Safety and tolerability of APL-3007 administered as a single dose in addition to background therapy with a C5 inhibitor in adults with paroxysmal nocturnal hemoglobinuria

To join this study, you need to be an adult, aged between 18 and 70 years old, and have been diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). You should have been on your current PNH treatment (a C5 inhibitor called ravulizumab-cwvz) for at least six months, and despite this, your blood tests still show signs that your PNH isn't fully controlled, such as low haemoglobin levels (below 10.5 g/dl) and signs of red blood cell breakdown.

There are also specific requirements for women. If you are female, you must either be unable to get pregnant (for example, if you've gone through menopause and haven't had a period for over a year, or have had certain surgeries) or, if you can get pregnant, you must have a negative pregnancy test before starting. If you can get pregnant, you would need to agree to use effective birth control methods throughout the study and not breastfeed.

Finally, you would also need to have proof of having received vaccinations against certain infections like pneumonia and meningitis. The study team will review all your medical information carefully to see if you meet all the requirements.

Taking part in this study means you would receive a single dose of the study medicine, APL-3007, in addition to your usual PNH treatment. The study team would closely monitor you before, during, and after you receive this dose. This would involve regular visits to the clinic for medical check-ups, blood tests, and to answer questions about how you are feeling. The total duration of your active participation in the study, including follow-up checks after receiving the medicine, would be explained in detail by the study team. They will ensure you understand what each visit involves and how long your commitment would be.