Paroxysmal Nocturnal Hemoglobinuria research hub

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.

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.

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.

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.

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.

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.

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.

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.

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.

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