COMPLETE
Research type
Research Study
Full title
A single arm, long term, multicentre observational study to evaluate effectiveness of pegcetacoplan under real world conditions in patients with paroxysmal nocturnal hemoglobinuria (PNH)
IRAS ID
326847
Contact name
Roochi Trikha
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AB (publ)
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
Clinicalrials.gov, NCT05776472
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
This is a 24-month multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with Paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare acquired chronic disorder of blood cells. The sponsor of this study is Swedish Orphan Biovitrum AB (publ). The study plans to include approximately 200 patients at 70 sites in Europe, Middle East, Canada and Australia. Patients meeting the eligibility criteria will be enrolled in the study at the enrollment visit and followed prospectively for 24 (+/- 3) months. The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective, collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use. The study will collect retrospective data before pegcetacoplan treatment start, which will consist of information on PNH treatment, blood transfusions and healthcare resource use. Retrospective data will be collected for up to 12 months prior to pegcetacoplan treatment start. As patients may have been treated with pegcetacoplan for up to 12 months prior to enrollment retrospective data may be collected for up to 24 months. This means that the total data collection period including both, the retrospective and the prospective part is up to 48 (+/- 3) months. Due to the observational nature of the study the protocol does not dictate any visit schedule and the patients visit their treating physician as per clinical routine. Data collection is expected to occur every 6 months starting from enrollment and is based on data available in the medical records as collected per clinical routine.
REC name
West of Scotland REC 3
REC reference
23/WS/0088
Date of REC Opinion
14 Jun 2023
REC opinion
Further Information Favourable Opinion