New pill combo aims to keep PNH patients stable for years

NCT ID NCT05389449

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

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

Summary

This study follows about 80 people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken danicopan in a previous trial. They will continue taking danicopan as an add-on to their standard C5 inhibitor therapy. The goal is to see if the combination remains safe and helps maintain healthy red blood cell levels over the long term.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: danicopan (oral tablet)
What this could lead to: If successful, this could show that adding danicopan to existing PNH therapy helps maintain better blood health over the long term.
What could go wrong: This is an extension study with no placebo group, so results are limited. It may not prove danicopan works better than standard care alone, and side effects are still being tracked.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal Hemolysis paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

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

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Contacts and locations

Locations

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Los Angeles, California, 90089, United States
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Chicago, Illinois, 60612, United States
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Baltimore, Maryland, 21231, United States
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Kansas City, Missouri, 64111, United States
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Long Island City, New York, 11101, United States
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Cleveland, Ohio, 44195, United States
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Dallas, Texas, 75390-9047, United States
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Porto Alegre, 90110-270, Brazil
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Rio de Janeiro, 20211-080, Brazil
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Toronto, Ontario, M5G 2C4, Canada
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Brno, 625 00, Czechia
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Lille, 59037, France
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Paris, 75010, France
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Pessac, 33604, France
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Pierre-Bénite, 69495, France
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Athens, 11527, Greece
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Thessaloniki, 57010, Greece
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Haifa, 31048, Israel
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Jerusalem, 91120, Israel
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Avellino, 83100, Italy
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Bassano del Grappa, 36061, Italy
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Florence, 50134, Italy
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Milan, 20122, Italy
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Reggio Calabria, 89131, Italy
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Roma, 161, Italy
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Bunkyō City, 113 8603, Japan
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Kyoto, 605-0981, Japan
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Osaka, 530-8480, Japan
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Shibuya-ku, 150-8935, Japan
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Tsukuba, 305-8576, Japan
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Kota Kinabalu, 88586, Malaysia
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Kuching, 93200, Malaysia
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Miri, 98000, Malaysia
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Gdansk, 80-952, Poland
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Seoul, 03722, South Korea
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Seoul, 06591, South Korea
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Suwon, 16247, South Korea
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Badalona, 8916, Spain
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Barcelona, 08036, Spain
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Majadahonda, 28222, Spain
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Seville, 41013, Spain
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Bangkok, 10330, Thailand
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Airdrie, ML6 0JS, United Kingdom
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Leeds, LS9 7TF, United Kingdom
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London, SE5 9RS, United Kingdom

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