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

NCT ID NCT07457151

First seen Jun 25, 2026 · Last updated Jun 30, 2026 · Updated 3 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.

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.

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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.

Contacts and locations

Study contacts

  • Contact

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

Locations

  • Research Site

    RECRUITING

    Hwasun-gun, South Korea

  • Research Site

    RECRUITING

    Seoul, South Korea