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

NCT05605678 | Terminated

• 1 other identifier: AP-0105 (PRESent-5)
• Study Type: observational
• Target: 108
• Locations: 16 countries
• Sites: 45

Brief Summary

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.

Conditions

Hemophilia a; Hemophilia B

Keywords

Hemophilia; Prospective

Outcome Measures

Primary Outcomes (2)

• Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds

  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

  Minimum 12 weeks

Study Arms (1)

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

Interventions

No Intervention OTHER

This is a non-interventional study.

All Eligible Participants

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Male participants greater than or equal to (\>=) 12 and less than or equal to (\<=) 65 years of age
• 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
• 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
• Who are able to use a diary to document bleeding events and associated treatment

You may not qualify if:

• With known thrombophilia
• With body weight greater than (\>)150 kilogram (kg) or body mass index \>40
• 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)
• With previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or stroke
• With history of intolerance to subcutaneous injections
• With known current uncontrolled hypertension (systolic blood pressure \>160 millimeter of mercury (mm Hg); diastolic blood pressure \>100 mm Hg; based on medical records)
• With active cancer or requires therapy for cancer, except for basal cell carcinoma
• With concurrent participation in an interventional clinical trial
• With current or planned use of emicizumab
• With prior, ongoing, or planned treatment with gene therapy for hemophilia
• 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)
• With known Human Immunodeficiency Virus (HIV) infection with CD4 count (or T-cell count) of \<200 cells/mcL within 24 weeks before enrollment
• With current or planned treatment with anticoagulant or antiplatelet drugs
• With any other significant conditions or comorbidities that, in the opinion of the Investigator or their designee, would make the participant unsuitable for enrollment

Sponsors & Collaborators

• ApcinteX Ltd: lead
• Centessa Pharmaceuticals plc: collaborator

Study Sites (45)

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

East Carolina univeristy

Greenville, South Carolina, 27834, United States

Location

Centre of Haematology named after prof. R. O. Yeolian

Yerevan, Yerevan, 0014, Armenia

Location

Royal Prince Alfred Hospital

Camperdown, Camperdown, NSW 2050, Australia

Location

Fundacao de Desenvolvimento da Unicamp FUNCAMP

Campina, São Paulo, 13083-878, Brazil

Location

HEMOES - Centro de Hemoterapia e Hematologia do ES

Vitória, Vitória, 29047105, Brazil

Location

Hamilton Health Sciences Corporation

Hamilton, Hamilton, L8S 4K1, Canada

Location

Hamilton Health Sciences Corporation

Hamilton, Ontario, L8S4K1, Canada

Location

Unity Health Toronto

Toronto, Toronto, M5B 1W8, Canada

Location

Ain Shams University

Cairo, Cairo Governorate, 1153, Egypt

Location

Hopital Necker - Enfants Malades

Paris, Paris, 75015, France

Location

Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)

Lyon, Rhone, 69500, France

Location

Hôpital Bicêtre

Le Kremlin-Bicêtre, 94270, France

Location

CHU Hotel Dieu

Nantes, 44000, France

Location

University Hospital Frankfurt

Frankfurt am Main, Hesse, 60590, Germany

Location

Universitätsklinikum Carl Gustav Carus Dresden

Dresden, Saxony, 1307, Germany

Location

Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde

Berlin, State of Berlin, 10249, Germany

Location

St. Johns Medical College

Bangalore, Bangalore, 560034, India

Location

K J Somaiya Super Speciality Hospital & Research Centre

Mumbai, Mumbai, 400022, India

Location

Christian Medical College & Hospital

Ludhiana, Punjab, 141008, India

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, Milano, 20122, Italy

Location

Azienda Ospedaliero-Universitaria Careggi

Florence, 50134, Italy

Location

Korczowski Bartosz, Gabinet Lekarski

Rzeszów, Podkarpackie Voivodeship, 35-302, Poland

Location

Kl Hemat Now Krwi i Trans USK

Wroclaw, Wrocław, 50-367, Poland

Location

King Edward VIII Hospital

Durban, Durban, 4001, South Africa

Location

Phoenix Pharma (Pty) Ltd

Port Elizabeth, Eastern Cape, 6001, South Africa

Location

Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil

Málaga, Malaga, 29010, Spain

Location

Hospital Clínico Universitario Virgen de la Arrixaca

Murcia, Murcia, 30120, Spain

Location

Hospital Universitario Miguel Servet

Zaragoza, Zaragoza, 50009, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 8035, Spain

Location

China Medical University Hospital

Taichung, Taichung, 40447, Taiwan

Location

Chung Shan Medical University

Taichung, 40201, Taiwan

Location

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

Location

National Taiwan University Hospital

Taipei, 100226, Taiwan

Location

Ege University Hospital

Izmir, İzmir, 35100, Turkey (Türkiye)

Location

Ege University Medical Faculty Pediatric Hospital

Izmir, 35100, Turkey (Türkiye)

Location

University Hospital of Wales

Cardiff, Cardiff, CF14 4XW, United Kingdom

Location

Glasgow Royal Infirmary

Glasgow, Glasgow, G4OSF, United Kingdom

Location

Barts and London School of Medicine and Dentistry

London, London, E1 2AT, United Kingdom

Location

Royal Free London NHS Foundation Trust

London, London, NW3 2QG, United Kingdom

Location

Imperial College Healthcare NHS Trust

London, London, W2 1NY, United Kingdom

Location

Southampton General Hospital

Southampton, Southampton, S016 6YD, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom

Location

Kent Canterbury Hospital

Canterbury, CT1 3NG, United Kingdom

Location

Central Manchester University Hospitals NHS Foundation Trust - Manchester Royal Infirmary

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 31, 2022
• First Posted: November 4, 2022
• Study Start: December 9, 2022
• Primary Completion: November 29, 2024
• Study Completion: November 29, 2024
• Last Updated: February 7, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

TU (2); AU (1); EG (1); IT (2); DE (3); IN (3); ZA (2); FR (4); GB (9); BR (2); US (2); CA (3); ES (4); AR (1); PL (2); TW (4)