Active clinical trials for "Hemoglobinuria, Paroxysmal"

The purpose of this registration form is to list all Managed Access Programs (MAPs) related to LNP023, Iptacopan

This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.

The primary objective is to provide access to eculizumab for PNH patient pending commercial availability.

This is an Early Access Program (EAP) which will provide access to pegcetacoplan for eligible participants with paroxysmal nocturnal hemoglobinuria (PNH).

Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder in which unregulated activation of the complement system leads to significant ischemic morbidities with shortened lifespan. Life-threatening thromboembolism (TE) is the most feared complication of PNH, accounting for up to 45% of patient deaths. It is estimated that 40% of PNH patients experience a clinically evident TE and 60% of patients without clinically diagnosed TE demonstrate TE by high-sensitivity MRI, indicating the ongoing thrombotic risk in most patients with PNH. Much of these data come from PNH patients from European descent. To understand the impact of TE in patients with PNH from non-European regions, we performed this study to evaluate the clinical characteristics of Korean patients with PNH.