Clinical trial NCT05605678: Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)
Official title: A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hemophilia a
Hemophilia B
Keywords: Hemophilia
Prospective
Lead sponsor: ApcinteX Ltd
Sponsor class: INDUSTRY
Collaborators: name: Centessa Pharmaceuticals plc
First posted: 2022-11-04
Enrollment: 108
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
timeFrame: Minimum 12 weeks
measure: Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
timeFrame: Minimum 12 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Greenville
country: United States
facility: East Carolina univeristy
state: South Carolina
city: Yerevan
country: Armenia
facility: Centre of Haematology named after prof. R. O. Yeolian
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
city: Campina
country: Brazil
facility: Fundacao de Desenvolvimento da Unicamp FUNCAMP
state: São Paulo
city: Vitória
country: Brazil
facility: HEMOES - Centro de Hemoterapia e Hematologia do ES
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Corporation
state: Ontario
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Corporation
city: Toronto
country: Canada
facility: Unity Health Toronto
city: Cairo
country: Egypt
facility: Ain Shams University
city: Lyon
country: France
facility: Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
state: Rhone
city: Le Kremlin Bicetre
country: France
facility: Hôpital Bicêtre
city: Nantes
country: France
facility: CHU Hotel Dieu
city: Paris
country: France
facility: Hopital Necker - Enfants Malades
city: Frankfurt
country: Germany
facility: University Hospital Frankfurt
state: Hesse
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus Dresden
state: Sachsen
city: Berlin
country: Germany
facility: Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde
city: Ludhiana
country: India
facility: Christian Medical College & Hospital
state: Punjab
city: Bangalore
country: India
facility: St. Johns Medical College
city: Mumbai
country: India
facility: K J Somaiya Super Speciality Hospital & Research Centre
city: Florence
country: Italy
facility: Azienda Ospedaliero-Universitaria Careggi
city: Milano
country: Italy
facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
city: Rzeszów
country: Poland
facility: Korczowski Bartosz, Gabinet Lekarski
state: Podkarpackie
city: Wrocław
country: Poland
facility: Kl Hemat Now Krwi i Trans USK
city: Port Elizabeth
country: South Africa
facility: Phoenix Pharma (Pty) Ltd
state: Eastern Cape
city: Durban
country: South Africa
facility: King Edward VIII Hospital
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil
city: Murcia
country: Spain
facility: Hospital Clínico Universitario Virgen de la Arrixaca
city: Zaragoza
country: Spain
facility: Hospital Universitario Miguel Servet
city: Taichung
country: Taiwan
facility: Chung Shan Medical University
city: Taichung
country: Taiwan
facility: China Medical University Hospital
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Taipei City
country: Taiwan
facility: National Taiwan University Hospital
city: Izmir
country: Turkey
facility: Ege University Hospital
city: Izmir
country: Turkey
facility: Ege University Medical Faculty Pediatric Hospital
city: Newcastle upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
state: Tyne and Wear
city: Canterbury
country: United Kingdom
facility: Kent Canterbury Hospital
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: Glasgow
country: United Kingdom
facility: Glasgow Royal Infirmary
city: London
country: United Kingdom
facility: Barts and London School of Medicine and Dentistry
city: London
country: United Kingdom
facility: Royal Free London NHS Foundation Trust
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospitals NHS Foundation Trust - Manchester Royal Infirmary
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
References: