A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

To be considered for this study, you need to be between 12 and 17 years old and weigh at least 20 kilograms (about 3 stone 2 pounds). You must have a confirmed diagnosis of PNH.

Even if you're already taking other PNH medicines (like eculizumab or ravulizumab), you might still be able to join, especially if you still have anaemia symptoms. If you're not on those medicines, you'll need to have signs of anaemia, which means low red blood cell counts and certain blood test results.

There are also some reasons you wouldn't be able to join. For example, if you're pregnant or breastfeeding, or if you have a history of certain medical conditions like hereditary complement deficiency (a different immune system problem), a bone marrow transplant, or meningococcal disease (like meningitis). You also can't have a known problem with how your body handles fructose (a type of sugar).

If you decide to take part, the first step is a 4-week screening period where doctors will do tests to see if the study is right for you. If you're currently on a different PNH medicine, there might be an extra 4-week period to carefully switch you over.

After screening, you'll enter a 16-week treatment period. During this time, you'll receive the study medicine, pegcetacoplan, twice a week. It's given as an injection under the skin, which you or your caregiver will be taught to do at home. This involves two small needles placed into the fatty layer just under your skin, through which the medicine flows.

Once the 16-week treatment is over, you might have the option to join a longer-term study to keep receiving the treatment, or you'll have a 2-month follow-up period where doctors will monitor your health.