New hope for kids with rare blood disease: Add-On pill may boost red blood cells

NCT ID NCT06449001

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

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 26 times

Summary

This study tests whether adding danicopan (a pill) to standard PNH therapy (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria (PNH) who still have anemia due to extravascular hemolysis. About 6 participants will take danicopan for 12 weeks, and doctors will measure changes in blood counts, transfusion needs, and quality of life. The goal is to better control the disease, not cure it, as lifelong treatment is still needed.

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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

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Study contacts

Contact

Phone: •••-•••-•••• 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

Conditions

Explore the condition pages connected to this study.

EXTRAVASCULAR HEMOLYSIS PAROXYSMAL NOCTURNAL HEMOGLOBINURIA PNH