Paroxysmal Nocturnal Hemoglobinuria research hub
10 indexed studies · 3 currently recruiting
- active not recruitingPHASE2
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria
This study looks at a new drug, OMS906, for a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). It checks how safe it is, if people can tolerate it, and if it helps over a long time. Patients get the drug through an IV every eight weeks.
Germany · Switzerland · Ukraine - recruitingPHASE3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
This study looks at two new drugs, pozelimab and cemdisiran, for a rare blood condition called PNH. Researchers want to see if the combination is safe and works well over a long time for adults with PNH.
Canada · Colombia · Hungary - recruiting
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study looks at how a new PNH treatment, pegcetacoplan, works in real life. It aims to see how effective and safe it is for patients with Paroxysmal Nocturnal Hemoglobinuria over several years.
Australia · Belgium · Canada - active not recruitingPHASE3
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
This study compares two medicines, crovalimab and eculizumab, for a rare blood condition called PNH. It's for people who haven't tried these types of medicines before, checking if crovalimab works as well and is as safe as eculizumab.
Argentina · Brazil · China - active not recruitingPHASE3
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
This study looks at the long-term safety and effects of a medicine called iptacopan for people with a rare blood condition called PNH. It's for patients who have already been taking iptacopan in other studies and found it helpful, to make sure they can continue treatment safely.
United States · Brazil · China - active not recruitingPHASE3
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
This study compares a new medicine, crovalimab, to an existing one, eculizumab, for people with a rare blood condition called PNH. It's looking at how safe and effective crovalimab is in patients already receiving similar treatments.
United States · Belgium · Brazil - recruitingPHASE3
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
This study looks at a new add-on medicine, danicopan, for young people (12-17 years old) with a rare blood condition called PNH. It aims to see if danicopan can improve red blood cell levels when added to their current treatment.
Canada · France · United Kingdom - active not recruitingPHASE3
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This study looks at the long-term safety and effectiveness of a drug called Danicopan. It's for people with a rare blood condition called PNH who are already taking another PNH medicine, a C5 inhibitor. Danicopan is given as an add-on treatment.
United States · Brazil · Canada - active not recruitingPHASE3
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
This study looks at a new drug combination, pozelimab and cemdisiran, for adults with a rare blood condition called PNH. We want to see how safe and effective it is compared to existing treatments, ravulizumab and eculizumab. It's for people who haven't recently had certain PNH treatments.
United States · Brazil · Canada - ongoing, recruitingHuman Pharmacology (Phase I)- Other
A Phase 2 Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in PNH Patients with a Sub-optimal Response to the C5 inhibitor, Ravulizumab
This study is testing a new medicine, OMS906, for adults with a rare blood condition called paroxysmal nocturnal hemoglobinuria (PNH). It's for people whose current PNH treatment (ravulizumab) isn't working as well as it could. We want to see if OMS906 is safe and helps improve their condition.
Greece · Germany