Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

February 6, 2025 updated by: ApcinteX Ltd

A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia

The primary objective of this study is to obtain prospective baseline documentation of annualized bleeding rates (ABRs) and treatment under standard-of-care (SOC) therapy among participants with hemophilia A or B. Participants in the study may be eligible to enroll in future planned interventional studies to be conducted by Sponsor.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

108

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Armenia
      • Yerevan, Armenia, 0014
        • Centre of Haematology named after prof. R. O. Yeolian
  • Australia
      • Camperdown, Australia, NSW 2050
        • Royal Prince Alfred Hospital
  • Brazil
      • Vitória, Brazil, 29047105
        • HEMOES - Centro de Hemoterapia e Hematologia do ES
    • São Paulo
      • Campina, São Paulo, Brazil, 13083-878
        • Fundacao de Desenvolvimento da Unicamp FUNCAMP
  • Canada
      • Hamilton, Canada, L8S 4K1
        • Hamilton Health Sciences Corporation
      • Toronto, Canada, M5B 1W8
        • Unity Health Toronto
    • Ontario
      • Hamilton, Ontario, Canada, L8S4K1
        • Hamilton Health Sciences Corporation
  • Egypt
      • Cairo, Egypt, 1153
        • Ain shams university
  • France
      • Le Kremlin Bicetre, France, 94270
        • Hôpital Bicêtre
      • Nantes, France, 44000
        • Chu Hotel Dieu
      • Paris, France, 75015
        • Hopital Necker - Enfants Malades
    • Rhone
      • Lyon, Rhone, France, 69500
        • Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
  • Germany
      • Berlin, Germany, 10249
        • Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde
    • Hesse
      • Frankfurt, Hesse, Germany, 60590
        • University Hospital Frankfurt
    • Sachsen
      • Dresden, Sachsen, Germany, 1307
        • Universitatsklinikum Carl Gustav Carus Dresden
  • India
      • Bangalore, India, 560034
        • St. Johns Medical College
      • Mumbai, India, 400022
        • K J Somaiya Super Speciality Hospital & Research Centre
    • Punjab
      • Ludhiana, Punjab, India, 141008
        • Christian Medical College & Hospital
  • Italy
      • Florence, Italy, 50134
        • Azienda Ospedaliero-Universitaria Careggi
      • Milano, Italy, 20122
        • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
  • Poland
      • Wrocław, Poland, 50-367
        • Kl Hemat Now Krwi i Trans USK
    • Podkarpackie
      • Rzeszów, Podkarpackie, Poland, 35-302
        • Korczowski Bartosz, Gabinet Lekarski
  • South Africa
      • Durban, South Africa, 4001
        • King Edward VIII Hospital
    • Eastern Cape
      • Port Elizabeth, Eastern Cape, South Africa, 6001
        • Phoenix Pharma (Pty) Ltd
  • Spain
      • Barcelona, Spain, 8035
        • Hospital Universitari Vall d'Hebron
      • Malaga, Spain, 29010
        • Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil
      • Murcia, Spain, 30120
        • Hospital Clínico Universitario Virgen de la Arrixaca
      • Zaragoza, Spain, 50009
        • Hospital Universitario Miguel Servet
  • Taiwan
      • Taichung, Taiwan, 40447
        • China Medical University Hospital
      • Taichung, Taiwan, 40705
        • Taichung Veterans General Hospital
      • Taichung, Taiwan, 40201
        • Chung Shan Medical University
      • Taipei City, Taiwan, 100226
        • National Taiwan University Hospital
  • Turkey
      • Izmir, Turkey, 35100
        • Ege University Hospital
      • Izmir, Turkey, 35100
        • Ege University Medical Faculty Pediatric Hospital
  • United Kingdom
      • Canterbury, United Kingdom, CT1 3NG
        • Kent Canterbury Hospital
      • Cardiff, United Kingdom, CF14 4XW
        • University Hospital of Wales
      • Glasgow, United Kingdom, G4OSF
        • Glasgow Royal Infirmary
      • London, United Kingdom, NW3 2QG
        • Royal Free London NHS Foundation Trust
      • London, United Kingdom, W2 1NY
        • Imperial College Healthcare NHS Trust
      • London, United Kingdom, E1 2AT
        • Barts and London School of Medicine and Dentistry
      • Manchester, United Kingdom, M13 9WL
        • Central Manchester University Hospitals NHS Foundation Trust - Manchester Royal Infirmary
      • Southampton, United Kingdom, S016 6YD
        • Southampton General Hospital
    • Tyne and Wear
      • Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE1 4LP
        • Royal Victoria Infirmary
  • United States
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • South Carolina
      • Greenville, South Carolina, United States, 27834
        • East Carolina univeristy

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants with severe HemA with or without inhibitors, or moderately severe to severe HemB without inhibitors or HemB with inhibitors treated with SOC on-demand or prophylactic hemophilia therapy.

