New hope for PNH patients with stubborn anemia?

NCT ID NCT07187401

First seen Nov 01, 2025 · Last updated May 19, 2026 · Updated 27 times

Summary

This study tests an experimental drug called ALN-CFB in 24 people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if ALN-CFB is safe and tolerable compared to a placebo, and how it affects blood levels of complement factor B. Participants must be on a stable dose of a C5 inhibitor and have hemoglobin at or below 10.5 g/dL.

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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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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Samsung Medical Center

    RECRUITING

    Seoul, 6351, South Korea

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

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Severance Hospital

    RECRUITING

    Seoul, 3722, South Korea

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

  • St. James's University Hospital

    RECRUITING

    Leeds, West Yorkshire, LS97TF, United Kingdom

    Contact Email: •••••@•••••

  • St. Vincent Hospital - The Catholic University of Korea

    RECRUITING

    Suwon, Gyeonggi-do, 16247, South Korea

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

  • The Catholic University of Korea, Seoul St. Mary's Hospital

    RECRUITING

    Seoul, 06591, South Korea

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

  • Toronto General Hospital

    RECRUITING

    Toronto, Ontario, M5G 2C4, Canada

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

Conditions

Explore the condition pages connected to this study.