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

Stopped early

Conditions studied: Hemophilia a, Hemophilia B

In brief

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.

Key facts

Study ID: NCT05605678
Run by: ApcinteX Ltd
People needed: 108
Starts: 2022-12-09
Expected to finish: 2024-11-29
Last updated by the study team: 2025-02-07

Who can join

Age: 12 and older, up to 65. Sex: male. Healthy volunteers: not accepted.

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

Where it is running

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, Yerevan, Armenia
Royal Prince Alfred Hospital — Camperdown, Camperdown, Australia
Fundacao de Desenvolvimento da Unicamp FUNCAMP — Campina, São Paulo, Brazil
HEMOES - Centro de Hemoterapia e Hematologia do ES — Vitória, Vitória, Brazil
Hamilton Health Sciences Corporation — Hamilton, Hamilton, Canada
Hamilton Health Sciences Corporation — Hamilton, Ontario, Canada
Unity Health Toronto — Toronto, Toronto, Canada
Ain Shams University — Cairo, Cairo Governorate, Egypt
Hopital Necker - Enfants Malades — Paris, Paris, France
Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME) — Lyon, Rhone, France
Hôpital Bicêtre — Le Kremlin-Bicêtre, France
CHU Hotel Dieu — Nantes, France
University Hospital Frankfurt — Frankfurt am Main, Hesse, Germany
Universitätsklinikum Carl Gustav Carus Dresden — Dresden, Saxony, Germany
Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde — Berlin, State of Berlin, Germany
St. Johns Medical College — Bangalore, Bangalore, India
K J Somaiya Super Speciality Hospital & Research Centre — Mumbai, Mumbai, India
Christian Medical College & Hospital — Ludhiana, Punjab, India
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico — Milan, Milano, Italy
Azienda Ospedaliero-Universitaria Careggi — Florence, Italy
Korczowski Bartosz, Gabinet Lekarski — Rzeszów, Podkarpackie Voivodeship, Poland
Kl Hemat Now Krwi i Trans USK — Wroclaw, Wrocław, Poland
King Edward VIII Hospital — Durban, Durban, South Africa

Full record on ClinicalTrials.gov

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