Description

Inclusion Criteria:

  1. Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age
  2. Who are capable of providing written informed consent (adolescent assent and parental/guardian consent when appropriate) for participation after reading the information and consent form and having the opportunity to discuss the study with the Investigator or their designee
  3. With historically documented severe HemA (defined as factor VIII [FVIII] less than (<) 0.01 International Units per milliliter (IU/mL) [<1 percent {%}]), with or without inhibitors, or moderately severe to severe HemB (defined as factor IX [FIX] <=0.02 IU/mL [<=2%]) without inhibitors. Participants must be currently included in a prophylactic treatment program or if undergoing an on-demand treatment regimen must have had >=6 documented acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months before enrollment OR: Historically documented HemB (defined as FIX <=0.05 IU/mL [<=5%]) with inhibitors with a historical or ongoing high titer inhibitor [>=5 Bethesda Units/mL] based on medical records or laboratory reports) and an ABR of >=6 in the 6 months before enrollment
  4. Who are able to use a diary to document bleeding events and associated treatment

Exclusion Criteria:

  1. With known thrombophilia
  2. With body weight greater than (>)150 kilogram (kg) or body mass index >40
  3. With known current inadequate hematologic function (eg, platelet count <100,000 per microliter [/mcL] and/or hemoglobin level <10 grams per deciliter [g/dL], <100 g/L), hepatic function (that is, total bilirubin >1.5*upper limit of normal [ULN] [excluding Gilbert syndrome], aspartate transferase and/or alanine aminotransferase levels >3*ULN; clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver) or renal function (that is, serum creatinine >2*ULN; based on medical records or available laboratory reports)
  4. With previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or stroke
  5. With history of intolerance to subcutaneous injections
  6. With known current uncontrolled hypertension (systolic blood pressure >160 millimeter of mercury (mm Hg); diastolic blood pressure >100 mm Hg; based on medical records)
  7. With active cancer or requires therapy for cancer, except for basal cell carcinoma
  8. With concurrent participation in an interventional clinical trial
  9. With current or planned use of emicizumab
  10. With prior, ongoing, or planned treatment with gene therapy for hemophilia
  11. With history of or other evidence of recent alcohol or drug abuse as determined by the Investigator or their designee (in the 12 months before enrollment)
  12. With known Human Immunodeficiency Virus (HIV) infection with CD4 count (or T-cell count) of <200 cells/mcL within 24 weeks before enrollment
  13. With current or planned treatment with anticoagulant or antiplatelet drugs
  14. With any other significant conditions or comorbidities that, in the opinion of the Investigator or their designee, would make the participant unsuitable for enrollment

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
All Eligible Participants
All Participants diagnosed with severe HemA with or without inhibitors, moderately severe to severe HemB, or HemB with inhibitors will be enrolled and continue to receive their usual hemophilia treatment regimen under SOC therapy. Bleeding episodes and treatment data will be collected during the prospective follow up period in a diary. No intervention will be administered as part of this study.
Other: No Intervention
This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
Time Frame: Minimum 12 weeks
ABR for treated bleeds and all bleeds at the participant and bleed level (excluding bleeding episodes during surgery and/or procedures).
Minimum 12 weeks
Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
Time Frame: Minimum 12 weeks
Minimum 12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ApcinteX Ltd

Collaborators

Centessa Pharmaceuticals plc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 9, 2022

Primary Completion (Actual)

November 29, 2024

Study Completion (Actual)

November 29, 2024

Study Registration Dates

First Submitted

October 31, 2022

First Submitted That Met QC Criteria

October 31, 2022

First Posted (Actual)

November 4, 2022

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

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AP-0105 (PRESent-5)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

There is no plan to share IPD due to the Sponsor's strategic and data-driven decision to discontinue the global clinical development of SerpinPC.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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