At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Pegcetacoplan (drug)
- How long the study runs
- Study runs about 74 months (dates as stated)
- About the drug or intervention
- Pegcetacoplan — drug: Pegcetacoplan will be prescribed according to the label in patients with PNH.
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at how well pegcetacoplan works in real life for adults with paroxysmal nocturnal haemoglobinuria (PNH), a rare blood condition. It is run by Swedish Orphan Biovitrum. The study does not decide who gets the medicine — doctors prescribe it as part of normal care.
Who can take part
- Adults aged 18 or over with a diagnosed PNH condition
- Started pegcetacoplan treatment up to 12 months before joining, or were prescribed it when joining — this choice was made by their doctor, not by the study
- Willing and able to give written informed agreement to take part, following local rules
Who may not be able to
- Taking part in another clinical trial of a treatment, or taking an investigational (unapproved) medicine, within three months before starting pegcetacoplan
- Started their current pegcetacoplan treatment as part of a clinical trial
What taking part involves
- • Pegcetacoplan, prescribed by the doctor as part of routine care
- • Other study activities: Not stated — ask the trial team
Time commitment: Visits and study length: Not stated — ask the trial team. Taking part means receiving pegcetacoplan as prescribed in normal care and agreeing to be observed in the study.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- 18 Years and over
- Who
- All
- Number of participants
- 165
- Started
- 2023-06-26
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for paroxysmal nocturnal hemoglobinuria
- • Clinical study - 165 participants
- • This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH
Who can take part?
- • Ages 18 Years and over
- • Diagnosed with paroxysmal nocturnal hemoglobinuria
Where?
- • London - King's College Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.
More detail
As pegcetacoplan is a new product on the market, with a new mechanism of action, there is an urgent need to provide data to treaters, payers and the PNH community on the real-world usage and effectiveness of pegcetacoplan. This study aims to fill part of that knowledge gap and to add to the knowledge base regarding the use of pegcetacoplan in routine medical practice. Another important rationale for this study is to provide information on RBC transfusions and health care resource utilization pre and post pegcetacoplan treatment initiation. The study plans to include approximately 200 patients at 70 sites in Europe, Middle East, Canada and Australia. Additional countries may be added in the study if necessary. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. Patients will come to their routine visits and the available data from each visit will be collected. 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 also collect retrospective data before pegcetacoplan treatment start, which will consist of information on PNH treatment, blood transfusions and healthcare resource use. 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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years and over
- Who can join: All genders
What the study is looking for
- ✓Patients ≥18 years of age with a documented PNH diagnosis.
- ✓Patient is willing and able to provide written agreement to take part to participate in the study in a manner approved by...
Who cannot take part
- ✗Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP),...
- ✗Initiated current treatment with pegcetacoplan in an interventional study.
See the full criteria
Where Is This Study? (1 UK site)
King's College Hospital
London, United Kingdom