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)
Key facts
- Where
- Florence, Milan, Rozzano, Turin, Udine és Verona, OlaszországSee the 6 sites
- Phase
- 2. fázis
Where in Olaszország
- Careggi University HospitalFlorence, OlaszországNo longer taking part
- Fondazione IRCCS Cà Granda Ospedale Maggiore PoliclinicoMilan, OlaszországNo longer taking part
- Humanitas Mirasole S.p.A.Rozzano, OlaszországNo longer taking part
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoTurin, OlaszországNo longer taking part
- Azienda Sanitaria Universitaria Friuli CentraleUdine, OlaszországNo longer taking part
- Azienda Ospedaliera Universitaria Integrata VeronaVerona, OlaszországNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Apcintex Limited
- Trial code
- EU 2022-502880-39-00
- Record updated
- 2024. október 21.
- Original description
To evaluate the efficacy and safety of prophylactic SerpinPC administered subcutaneously (SC) in subjects with moderately severe to severe hemophilia B (HemB)Read full original descriptionShow less
To evaluate the efficacy and safety of prophylactic SerpinPC administered subcutaneously (SC) in subjects with moderately severe to severe hemophilia B (HemB)
Inclusion: 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/L.
Exclusion: 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; 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; Body weight < 30 kg OR >150 kg OR body mass index >40 kg/m2.