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We support a community of people impacted by paroxysmal nocturnal hemoglobinuria (PNH). We share insights about PNH, and information about PNH clinical trials.

Scroll down to view our doctor-approved Questions & Answers section about PNH. There, you can find insights about PNH, review important medical terms and learn about the purpose of Roche’s new COMPOSER clinical trial for PNH.

People with PNH may follow a lengthy and uncertain path leading up to a confirmed diagnosis

It is our wish that this website can support you with knowledge and insights about clinical trials for PNH. The more you know, the more you can participate in your own treatment decisions and support others facing a PNH diagnosis.

Today’s clinical trials have a critical impact on how PNH may be treated in the future. Clinical trials may reveal that an investigational treatment, the new medical treatment being studied in the clinical trial, is safe and more effective than the standard treatments available.

Clinical trials can also find out whether an investigational treatment brings down the burden of treatment on your day-to-day life.

How do we support you as you learn about clinical trials?

We can put you in contact with people who can answer your questions about Roche clinical trials. These people can be reached by email and you can arrange to be contacted over the phone.

Use the link below to find the nearest contact person that is able to answer your questions.

Have you talked to your doctor about participating in a PNH clinical trial? We provide useful information to discuss clinical trials with your doctor. This information is presented in clear terms and without confusing jargon.

PNH is a rare disease and a diagnosis with PNH can be life-changing. You are unlikely to meet other people with PNH on a day-to-day basis. This may feel very isolating, especially as PNH requires prompt treatment.

Online communities enable you to reach others who face a similar diagnosis. Many people find it helpful to follow patient-led social media activities and to connect online with others who are impacted by PNH.

Questions and Answers about PNH

What do you know about your PNH diagnosis?

Paroxysmal nocturnal hemoglobinuria is an extremely rare disease in which red blood cells develop a vulnerability to attack, and destruction, by the body’s own immune system.

Normal red blood cells are shielded from such an attack. A protein on the surface of normal red blood cells, called PIG-A, prevents the immune system from launching an attack on them.

The vulnerable red blood cells of a PNH patient, which doctors call your PNH clone, have a defect in this shielding protein. Without a working shield the PNH red blood cells are attacked uncontrollably.

The cells break apart in excessive numbers and release hemoglobin into the blood stream.

This is called hemolytic anemia.

How does hemolytic anemia affect my health? Why does hemolysis need to be managed?

The excessive breakdown of red blood cells causes many PNH symptoms. Anemia, which means a lack of red blood cells, contributes to some of the symptoms. It causes fatigue, a strong feeling of tiredness that does not get better with rest.

When excessive hemolysis stops, quality of life improves

The release of hemoglobin into the blood stream creates other problems. When hemoglobin is inside red blood cells it is able to transport life-giving oxygen around your body.

When released into the blood stream, hemoglobin increases the risk of life-threatening blood clots. Thrombosis is the medical term for blood clots. Thrombosis can cause strokes, breathing events and heart attack. The kidneys are also affected by the excessive hemolysis in PNH.

Managing the excessive hemolysis associated with PNH is therefore critical.

Various treatments for PNH patients manage hemolysis in different ways. A blood transfusion provides a temporary respite by replacing PNH red blood cells with healthy ones.

Blood thinners can lower the chances of life-threatening blood clots. However, blood transfusions and medications to thin the blood do not target the excessive hemolysis directly. These treatments do not reduce the attack and destruction PNH red blood cells by the immune system.

In some countries patients may have access to a treatment that targets the immune system selectively. The treatment targets the so-called complement system, the part of the immune system that is responsible for attacking PNH red blood cells.

This targeted approach to treating PNH is the focus of ongoing clinical trials to improve the lives of people living with PNH.

What is the purpose of Roche’s new COMPOSER PNH trial?

Clinical trials are needed to ensure that patients see continued improvements to the treatment of PNH. When an investigational treatment brings greater benefits compared to existing therapies, that treatment might one day become the new standard of care for PNH.

Roche is investigating a next-generation targeted treatment for PNH and is now conducting clinical trials at hospitals around the world.

All participants in these trials will receive the COMPOSER investigational treatment. No patients enrolled on the trial will receive a placebo drug as treatment.

Who will pay the costs associated with being part of the trial?

Roche will cover the cost of study medication, visits, assessments and reasonable costs of travel.

Roche also provides a free and optional service to support day-to-day participation in the COMPOSER trial. This service is provided through a convenient smart phone App.

The App contains information about the trial and instructions about how to participate in the trial. The App sends reminders for medical appointments relating to the trial, and enables you to define and track personal goals as you progress through the study.

What is a clinical trial? - Watch the explainer video

Our clinical trials are conducted in hospitals by medical teams who are specialized in the treatment of PNH.

A team of doctors, members of the general public and health authorities review each clinical trial. Formal approval by this team is needed before the clinical trial can begin.

Every trial is monitored closely by the healthcare team providing the investigational treatment and by the health authorities.

To join a trial, a medical team must carefully evaluate whether you are eligible to participate.

This evaluation is made on a case-by-case basis. The team conducting the clinical trial must consider medical test results to decide if you are eligible.

It is important to make this evaluation with the clinical trial team as early as possible. Even if you have a confirmed PNH diagnosis, the team may never-the-less find that you are not eligible to participate in our current trial.

The information on this website may support you and your doctor to understand our clinical trial. If you are eligible you will then have the option to give your consent to take part, and to receive our investigational treatment.

Thank you for stopping by and learning about Roche’s clinical trials for PNH.

A confirmed PNH diagnosis may bring a clearer explanation for some of the symptoms you have experienced

Perhaps someone in your health care team has mentioned a clinical trial for PNH? Follow the Find My Trial link to locate the nearest hospital studying Roche’s investigational treatment for PNH.

Participation in a clinical trial may contribute to the development of better treatments for PNH.