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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05605678
Recruitment Status : Recruiting
First Posted : November 4, 2022
Last Update Posted : February 20, 2024
Sponsor:
Collaborator:
Centessa Pharmaceuticals plc
Information provided by (Responsible Party):
ApcinteX Ltd

Tracking Information
First Submitted Date October 31, 2022
First Posted Date November 4, 2022
Last Update Posted Date February 20, 2024
Actual Study Start Date December 9, 2022
Estimated Primary Completion Date November 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 31, 2022)
  • Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds [ Time Frame: Minimum 12 weeks ]
    ABR for treated bleeds and all bleeds at the participant and bleed level (excluding bleeding episodes during surgery and/or procedures).
  • Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode [ Time Frame: Minimum 12 weeks ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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
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 Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition
  • Hemophilia A
  • Hemophilia B
Intervention Other: No Intervention
This is a non-interventional study.
Study Groups/Cohorts 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.
Intervention: Other: No Intervention
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 31, 2022)
200
Original Estimated Enrollment Same as current
Estimated Study Completion Date November 2024
Estimated Primary Completion Date November 2024   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: Male
Ages 12 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Centessa Pharmaceuticals 617 468 5770 presentprogram@centessa.com
Listed Location Countries Armenia,   Australia,   Brazil,   Canada,   Egypt,   France,   Germany,   India,   Italy,   Poland,   South Africa,   Spain,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries Barbados
 
Administrative Information
NCT Number NCT05605678
Other Study ID Numbers AP-0105 (PRESent-5)
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: No
Current Responsible Party ApcinteX Ltd
Original Responsible Party Same as current
Current Study Sponsor ApcinteX Ltd
Original Study Sponsor Same as current
Collaborators Centessa Pharmaceuticals plc
Investigators Not Provided
PRS Account ApcinteX Ltd
Verification Date February 2024