The Long-term Safety and Efficacy of SerpinPC in Subjects with Hemophilia Who Completed a Sponsored SerpinPC Clinical Trial (PRESent-6)

A Global Open-label Extension Study to Observe the Long-term Safety and Efficacy of SerpinPC in Subjects with Hemophilia Who Completed a Sponsored SerpinPC Clinical Trial

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with hemophilia

Study Overview

• Status: Terminated
• Conditions: Hemophilia B; Hemophilia a; Hemophilia a with Inhibitor; Hemophilia B with Inhibitor
• Intervention / Treatment: Drug: SerpinPC

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 3

Contacts and Locations

Study Locations

• Georgia
  • Tbilisi, Georgia, 0112
    • Arensia Exploratory Medicine Llc
• Moldova, Republic of
  • Chisinau, Moldova, Republic of, MD-2025
    • Institute of Oncology, ARENSIA Exploratory Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age at the time of informed consent
• Completed participation in a sponsored SerpinPC hemophilia clinical trial and, in the opinion of the investigator, was compliant with the study including compliance with diary entries.
• Capable of providing written informed consent (adolescent assent and parent/guardian consent when appropriate) for participation

Exclusion Criteria:

• Previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or embolic stroke
• Participation in another interventional clinical trial, except for SerpinPC trials
• Any other significant conditions or comorbidities that, in the opinion of the investigator, would make the subject unsuitable for enrolment or could interfere with participation in or completion of the study
• Treatment with anticoagulant or antiplatelet drugs

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SerpinPC; Participants will receive SerpinPC 1.2 milligrams/kilograms (mg/kg) subcutaneous injection every 2 weeks (Q2W) once they have completed treatment in a SerpinPC parent study.	Drug: SerpinPC; Administered as SC injection.; Other Names: Activated Protein C (APC) inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Treated bleeds, expressed as annualized bleeding rate (ABR)	Month 0 to Month 25 or Early termination

Secondary Outcome Measures

Outcome Measure	Time Frame
Treated spontaneous bleeds (expressed as ABR)	Month 0 to Month 25 or Early termination
Treated spontaneous joint bleeds (expressed as ABR)	Month 0 to Month 25 or Early termination
All bleeds requiring treatment (expressed as ABR, ie, all treated bleeds and all bleeds that would ordinarily be treated with factor concentrate/bypass agent if therapy were available)	Month 0 to Month 25 or Early termination
Total coagulation factor and/or bypass product consumption	Month 0 to Month 25 or Early termination

Collaborators and Investigators

• Sponsor: ApcinteX Ltd
• Collaborators: Centessa Pharmaceuticals plc

Study record dates

Study Major Dates

• Study Start (Actual): July 11, 2024
• Primary Completion (Actual): January 29, 2025
• Study Completion (Actual): January 29, 2025

Study Registration Dates

• First Submitted: August 21, 2024
• First Submitted That Met QC Criteria: August 21, 2024
• First Posted (Actual): August 23, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 6, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Hemophilia; SerpinPC
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A; Hemophilia B; Molecular Mechanisms of Pharmacological Action; Fibrin Modulating Agents; Fibrinolytic Agents; Anticoagulants; Protein C
• Other Study ID Numbers: AP-0106; 2023-509965-19 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No