RecruitingOBSERVATIONAL

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.

At a glance

Status

Recruiting

Sponsor

Swedish Orphan Biovitrum

Enrolment target

200

Start

26 Jun 2023

Estimated completion

15 Aug 2029

Paroxysmal Nocturnal Hemoglobinuria

What is this study about?

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.

Key takeaways

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.

Who may be eligible?

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.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
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?

Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

This study is about observing how a medicine you're already taking for PNH works in real life, so it doesn't involve new medicines or procedures. A potential benefit is that the information gathered from you and others will help doctors and researchers better understand how pegcetacoplan works for a wide range of patients, which could improve care for others with PNH in the future. As it's an observational study, the direct risks are generally low, focusing on data collection from your routine care. However, as with any study, there's always a small risk to your privacy, though researchers take steps to protect your personal information. You have the right to withdraw from the study at any time without affecting your medical care.

Locations (75)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Liverpool Hospital
Verified postcode
Liverpool, Australia· Recruiting
Calvary Mater Newcastle
Verified postcode
Waratah, Australia· Recruiting
Sunshine Coast University Hospital
Verified postcode
Birtinya, Australia· Recruiting
AZ Sint-Jan
Verified postcode
Bruges, Belgium· Active not recruiting
CHU Brugmann-Site Horta
Verified postcode
Brussels, Belgium· Active not recruiting
Centre Hospitalier Universitaire de Liege
Verified postcode
Liège, Belgium· Recruiting
AZ Turnhout - Campus Sint-Elisabeth
Verified postcode
Turnhout, Belgium· Active not recruiting
University Health Network
Verified postcode
Toronto, Canada· Recruiting
Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal
Verified postcode
Montreal, Canada· Active not recruiting
Fakultni nemocnice Brno
Verified postcode
Brno, Czechia· Recruiting
Ustav hematologie a krevni transfuze
Verified postcode
Prague, Czechia· Recruiting
Helsingin yliopistollinen keskussairaala
Verified postcode
Helsinki, Finland· Recruiting

Common questions

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.

How long will the study last for me?

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.

How to find out more

Michael O'Malley

michael.omalley@sobi.com +41797977276 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.