PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A）

NCT07406139 | Recruiting Phase 4

• 1 other identifier: IIT2022048
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Hemophilia; Inhibitors

Outcome Measures

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.

  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.

  From enrollment to the end of treatment at 24 hours

Study Arms (1)

PCC treatment

EXPERIMENTAL

Drug: PCC

Interventions

PCC DRUG

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.

PCC treatment

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• 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

Study Sites (1)

Institute of haematology and Blood diseases hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Mankai Ju

CONTACT

+8615620988690; jumankai@ihcams.ac.cn

Feng Xue

CONTACT

+862223608174; xuefeng@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2025
• First Posted: February 12, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): December 20, 2026
• Study Completion (Estimated): December 20, 2026
• Last Updated: February 12, 2026

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)