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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05605678
• Other study ID #: AP-0105 (PRESent-5)
• Status: Recruiting
• Start date: December 9, 2022
• Est. completion date: November 2024

Study information

• Verified date: May 2024
• Source: ApcinteX Ltd
• Contact: Centessa Pharmaceuticals
• Phone: 617 468 5770
• Email: presentprogram[@]centessa.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: November 2024
• Est. primary completion date: November 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Other:
• No Intervention
This is a non-interventional study.

Locations

Country	Name	City	State
Armenia	Centre of Haematology named after prof. R. O. Yeolian	Yerevan
Australia	Royal Prince Alfred Hospital	Camperdown
Brazil	Fundacao de Desenvolvimento da Unicamp FUNCAMP	Campina	São Paulo
Brazil	HEMOES - Centro de Hemoterapia e Hematologia do ES	Vitória
Canada	Hamilton Health Sciences Corporation	Hamilton	Ontario
Canada	Hamilton Health Sciences Corporation	Hamilton
Canada	Unity Health Toronto	Toronto
Egypt	Ain Shams University	Cairo
France	Hôpital Bicêtre	Le Kremlin Bicetre
France	Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)	Lyon	Rhone
France	CHU Hotel Dieu	Nantes
France	Hopital Necker - Enfants Malades	Paris
Germany	Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde	Berlin
Germany	Universitätsklinikum Carl Gustav Carus Dresden	Dresden	Sachsen
Germany	University Hospital Frankfurt	Frankfurt	Hesse
India	St. Johns Medical College	Bangalore
India	Christian Medical College & Hospital	Ludhiana	Punjab
India	K J Somaiya Super Speciality Hospital & Research Centre	Mumbai
Italy	Azienda Ospedaliero-Universitaria Careggi	Florence
Italy	Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milano
Poland	Korczowski Bartosz, Gabinet Lekarski	Rzeszów	Podkarpackie
Poland	Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie	Warszawa
Poland	Kl Hemat Now Krwi i Trans USK	Wroclaw
South Africa	King Edward VIII Hospital	Durban
South Africa	Phoenix Pharma (Pty) Ltd	Port Elizabeth	Eastern Cape
Spain	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
Taiwan	China Medical University Hospital	Taichung
Taiwan	Chung Shan Medical University	Taichung
Taiwan	Taichung Veterans General Hospital	Taichung
Taiwan	National Taiwan University Hospital	Taipei City
Turkey	Ege University Hospital	Izmir
Turkey	Ege University Medical Faculty Pediatric Hospital	Izmir
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	Imperial College Healthcare NHS Trust	London
United Kingdom	Royal Free London NHS Foundation Trust	London
United Kingdom	Central Manchester University Hospitals NHS Foundation Trust - Manchester Royal Infirmary	Manchester
United Kingdom	Royal Victoria Infirmary	Newcastle upon Tyne	Tyne And Wear
United Kingdom	Southampton General Hospital	Southampton
United States	University of Michigan	Ann Arbor	Michigan
United States	East Carolina univeristy	Greenville	South Carolina

Sponsors (2)

Lead Sponsor	Collaborator
ApcinteX Ltd	Centessa Pharmaceuticals plc

Countries where clinical trial is conducted

United States,  Armenia,  Australia,  Brazil,  Canada,  Egypt,  France,  Germany,  India,  Italy,  Poland,  South Africa,  Spain,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode		Minimum 12 weeks