A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

Phase 1

Recruiting

• Conditions: Severe Hemophilia A or Moderately Severe to Severe Hemophilia B; MedDRA version: 20.1Level: LLTClassification code: 10053754Term: Hemophilia B without inhibitors Class: 10010331; MedDRA version: 20.0Level: LLTClassification code: 10053753Term: Hemophilia A without inhibitors Class: 10010331; MedDRA version: 20.0Level: LLTClassification code: 10053751Term: Hemophilia A with anti factor VIII Class: 10010331; MedDRA version: 20.0Level: LLTClassification code: 10018937Term: Haemophilia A Class: 10010331; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2022-502880-39-00
• Lead Sponsor: Apcintex Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-04-12
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Male
• Target Recruitment: 154

Inclusion Criteria

Male subjects =12 and =65 years of age with severe HemA, with or without inhibitors, or moderately severe to severe HemB, without high titer inhibitor (high titer inhibitor defined as =5 Bethesda Units [BU]/mL) and not requiring current treatment with bypass agents., Subject is currently included in a prophylaxis program OR subject is undergoing an on-demand treatment regimen; if the latter, must have had =6 documented acute bleeding episodes that required treatment during the 6 months before Screening, At least 12 (Part 1) or 24 (Part 2) weeks of prospective documentation of bleeding episodes in the AP-0105 noninterventional study before dosing, or willing to complete the observation period in AP-0102, No bleeding in the 7 days before Baseline (observation period can be extended if actively bleeding), Adequate hematologic, hepatic, and renal function, and D-dimer of =750 µg/

Exclusion Criteria

Known severe thrombophilia, previous deep vein thrombosis (excluding line-associated thrombosis), pulmonary embolism, myocardial infarction, stroke, uncontrolled hypertension (>160/100 mmHg) or has active cancer and/or requires therapy for cancer, except for basal cell carcinoma, Subject with previous factor VIII or factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate, Body weight < 30 kg OR >150 kg OR body mass index >40 kg/m2, Use of emicizumab in the 24 weeks before Baseline (Day 0) / Ongoing, or planned treatment with gene therapy for hemophilia / Current or planned treatment with anticoagulant or antiplatelet drugs

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified