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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05605678




Registration number
NCT05605678
Ethics application status
Date submitted
31/10/2022
Date registered
4/11/2022
Date last updated
19/09/2024

Titles & IDs
Public title
Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)
Scientific title
A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients With Hemophilia
Secondary ID [1] 0 0
AP-0105 (PRESent-5)
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - No Intervention

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 interventions: No Intervention
This is a non-interventional study.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
Timepoint [1] 0 0
Minimum 12 weeks
Primary outcome [2] 0 0
Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
Timepoint [2] 0 0
Minimum 12 weeks

Eligibility
Key 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
Minimum age
12 Years
Maximum age
65 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2024
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment postcode(s) [1] 0 0
NSW 2050 - Camperdown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Michigan
Country [2] 0 0
United States of America
State/province [2] 0 0
South Carolina
Country [3] 0 0
Armenia
State/province [3] 0 0
Yerevan
Country [4] 0 0
Brazil
State/province [4] 0 0
São Paulo
Country [5] 0 0
Brazil
State/province [5] 0 0
Vitória
Country [6] 0 0
Canada
State/province [6] 0 0
Ontario
Country [7] 0 0
Canada
State/province [7] 0 0
Hamilton
Country [8] 0 0
Canada
State/province [8] 0 0
Toronto
Country [9] 0 0
Egypt
State/province [9] 0 0
Cairo
Country [10] 0 0
France
State/province [10] 0 0
Rhone
Country [11] 0 0
France
State/province [11] 0 0
Le Kremlin Bicetre
Country [12] 0 0
France
State/province [12] 0 0
Nantes
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
Germany
State/province [14] 0 0
Hesse
Country [15] 0 0
Germany
State/province [15] 0 0
Sachsen
Country [16] 0 0
Germany
State/province [16] 0 0
Berlin
Country [17] 0 0
India
State/province [17] 0 0
Punjab
Country [18] 0 0
India
State/province [18] 0 0
Bangalore
Country [19] 0 0
India
State/province [19] 0 0
Mumbai
Country [20] 0 0
Italy
State/province [20] 0 0
Florence
Country [21] 0 0
Italy
State/province [21] 0 0
Milano
Country [22] 0 0
Poland
State/province [22] 0 0
Podkarpackie
Country [23] 0 0
Poland
State/province [23] 0 0
Warszawa
Country [24] 0 0
Poland
State/province [24] 0 0
Wroclaw
Country [25] 0 0
South Africa
State/province [25] 0 0
Eastern Cape
Country [26] 0 0
South Africa
State/province [26] 0 0
Durban
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
Malaga
Country [29] 0 0
Spain
State/province [29] 0 0
Murcia
Country [30] 0 0
Spain
State/province [30] 0 0
Zaragoza
Country [31] 0 0
Taiwan
State/province [31] 0 0
Taichung
Country [32] 0 0
Taiwan
State/province [32] 0 0
Taipei City
Country [33] 0 0
Turkey
State/province [33] 0 0
Izmir
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Tyne And Wear
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Canterbury
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Cardiff
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Glasgow
Country [38] 0 0
United Kingdom
State/province [38] 0 0
London
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Manchester
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ApcinteX Ltd
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Centessa Pharmaceuticals plc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05605678
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Centessa Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
617 468 5770
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05605678