such as activated PCC (aPCC) and recombinant activated factor VII (rFVIIa) have achieved >80% hemostatic success in prior studies, so the general bypassing approach has established efficacy though formulations and availability vary.

Eligibility criteria

Inclusion Criteria:

1. 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;
2. Age between 12 and 65 years;
3. At least three joint bleeding episodes within the past six months;
4. Signed informed consent form.

Exclusion Criteria:

1. Presence of other congenital or acquired bleeding disorders;
2. 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;
3. Currently receiving immune tolerance induction (ITI) therapy with an inhibitor titer \<5 BU;
4. History of thrombotic events;
5. Known history of drug allergy, asthma, urticaria, or other allergic conditions;
6. Deemed unsuitable for study participation by the investigator.

Where this trial is running

Tianjin, Tianjin Municipality

• Institute of haematology and Blood diseases hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: Mankai Ju

Conditions Hemophilia; Inhibitors