Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)
- Conditions
- Hemophilia aHemophilia B
- Interventions
- Other: No Intervention
- Registration Number
- NCT05605678
- Lead Sponsor
- ApcinteX Ltd
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 108
- 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
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description All Eligible Participants No Intervention 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.
- Primary Outcome Measures
Name Time Method Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode Minimum 12 weeks Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds Minimum 12 weeks ABR for treated bleeds and all bleeds at the participant and bleed level (excluding bleeding episodes during surgery and/or procedures).
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (44)
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
East Carolina univeristy
🇺🇸Greenville, South Carolina, United States
Centre of Haematology named after prof. R. O. Yeolian
🇦🇲Yerevan, Armenia
Royal Prince Alfred Hospital
🇦🇺Camperdown, Australia
Fundacao de Desenvolvimento da Unicamp FUNCAMP
🇧🇷Campina, São Paulo, Brazil
HEMOES - Centro de Hemoterapia e Hematologia do ES
🇧🇷Vitória, Brazil
Hamilton Health Sciences Corporation
🇨🇦Hamilton, Canada
Unity Health Toronto
🇨🇦Toronto, Canada
Ain Shams University
🇪🇬Cairo, Egypt
Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
🇫🇷Lyon, Rhone, France
Hôpital Bicêtre
🇫🇷Le Kremlin Bicetre, France
CHU Hotel Dieu
🇫🇷Nantes, France
Hopital Necker - Enfants Malades
🇫🇷Paris, France
University Hospital Frankfurt
🇩🇪Frankfurt, Hesse, Germany
Universitätsklinikum Carl Gustav Carus Dresden
🇩🇪Dresden, Sachsen, Germany
Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde
🇩🇪Berlin, Germany
Christian Medical College & Hospital
🇮🇳Ludhiana, Punjab, India
St. Johns Medical College
🇮🇳Bangalore, India
K J Somaiya Super Speciality Hospital & Research Centre
🇮🇳Mumbai, India
Azienda Ospedaliero-Universitaria Careggi
🇮🇹Florence, Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Korczowski Bartosz, Gabinet Lekarski
🇵🇱Rzeszów, Podkarpackie, Poland
Kl Hemat Now Krwi i Trans USK
🇵🇱Wrocław, Poland
Phoenix Pharma (Pty) Ltd
🇿🇦Port Elizabeth, Eastern Cape, South Africa
King Edward VIII Hospital
🇿🇦Durban, South Africa
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil
🇪🇸Malaga, Spain
Hospital Clínico Universitario Virgen de la Arrixaca
🇪🇸Murcia, Spain
Hospital Universitario Miguel Servet
🇪🇸Zaragoza, Spain
Chung Shan Medical University
🇨🇳Taichung, Taiwan
China Medical University Hospital
🇨🇳Taichung, Taiwan
Taichung Veterans General Hospital
🇨🇳Taichung, Taiwan
National Taiwan University Hospital
🇨🇳Taipei City, Taiwan
Ege University Hospital
🇹🇷Izmir, Turkey
Ege University Medical Faculty Pediatric Hospital
🇹🇷Izmir, Turkey
Royal Victoria Infirmary
🇬🇧Newcastle upon Tyne, Tyne and Wear, United Kingdom
Kent Canterbury Hospital
🇬🇧Canterbury, United Kingdom
University Hospital of Wales
🇬🇧Cardiff, United Kingdom
Glasgow Royal Infirmary
🇬🇧Glasgow, United Kingdom
Barts and London School of Medicine and Dentistry
🇬🇧London, United Kingdom
Royal Free London NHS Foundation Trust
🇬🇧London, United Kingdom
Imperial College Healthcare NHS Trust
🇬🇧London, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust - Manchester Royal Infirmary
🇬🇧Manchester, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom