New drug danicopan under Real-World review for rare blood disorder

NCT ID NCT07457151

Recruiting now Disease control Sponsor: AstraZeneca Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 6 times

Summary

This study tracks 8 Korean patients with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Danicopan alongside standard C5 inhibitors like eculizumab or ravulizumab. The goal is to monitor safety and check if the drug works as expected in everyday medical practice. It is a post-marketing commitment required by Korean health authorities.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Danicopan
What this could lead to: If successful, this study will confirm that Danicopan is safe and effective for controlling PNH when used alongside standard treatments in real-world settings.
What could go wrong: This is a small, observational post-marketing study with only 8 participants, so results may not apply broadly. It is designed to monitor known risks, not to prove new benefits.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

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

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

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

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Contacts and locations

Locations

Research Site
RECRUITING

Busan, South Korea
Research Site
RECRUITING

Hwasun-gun, South Korea
Research Site
RECRUITING

Seoul, South Korea
Research Site
ACTIVE_NOT_RECRUITING

Seoul, South Korea

More trials for these conditions

Other studies related to the condition(s) this trial covers.