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New drug combo aims to boost red blood cells in kids with rare blood disorder

NCT ID NCT06449001

Recruiting now Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participants will receive weight-based danicopan doses for 12 weeks, with follow-up on blood counts, transfusion needs, and quality of life. The goal is better disease control, not a cure.

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 it works, this could offer a new add-on treatment option to better control anemia in children with PNH who still have symptoms despite current therapy.
What could go wrong
This is a very small early-phase trial with only 6 participants, so results may not apply broadly. The drug is added to existing treatments, not a standalone cure, and risks include infection and side effects from the combination.

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

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal Hemolysis hemolytic anemia paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

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

EXTRAVASCULAR HEMOLYSIS PAROXYSMAL NOCTURNAL HEMOGLOBINURIA PNH

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Research Site

    RECRUITING

    Saskatoon, Saskatchewan, S7N 0W8, Canada

  • Research Site

    RECRUITING

    Paris, 77019, France

  • Research Site

    RECRUITING

    Leeds, LS9 7TF, United Kingdom

  • Research Site

    RECRUITING

    London, SE5 9RS, United Kingdom

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