New hope for rare blood and kidney diseases: early access to iptacopan

NCT ID NCT05222412

First seen Jan 05, 2026 · Last updated May 14, 2026 · Updated 23 times

Summary

This program offers early access to the drug iptacopan for people with serious conditions like C3 glomerulopathy (a kidney disease) or paroxysmal nocturnal hemoglobinuria (a blood disorder) who have no other treatment options. Doctors can request the drug for patients who are not eligible for clinical trials or have exhausted all standard therapies. The goal is to provide a potential treatment when nothing else is available.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Study contacts

  • Contact

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

  • Contact

    Phone: •••-•••-••••

Conditions

Explore the condition pages connected to this study.