Active not recruitingPHASE3INTERVENTIONAL

Pegcetacoplan Long Term Safety and Efficacy Extension Study

This research is an ongoing study for individuals in the UK who have a blood condition called PNH (Paroxysmal Nocturnal Hemoglobinuria) and have previously taken part in a trial for the medicine pegcetacoplan. It’s designed to closely monitor the long-term safety and effectiveness of pegcetacoplan. The study is 'open-label', meaning both you and the study team will know you're receiving the treatment. It's also 'non-randomized', so there's no chance element involved in which treatment you get, as you'll be continuing with pegcetacoplan. The study is taking place in several different medical centres. Your doctor must believe that pegcetacoplan is helping you or is likely to help you to join.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Apellis Pharmaceuticals, Inc.

Enrolment target

137

Start

27 Aug 2018

Estimated completion

01 Jul 2026

PNH

What is this study about?

This study is like a continuation of previous research into a medicine called pegcetacoplan, which is used to treat a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. If you've already been part of a trial for pegcetacoplan, and your doctors think it's been helpful for you, this study offers a chance to keep receiving the treatment and be closely monitored over a longer period.

The main aim of this study is to gather more information about how pegcetacoplan works over many months or years, and to keep an eye on any side effects that might appear with long-term use. This kind of research is really important because it helps doctors and scientists understand how medicines work in real-world situations and if they remain safe and effective over time. This information can then help more people with PNH in the future.

Because this is an 'extension study', it means that everyone who joins will continue to receive pegcetacoplan. It’s not a study where you might get a placebo (a dummy treatment) or a different drug. The study is run by different hospitals and clinics, which helps gather varied experiences. By joining, you're contributing valuable information that can improve the understanding and treatment of PNH.

Key takeaways

This study is for long-term monitoring of pegcetacoplan.
You must have previously participated in a pegcetacoplan trial.
Everyone in this study will receive pegcetacoplan.
Regular clinic visits and blood tests will be part of your participation.
Vaccinations are required to join the study.
You can leave the study at any time without affecting your medical care.

Who may be eligible?

To be considered for this study, you must be at least 18 years old and have PNH. A key requirement is that you've already taken part in a clinical study involving pegcetacoplan. For most people, your doctors need to be confident that pegcetacoplan has been helping you and that you've tolerated it well.

However, even if you were in a previous pegcetacoplan study but didn't receive the treatment for long, or if you didn't receive pegcetacoplan at all, you might still be able to join. In these cases, your study doctor would need to believe that starting or continuing pegcetacoplan would benefit you.

Before starting, you would also need to have certain vaccinations up to date, or be willing to get them. This is to protect you from specific infections while on treatment. If you are a woman who could become pregnant, you'd need to have a negative pregnancy test and agree to use effective contraception. Men also need to agree to use contraception and not donate sperm during the study and for 90 days afterwards.

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 at least 18 years old?
Do you have PNH?
Have you been part of a previous pegcetacoplan research study?
Are you up-to-date with specific vaccinations, or willing to get them?
If you are a woman who could get pregnant, are you willing to use effective contraception?
If you are a man, are you willing to use effective contraception and not donate sperm?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will continue to receive the pegcetacoplan treatment, which you, or a caregiver, will learn to give yourself. You'll have regular visits to the clinic so the study team can check on your health and how the treatment is working. This will likely involve blood tests and other examinations.

Before you start, and periodically throughout the study, you'll need to make sure your vaccinations are up-to-date for certain infections like meningococcal disease. The study team will help you check your vaccination status and arrange any necessary booster shots. The total duration of your participation will depend on the study plan, but these types of extension studies can run for several years, providing ongoing access to the treatment.

Potential risks and benefits

A potential benefit of joining this study is continued access to pegcetacoplan, which your doctor believes is helping you manage your PNH. Your health will be closely monitored by a dedicated medical team. As with any medication, there are potential risks and side effects associated with pegcetacoplan, and these will be explained to you in detail by the study team. You always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (62)

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

University of Southern California
Verified postcode
Los Angeles, United States
Denver Health Medical Center
Verified postcode
Denver, United States
Cancer Specialists of North Florida
Verified postcode
Jacksonville, United States
Lakes Research
Verified postcode
Miami, United States
Northwestern University
Verified postcode
Chicago, United States
Investigative Clinical Research of Indiana
Verified postcode
Indianapolis, United States
Duke University Medical Center
Verified postcode
Durham, United States
Cleveland Clinic
Verified postcode
Cleveland, United States
Baptist Cancer Center
Verified postcode
Memphis, United States
Royal Melbourne Hospital
Verified postcode
Melbourne, Australia
Cliniques Universitaires Saint-luc
Verified postcode
Brussels, Belgium
AZ Delta
Verified postcode
Roeselare, Belgium

Common questions

What is PNH?

PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare, chronic blood disorder where your red blood cells are destroyed too soon, which can lead to various symptoms like anaemia and fatigue.

What is pegcetacoplan?

Pegcetacoplan is a medicine used to treat PNH. It works by targeting a part of the immune system that causes red blood cells to be destroyed.

Will I receive a placebo in this study?

No, this is an 'open-label' study and an 'extension' study. Everyone who joins will receive pegcetacoplan, not a placebo.

Why do I need vaccinations to join?

Some treatments for PNH, including pegcetacoplan, can increase your risk of certain infections, particularly meningitis. Being vaccinated beforehand helps protect you against these risks.

How long will the study last for me?

The study is designed for long-term monitoring, and your individual participation length will be discussed with you by the study team. It could potentially last for several years.