Severe Hemophilia Non-Interventional Study
Research type
Research Study
Full title
A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia
IRAS ID
316858
Contact name
Pratima Chowdary
Contact email
Sponsor organisation
ApcinteX Ltd
Clinicaltrials.gov Identifier
157488, IND Number
Duration of Study in the UK
2 years, 0 months, 2 days
Research summary
The purpose of this prospective, multinational, multisite, non-interventional study is to collect information about bleeding episode(s) and standard treatment use in patients with haemophilia A or B.
The goal of the study is simply to observe the occurrence of bleeding episodes and treatment use under participants current clinical care. After participants consent to take part in the study, past and current medical information will be collected via a patient interview as well as reviewing medical records from the study doctor.
All eligible HemA and HemB participants will be invited for study enrolment by Investigators or their designees from participating specialised centres/hospitals known to treat haemophilia patients in approximately 20 countries across the world. No investigational product will be administered as part of this study.After obtaining informed consent from participants (adolescent assent and parent/legal guardian consent when appropriate), relevant demographic and medical history data available from routine care visits before enrolment will either be extracted from medical records or patient-reported and entered in an electronic case report form.
During prospective follow-up, haemophilia participants will keep an e-diary in which they document bleeding episodes and associated treatment from enrolment until entering a SerpinPC interventional study, the decision against SerpinPC interventional study enrolment, a treatment switch or study discontinuation for any reason; whichever comes first. The Investigator or qualified staff will review bleeding episode e-diary entries and adverse events with the participant monthly by telephone. In case a participant is unable to complete an e-diary, the participant can complete a paper print out of the e-diary.
The information collection will continue for as long as the participant and the study doctor think it is appropriate or until the participant has completed the study. The total study duration is expected to be approximately 2 years.
REC name
London - Dulwich Research Ethics Committee
REC reference
22/PR/1274
Date of REC Opinion
28 Oct 2022
REC opinion
Further Information Favourable Opinion