Paroxysmal Nocturnal Hemoglobinuria (PNH) research hub
7 indexed studies · 4 currently recruiting
- recruitingPHASE1, PHASE2
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
This study looks at a new drug, ALN-CFB, for people with a blood condition called PNH who have ongoing anaemia despite current treatment. It aims to see how safe and effective the new drug is compared to a dummy drug.
Canada · South Korea · United Kingdom - recruitingPHASE2
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study is for young people aged 12-17 with a blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). It's testing a new medicine, pegcetacoplan, to see if it's safe and effective, and how it works in the body.
United States · Czechia · France - authorisedTherapeutic confirmatory (Phase III)
A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria with Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy
This study looks at a new drug combination, Pozelimab and Cemdisiran, for adults with a rare blood condition called PNH. It aims to see if these drugs can better control the condition for those whose current treatments aren't working well enough. The main goal is to reduce a marker of blood cell damage.
Poland · Italy · Spain - recruiting
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
This study looks at the safety of Ultomiris (a medicine) during pregnancy and breastfeeding. It checks for any effects on the pregnant person and the baby, from before birth up to the baby's first birthday. It’s for people who have already used Ultomiris.
United States · Australia · France - recruitingPHASE1, PHASE2
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
This study is testing a new medicine called ADX-038 for a rare blood condition called PNH. It's looking at how safe it is, what effects it has on the body, and how the body handles it, first in healthy people and then in PNH patients.
Australia · United Kingdom - recruitingPhase I
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
This study is testing a new medicine, APL-3007, for adults with a rare blood condition called PNH. We want to see how safe it is and if people can take it well alongside their usual PNH treatment. It's an early-stage study to gather important safety information.
United Kingdom · England - ongoing, recruitingTherapeutic confirmatory (Phase III)
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan
This study looks at a new medicine called iptacopan for adults with a rare blood condition called PNH. It checks if switching to iptacopan is safe and works well for patients who are already feeling better on other treatments.
Spain · Germany · France