PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A）
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age12-65
SponsorInstitute of Hematology & Blood Diseases Hospital, China
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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;
Trial design
Design model
Single group
Treatments tested in this trial
PCC
DrugOn-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.
Treatment groups
30 Participants
are divided into 1 treatment groupGroup A: PCC treatmentExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
From enrollment to the end of treatment at 24 hoursSecondary 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.
Time frame
From enrollment to the end of treatment at 24 hoursSponsors and contacts
Click on the lead sponsor to view all of their trials.
Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
North China University of Science and Technology
Collaborator
The Second Affiliated Hospital of Kunming Medical University
Collaborator
The Second People's Hospital of Anhui Province
Collaborator
Children's Hospital of Chongqing Medical University
Collaborator
Union Hospital, Affiliated to Fujian Medical University
Collaborator
The First Affiliated Hospital of Xiamen University
Collaborator
First Affiliated Hospital of Guangxi Medical University
Collaborator
Source ClinicalTrials.gov