NCT05789524

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (actual)
• Sponsor: ApcinteX Ltd · Industry
• Sex: Male
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program. This study consists of 3 parts: Part 1: dose-justification phase, Part 2: dose-confirmatory phase, Part 3: extension phase for participants who complete either Part 1 or Part 2. This adaptive design study has a randomized dose-justification component to investigate the efficacy and safety of SerpinPC as a therapeutic option, principally for participants with HemB without inhibitors. SerpinPC has a novel mechanism of action compared with marketed treatments and those that are in development.

Conditions

• Hemophilia a
• Hemophilia B

Interventions

Type	Name	Description
DRUG	SerpinPC	Administered as SC injection.

Timeline

• Start date: 2023-07-06
• Primary completion: 2024-11-14
• Completion: 2025-02-28
• First posted: 2023-03-29
• Last updated: 2025-03-11

Locations

59 sites across 15 countries: United States, Armenia, Australia, Belgium, Canada, France, Germany, India, Italy, Poland, South Africa, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study