New hope for PNH patients with stubborn anemia

NCT ID NCT07187401

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

Summary

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive either the drug or a placebo, and researchers will monitor side effects and blood levels.

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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.

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

  • Kings College Hospital NHS Foundation Trust

    RECRUITING

    London, SE5 9RS, United Kingdom

  • Samsung Medical Center

    RECRUITING

    Seoul, 6351, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Severance Hospital

    RECRUITING

    Seoul, 3722, South Korea

  • St. James's University Hospital

    RECRUITING

    Leeds, West Yorkshire, LS97TF, United Kingdom

  • St. Vincent Hospital - The Catholic University of Korea

    RECRUITING

    Suwon, Gyeonggi-do, 16247, South Korea

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

    RECRUITING

    Seoul, 06591, South Korea

  • Toronto General Hospital

    RECRUITING

    Toronto, Ontario, M5G 2C4, Canada