New hope for rare blood and kidney diseases: iptacopan access program opens

NCT ID NCT05222412

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exhausted all available therapies. The goal is to offer a potential treatment when no satisfactory alternative exists.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

complement 3 glomerulopathy Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-••••