A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study, lasting about three years, is observing how a new medicine called pegcetacoplan helps people with Paroxysmal Nocturnal Hemoglobinuria (PNH) in their daily lives. Doctors want to understand its real-world benefits and any side effects. They'll collect information from patients already taking pegcetacoplan, both from before they started treatment and as they continue. This helps provide a clearer picture of how well the medicine works and affects patients' overall health and need for hospital care. The study includes about 200 patients across many countries, helping bridge a knowledge gap about this new treatment.
Överblick
Vad handlar denna studie om?
This study is a bit like gathering a long-term diary about a new medicine called pegcetacoplan. This medicine is for a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. Because pegcetacoplan is quite new, doctors and healthcare providers need to understand how it truly works for people in their everyday lives, not just in strict research settings. This study aims to fill that gap by observing real patients.
The main goal is to see how effective pegcetacoplan is at managing PNH symptoms and how safe it is over a long period. Researchers will also look at things like how often patients need blood transfusions and how much healthcare they use before and after starting treatment. This helps them understand the full impact of the medicine on patients' health and their use of medical services.
About 200 patients from various countries, including Europe, Canada, and Australia, will take part. For each patient, data will be collected for roughly three years. This includes looking back at information from when they first started pegcetacoplan and then continuing to gather updates as they go along. This detailed, long-term look will give a much clearer picture of pegcetacoplan's role in treating PNH.
Viktiga punkter
- Observational study looking at an existing PNH treatment.
- Aims to understand pegcetacoplan's real-world effectiveness and safety.
- Gathers information from routine medical appointments over about three years.
- Helps fill knowledge gaps for doctors and patients about this new medicine.
- Your standard medical care for PNH will not change if you join.
- Information from about 200 patients across multiple countries will be collected.
Vem kan vara berättigad?
To be part of this study, you need to be at least 18 years old and have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH). You also need to have either started taking pegcetacoplan for your PNH within the last 12 months, or your doctor must decide to prescribe it at the time you join the study. Importantly, the decision for you to take pegcetacoplan must be made by your doctor independently of whether you join the study.
You'll also need to be willing to read and sign a consent form, which explains everything about the study, before you can take part.
There are a couple of reasons you might not be able to join. For example, if you're currently in another medical study where you're trying out an experimental medicine, or if you've been in one recently (within the last three months), you wouldn't be eligible. Also, if you initially started pegcetacoplan as part of another research study, you wouldn't be able to join this one.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Är du 18 år eller äldre?
- Do you have a confirmed diagnosis of PNH?
- Are you currently taking pegcetacoplan, or is your doctor planning to start you on it for PNH?
- Are you willing to sign a consent form to participate?
- Are you NOT currently part of another clinical study testing an experimental medicine?
Vad innebär deltagande?
If you join this study, your journey will involve ongoing observation for about 36 months, which is around three years. You won't be given specific study medication; instead, the researchers will collect information about your existing pegcetacoplan treatment, which your doctor has already prescribed for you as part of your normal care. They'll gather data from your routine doctor visits and medical records.
Data will be collected from when you started pegcetacoplan, even if that was up to 12 months before you joined the study. This retrospective data combined with the ongoing collection means they might have information on your treatment for up to six years in total. During the study, they'll check on things like how well the treatment is working, any side effects you experience, and how your health impacts your daily life and need for healthcare.
If you stop taking pegcetacoplan at any point during the study, you'll still be followed for another eight weeks to make sure any lasting effects or side effects are noted. You'll continue to attend your regular appointments with your doctor, and the study will simply collect information from these visits.
Potentiella risker och fördelar
Platser (87)
- Liverpool SjukhusVerifierat postnummerLiverpool, Australia
- Calvary Mater NewcastleVerifierat postnummerWaratah, Australia
- Sunshine Coast University HospitalVerifierat postnummerBirtinya, Australia
- Canberra HospitalVerifierat postnummerGarran, Australia
- Institut Jules BordetVerifierat postnummerAnderlecht, Belgium
- AZ Sint-JanVerifierat postnummerBrygge, Belgien
- CHU Brugmann-Site HortaVerifierat postnummerBryssel, Belgien
- Centre Hospitalier Universitaire de LiegeVerifierat postnummerLiège, Belgien
- AZ HerentalsVerifierat postnummerTurnhout, Belgium
- London Health Sciences Centre (LHSC) - Victoria HospitalVerifierat postnummerLondon, Kanada
- Universitetets hälsovårdsnätverkVerifierat postnummerToronto, Kanada
- Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-MontréalVerifierat postnummerMontreal, Kanada
Vanliga frågor
What is Paroxysmal Nocturnal Hemoglobinuria (PNH)?
PNH is a rare blood disorder where red blood cells are destroyed too early by the body's immune system, leading to symptoms like tiredness, anaemia, and blood clots.
What is pegcetacoplan?
Pegcetacoplan is a newer medicine approved for treating PNH. This study is looking at how well it works and its safety in standard medical care.
Will I get a new medicine if I join the study?
No, you won't get a new medicine as part of this study. It's observing people who are already on or starting pegcetacoplan as part of their routine care.
Hur länge kommer studien att pågå för mig?
You'll be followed for roughly three years from when you join. However, the study will also look at information from when you first started pegcetacoplan, so information might cover a longer period.
Do I have to do anything extra for the study?
No, you don't need to do anything extra. The study will collect information from your existing doctor's visits and medical records.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Diskussion
Gemenskapsdiskussion
Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.